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Lemtrada (alemtuzumab)

Lemtrada

 

 

 

 

 

 

 

MS drugs Lemtrada and Aubagio launched in Ireland(03/09/14)

Genzyme's plans to grow its multiple sclerosis business in Europe continued with the launch of two new treatments in the Republic of Ireland.

The company, which serves as the biotech arm of French pharma firm Sanofi, today launched the injectable Lemtrada (alemtuzumab) in the country just weeks after oral multiple sclerosis (MS) treatment Aubagio (teriflunomide) hit the market.

Lemtrada will be made available to adults with active relapsing-remitting multiple sclerosis (RRMS), in accordance with its approval from the EC. It is to be given as an intravenous infusion in two annual treatment courses.

Aubagio is also available for adults with active RRMS, although it is one of several new MS treatments that come in an oral formulation, offering greater convenience to patients. It is to be taken once daily.

According to Genzyme, there are 8,000 people in Ireland with MS and around 85% will be affected by RRMS.

The launch of Lemtrada follows a recommendation in July this year from the National Centre for Pharmacoeconomics (NCPE), which provides guidance on what drugs should be reimbursed on Ireland's healthcare system to the Health Service Executive (HSE).

By contrast, Aubagio was turned down by the NCPE in June with after its assessment found that the drugs cost was not justified by its benefits.

However, in a conversation with PMLiVE, Henry Featherstone, director of public affairs at Genzyme UK & Ireland confirmed that Genzyme has since held discussions with the HSE and they have agreed that the drug can be reimbursed in Ireland.

As for where the drugs fit on the MS treatment pathway, Featherstone said that the broad indications for both products allowed doctors to discuss suitable options with patients. Further down the line, however, it's possible that Aubagio will be better suited as a first-line treatment for people with MS, while Lemtrada will be reserved for more aggressive forms of the disease.

"We hope to have these treatments available to as many people with MS as possible," Featherstone told PMLiVE. "We passionately believe in these products."

Backing Featherstone's belief, both drugs are making headway in western Europe where Lemtrada had revenues of €10m for the first six months of the year and Aubagio had revenues of €38m.

It's a different story for Lemtrada in the US, however, as the drug was turned down by the FDA at the start of 2014, much to the shock of Genzyme and Sanofi.

The decision, which was based on concerns over the drug's safety, drew criticism from the healthcare community and dozens of US doctors added their name to an open letter to the FDA to appeal the negative guidance.

Source: PMLive © PMGroup Worldwide Ltd 2014 (03/09/14)

Scottish Medicines Consortium approves Lemtrada for RRMS within NHS Scotland(08/07/14)

"Scotland has one of the highest rates of multiple sclerosis in the world, and the approval of Lemtrada in Scotland is an important step forward for people with active RRMS who remain in need of new treatment options. MS treatments have come a long way in the past twenty years and the availability of Lemtrada provides an opportunity for neurologists to offer a new therapy to people with multiple sclerosis," commented Dr. Belinda Weller, Consultant Neurologist, Western General Hospital, Edinburgh.

The Scottish Medicines Consortium (SMC) today published its advice that Lemtrada has been accepted for use within NHS Scotland for the treatment of adult patients with relapsing-remitting multiple sclerosis (RRMS), with active disease defined by clinical or imaging features.

"RRMS accounts for eighty-five percent of all initial diagnoses in MS. We are pleased that after many years in development, Lemtrada is now available to patients in Scotland. This provides people with MS with an important and innovative treatment option to consider in partnership with their MS specialists," said Amy Bowen, Director of Service Development at the MS Trust.

There are approximately 10,000 people living with MS in Scotland. The majority of people with RRMS experience approximately one or two relapses per year. Around half of all relapses may leave people with lingering problems and disability may accumulate over time.[

Lemtrada is the second of Genzyme's treatments for MS to receive approval for use from the SMC and become available for use within NHS Scotland.Lemtrada has also been approved by NICE and is available for NHS patients in England & Wales.

"We are thrilled by today's news that the SMC has approved Lemtrada for NHS use for people with RRMS. At Genzyme, patients at the heart of everything we do and this final milestone brings a treatment option to people with MS that could really reshape the management of their condition. We are also immensely proud of our association with Lemtrada as a home-grown product, developed and pioneered in Cambridge by a team of UK scientists. This reminds us of the UK's position at the forefront of science-led medicine, the importance of industry collaboration which brings global expertise in clinical development and our joint commitment to MS patients," commented Brendan Martin, General Manager for Genzyme UK and Ireland.

Source: Medwatch Copyright © 2014 MarketWatch, Inc (08/07/14)

US FDA accepts MS drug Lemtrada resubmission for review(03/06/14)

The US Food and Drug Administration (FDA) has accepted for review the Genzyme's resubmission of supplemental Biologics License Application (sBLA) seeking approval of Lemtrada (alemtuzumab) for the treatment of relapsing forms of multiple sclerosis. A six-month review period has been assigned for the Lemtrada sBLA. Genzyme expects FDA action on the sBLA in the fourth quarter.

This resubmission is based on data from the same clinical studies included in the original sBLA, and provides supplemental analyses and additional information to specifically address issues previously noted by the FDA in its December 27, 2013 Complete Response Letter. The company resubmitted the sBLA earlier this month following constructive discussions with the agency.

Source: PHARMAbiz.com Copyright © 2010 Saffron Media Pvt. Ltd (03/06/14)

Alemtuzumab: Do the benefits outweigh the risks in treating multiple sclerosis? (01/06/14)

Serious questions remain regarding the side effects and benefits associated with the use of alemtuzumab for the treatment of multiple sclerosis.

The 2014 Annual Meeting of the Consortium of Multiple Sclerosis Centers (CMSC) and the Sixth Cooperative Meeting with Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) concluded Saturday with “Controversies in the Treatment of Multiple Sclerosis,” a debate-style session that focused on discussion of several hot-button issues in current practice. The third and final segment, “Controversy III: Alemtuzumab is Too Toxic to Use in Most Patients with MS,” featured a debate over the benefit vs. risk profile of alemtuzumab, an anti-CD52 monoclonal antibody that is currently approved for the treatment of several forms of leukemia and lymphoma and is being studied for the treatment of multiple sclerosis.

Late last year, the FDA rejected the drug for the treatment of multiple sclerosis, saying that more evidence from high-quality clinical trials was need regarding the benefits and risks of the drug in this patient population.

One participant in the debate session, Olaf Stüve, MD, PhD, said “there is a need for more effective yet safe therapies; currently available treatment options are only partially effective and have potentially life-threatening side effects.”

Stüve is associate professor in the Department of Neurology and Neurotherapeutics and associate professor in the Department of Immunology at University of Texas Southwestern Medical Center, and chief of the Neurology Section at VA North Texas Health Care Systems.

The clinical phase II CAMMS223 study (which was not an open-label study) compared alemtuzumab with interferon beta-1a in patients with early, active relapsing-remitting multiple sclerosis. Over five years of follow up, researchers found that treatment with alemtuzumab lowered the risk of sustained accumulation of disability by 72% compared with interferon beta-1a. Treatment with alemtuzumab also lowered relapse rate by 69% compared with interferon beta-1a. In the CARE-MS I trial, treatment with alemtuzumab led to a 55% risk reduction in relapses compared with interferon beta-1a.

Stüve also noted that in CARE-MS II the secondary co-primary endpoint (sustained accumulation of disability) was met and there was a 42% improvement in risk reduction in the alemtuzumab group compared with the interferon beta-1a group.

“Alemtuzumab is highly effective; with it patients can be treated early to prevent accumulation of disability,” he said.

“There is no need to treat patients long-term, sides effects are mostly mild to moderate, disease and organ specific, and a pharmacovigilance program could be established to screen patients,” said Stüve.

Fellow debater, Mitchell Wallin, MD, MPH, disagreed, pointing out that there are “serious GI side effects” associated with treatment with alemtuzumab, including abdominal pain and flushing.

Wallin is Clinical Associate Director of the Multiple Sclerosis Center of Excellence based in the Baltimore Veterans Affairs (VA) Medical Center/University of Maryland, and associate director of Clinical Care and associate professor of medicine at Georgetown University.

Safety issues potentially preventing the approval of alemtuzumab “include autoimmune cytopenias, nephropathy, serious infections, and malignancies,” said Wallin.

He noted there are also concerns over treatment adherence and challenges of maintaining long term injection. Adherence to self-administered disease-modifying therapies in multiple sclerosis has been the subject of several papers. Based on study results, between 12-87% of patients with multiple sclerosis do not adhere to their disease-modifying therapies.

In addition, because of the risk for life-threatening infections, rapid diagnosis of autoimmune cytopenias is imperative. He said the company had recommended monitoring complete blood count in patients.

If approved for the treatment of multiple sclerosis, the drug would have to have labeling regarding the risks, said Wallin.

Source: Copyright HCPLive 2006-2013 Intellisphere, LLC. All Rights Reserved. (01/06/14)

NHS patients to get new MS drug(28/05/14)

- Scientists have spent 25 years developing treatment at Cambridge
- Alemtuzumab infusion is given in two short courses over two years
- Despite costing £56,000, NICE has ruled treatment is cost-effective

A new treatment for Multiple Sclerosis not only stops the disease from advancing but may help patients recover from disability.

Remarkable results for the drug alemtuzumab mean it has been approved for use on the NHS and is now available in England.

Originally a pioneering cancer therapy, Cambridge University scientists have spent almost 25 years developing it as a treatment for MS patients.

Trials involving more than 1,500 patients show treatment led to fewer relapses compared with multiple jabs of the treatment beta interferon each week, cutting further disability and even allowing some existing damage to recover.

Alemtuzumab is given in two short courses with one infusion a day for five days during the first year and three days during the second year followed by regular monitoring. Despite the £56,000 price tag, the drug has got the go-ahead as cost-effective from the rationing watchdog the National Institute for Health and Care Excellence (Nice). Professor Alastair Compston, Professor of Neurology and the Head of the Department of Clinical Neurosciences at the University of Cambridge, said the drug worked in the early stages of MS, but did not help patients with advanced disease.

Multiple Sclerosis affects almost 100,000 Britons, causing attacks or relapses that may lead to progressive loss of physical skills, sensation, vision, bladder control, and intellectual abilities.

About 2,500 people are diagnosed each year, with four out of five having relapsing-remitting MS - many of them in their 20s and 30s.

Prof Compston said more than half of patients with this type of MS could benefit.

He said ‘This is a real step forward and brings to a conclusion work involving a number of research groups in Cambridge, stretching back to 1991.

‘The decision from Nice now provides an opportunity for neurologists to offer a highly effective therapy for patients with multiple sclerosis early in the course of their illness.’

Alemtuzumab, marketed as Lemtrada by makers Genzyme, was being used in chronic lymphocytic leukaemia when Prof Compston identified its potential for treating MS.

It works by destroying a key class of immune cells that are attacking the body’ s healthy nerve cells and then rebooting the immune system so it no longer turns on itself. A main side effect is patients can develop other autoimmune diseases as the immune system gradually recovers following exposure to the drug.

These include thyroid problems and a low blood platelet count that can rarely can prove fatal. Platelets are cells that circulate in the blood and clot to keep us from bleeding. The complications can be easily treated if promptly recognised which makes monthly blood tests essential, said Prof Compston.

‘This drug offers a bright future but it is coloured by the risk.

‘It’s not a cure and it doesn’t work for everyone but it can stabilise the disease for a long time. Patients are less disabled, they can look after their families and the societal savings are considerable’ he added.

Unlike most treatments newly approved for NHS use, patients don’t have to try other drugs first and more than two courses of treatment may be offered.

Prof Compston said ‘Twenty years ago when a young person was diagnosed, I’d have to say I’m sorry to tell you that you do have MS – we’ll do our best to look after you but there are limited treatments.

‘Today this drug may well be suitable for 50 to 60 per cent of early cases.’

Nick Rijke, Director for Policy & Research at the MS Society, said it was ‘a major step forward’.

He said ‘This drug has taken decades to develop, and we applaud the team at Cambridge for all their work in making it a reality.

‘While it’s not without risk, it’s proven to be a highly effective medicine for people with relapsing remitting MS and we look forward to seeing it made available to those who could benefit.’

Amy Bowen, of the MS Trust said ‘This provides people with MS with an important treatment option to consider in partnership with their MS specialists.’

Source: Daily Mail © Associated Newspapers Ltd 2014 (28/05/14)

Accelerated lymphocyte recovery after alemtuzumab does not predict multiple sclerosis activity(19/05/14)

ABSTRACT

Objective: To test the hypothesis that accelerated peripheral blood mononuclear cell recovery after alemtuzumab treatment of multiple sclerosis is associated with recurrent disease activity and to investigate the claim that CD4 counts greater than 388.5 × 106 cells/mL at 12 months can be used to identify patients who may benefit from further treatment.

Methods: A total of 108 patients were followed for a median of 99 months post alemtuzumab. Patients were classified as active or nonactive after each cycle of treatment based on clinical relapse, increasing disability, or new T2/enhancing MRI lesions. These outcomes were correlated with CD4, CD8, CD19, CD56+ NK, and monocyte counts.

Results: Of 108 patients, 56 (52%) relapsed at some point during follow-up. Mean annualized relapse rate after alemtuzumab was 0.17 vs 1.67 prior to treatment (equating to a 90% reduction). Of 108 patients, 28 (26%) met the criteria for sustained accumulation of disability. Median time to the lower limit of normal for CD19, CD8, and CD4 was 3, 19.5, and 32 months, respectively. There was no significant difference in the recovery of any cell population between patients with and without disease activity or accumulation of disability after treatment.

Conclusion: This study does not support the use of cell counts as biomarkers for identifying patients at greater risk of active disease following treatment with alemtuzumab.

Onajite Kousin-Ezewu, MRCP, Laura Azzopardi, MRCP, Richard A. Parker, MSc, Orla Tuohy, MRCP, Alastair Compston, FRCP, PhD, Alasdair Coles, FRCP, PhD and Joanne Jones, MRCP, PhD

Source: Neurology 10.1212/WNL.0000000000000520 © 2014 American Academy of Neurology (19/05/14)

Lemtrada slows brain atrophy and reduces new Multiple Sclerosis lesions(01/05/14)

- Consistent effects seen across key MRI measures of disease activity; Effects were sustained beyond two-year pivotal MS studies.

- Approximately 80 percent of patients treated with Lemtrada did not receive a third course of treatment in the first year of the extension.

Genzyme , a Sanofi company , announced today new magnetic resonance imaging (MRI) data from the Lemtrada (alemtuzumab) clinical development program. In Lemtrada patients from the two Phase III clinical trials (both treatment-naïve patients and patients who had active disease on another therapy), MRI effects observed after two years were maintained during the first year of the extension study. These data, which are being presented today at the 66th American Academy of Neurology (AAN) Annual Meeting, include:

Consistent effects were seen across key measures of disease activity (gadolinium (gd)-enhancing, T2 hyperintense and T1 hypointense lesion activity) and effects seen after two years of treatment were sustained at year three.

During the third year of follow-up, more than 70% of patients were free of MRI activity indicative of acute inflammation, defined as gd-enhancing or new or enlarging T2 hyperintense lesions.

T2 lesion volumes, which reflect the combined burden of permanent brain injury and new lesion formation, increased from year two to three but remained below pre-treatment baseline.

The rate of atrophy, as measured by brain parenchymal fraction, already reduced after two years, continued to slow in the third year of follow-up.

Approximately 80 percent of patients treated with Lemtrada did not receive a third course of treatment in the first year of the extension.

“What’s remarkable about these data is that the positive MRI effects of Lemtrada were sustained into the extension study, even though most patients did not receive additional Lemtrada treatment. This observation is unique amongst the current landscape of MS therapeutics,” said Douglas Arnold, M.D., NeuroRx Research and Department of Neurology and Neurosurgery, Montreal Neurological Institute, McGill University. “The new MRI results are an important addition to the clinical data from the extension study that demonstrated Lemtrada’s effect on key measures of clinical disease activity including annualized relapse rates and sustained accumulation of disability.”

The most common side effects of Lemtrada are infusion associated reactions (headache, rash, pyrexia, nausea, fatigue, urticaria, insomnia, pruritus, diarrhea, chills, dizziness, and flushing), infections (upper respiratory tract and urinary tract), and lymphopenia. Autoimmune conditions (including immune thrombocytopenia, other cytopenias, glomerulonephritis and thyroid disease) and serious infections can occur in patients receiving Lemtrada. A comprehensive risk management program incorporating education and monitoring will support early detection and management of these identified risks. Safety results from the first year of the extension study were previously reported for patients who received Lemtrada in the Phase III CARE-MS studies. No new risks were identified. As previously reported, there were two deaths in the extension study. One was from sepsis and the other was presumed accidental and deemed unrelated to study treatment.

The Phase III trials of Lemtrada were randomized, two-year studies comparing treatment with Lemtrada to high-dose subcutaneous interferon beta-1a (Rebif®) in patients with RRMS who had active disease and were either new to treatment (CARE-MS I) or who had relapsed while on prior therapy (CARE-MS II). In these studies, patients on Lemtrada received two courses of treatment, the first administered via intravenous infusion on five consecutive days, and the second administered on three consecutive days, 12 months later.

Lemtrada-treated patients who continued uninterrupted follow-up in the extension study were eligible for re-treatment on evidence of disease activity. This analysis included 349 Lemtrada-treated patients from CARE-MS I and 393 Lemtrada-treated patients from CARE-MS II; 18 percent and 20 percent, respectively, received re-treatment. MRI scans were taken at CARE-MS baseline, and at 12, 24, and 36 months.

“Given the importance of MRI in measuring disease activity in MS, the Lemtrada data announced today are significant,” said Genzyme President and CEO, David Meeker, M.D. “These results reinforce the potential that Lemtrada holds to transform the treatment of MS.”

In CARE-MS I, Lemtrada was significantly more effective than interferon beta-1a at reducing annualised relapse rates; the difference observed in slowing disability progression did not reach statistical significance. In CARE-MS II, Lemtrada was significantly more effective than interferon beta-1a at reducing annualized relapse rates, and accumulation of disability was significantly slowed in patients given Lemtrada vs. interferon beta-1a.

Source: MarketWatch Copyright © 2014 MarketWatch, Inc (01/05/14)

MS drug Lemtrada to be resubmitted to FDA for approval(09/04/14)

Nearly four months after the Food and Drug Administration rejected a multiple sclerosis drug developed by Genzyme, citing the clinical trial’s design and a risk of serious side effects, the Cambridge biotech said Monday that it will resubmit an application for approval to the agency.

Rather than appeal the FDA’s decision, as expected, Genzyme said it will file an updated license application for the drug candidate, called Lemtrada, in the second quarter. The new submission will specifically address issues raised by the FDA in its Dec. 27 decision turning down the experimental treatment, Genzyme said in a statement.

The move follows a series of discussions early this year between Genzyme executives and regulators in the neurology products division of the FDA’s Office of Drug Evaluation, said Genzyme spokeswoman Erin Walsh. She declined to elaborate on the talks and said company officials would not comment until after they resubmit their application. But she did say the drug maker has no plans to conduct additional clinical studies before the FDA rules.

“We had constructive discussions with the FDA that have led us down this path where we’re resubmitting our application rather than filing an appeal,” Walsh said. “We don’t believe further trials are needed prior to an approval. But we would entertain any kind of post-approval commitments” to the FDA, including possible further tests of the drug, she said.

Similarly, FDA officials would not talk about Genzyme’s plans to resubmit its Lemtrada application.

The agency rejected the drug for use in the United States, even though it had been approved by regulators in Europe, Canada, and Australia. The decision was criticised by MS patients, who said the drug had proven effective in clinical trials, and by investors who own a special class of stock that is tied to Lemtrada’s regulatory and sales milestones.

“We are not able to discuss any aspect of a drug application that is under agency review,” said FDA spokeswoman Sandy Walsh. In November, an FDA advisory committee warned that the drug’s side effects could include rashes, bleeding, and even thyroid cancer.

Genzyme scientists spent more than a decade developing Lemtrada for the treatment of multiple sclerosis, a disease that affects the central nervous system. About 1,700 patients were involved in clinical testing of the drug. More than 2.3 million people have been diagnosed with MS worldwide, including about 400,000 in the United States.

The potential of that market, and anticipation that Lemtrada would be approved in the United States, was a major attraction for the French drug maker Sanofi SA, which paid $20.1 billion to acquire Genzyme in 2011. In that deal, Sanofi agreed to reward former Genzyme stockholders if the drug achieved certain milestones in the US market.

Source: The Boston Globe © 2014 BOSTON GLOBE MEDIA PARTNERS, LLC (09/04/14)

UK’s NICE says “yes” to Genzyme’s MS drug Lemtrada(04/04/14)

In final draft guidance, UK drugs watchdog the National Institute for Health and Care Excellence (NICE) has recommended the use of French drug major Sanofi (Euronext: SAN) subsidiary Genzyme’s multiple sclerosis drug Lemtrada (alemtuzumab).

The NICE is appraising alemtuzumab as a treatment for adults with relapsing–remitting multiple sclerosis, a chronic and disabling neurological condition that, as it progresses, can have a substantial negative impact on a person’s quality of life.

Currently available treatments for this type of MS are either oral daily tablets or injections given several times each week and can have unpleasant side effects. The NICE pointed out. Alemtuzumab is taken intravenously once a year for two years. The first course is administered for five consecutive days, and the second course is administered for three consecutive days, 12 months later allowing those with the condition to lead their lives without treatment for the rest of the time. Alemtuzumab does have some possible side effects associated with it which, according to clinical specialists, are manageable. Compared with most available treatments, alemtuzumab has a shorter time period during which it is not recommended that a person gets pregnant. This could be seen as advantageous for patients who are planning to have a baby.

Carole Longson, NICE Health Technology Evaluation Centre director, said: “We are very pleased to be able to recommend alemtuzumab for adults with relapsing-remitting multiple sclerosis. Following a request for more information by our independent Appraisal Committee, the manufacturer was able to provide further evidence, which has led to this positive recommendation.”

Until the NICE issues final guidance, National Health Service bodies should make decisions locally on the funding of specific treatments. Once NICE issues its guidance on a technology it replaces local recommendations across the country.

Pricing to be confirmed

The indicative price of alemtuzumab is £7,045 ($9,722) per 12 mg vial, which equates to £56,360 for the full course of treatment consisting of five daily consecutive 12mg doses in year one, followed by three daily consecutive 12mg doses 12 months later in year twp. The price of alemtuzumab has not yet been confirmed by the Department of Health. However, the Department has exceptionally agreed for this indicative price to be used for the purpose of this appraisal. Costs may vary in different settings because of negotiated procurement discounts.

Last year, when issuing draft guidance (The Pharma Letter December 5, 2013), Prof Longson said: “When reviewing the evidence for alemtuzumab, the Appraisal Committee concluded that there were still questions to be answered. This is why we have requested more details from the manufacturer; to ensure that we have the information the Appraisal Committee needs to reach their conclusions.”

Source: www.thepharmaletter.com (04/04/14)

Brazil health agency approves Genzyme’s Lemtrada to treat multiple sclerosis(24/03/14)

ANVISA, Brazil’s national health surveillance agency, has approved Sanofi company Genzyme's Lemtrada (alemtuzumab) for the treatment of patients with relapsing forms of multiple sclerosis (MS) to slow or reverse the accumulation of physical disability and reduce the frequency of clinical exacerbations.

Lemtrada is supported by a clinical development program that involved about 1,500 patients and 5,400 patient-years of follow-up.

The approval in Brazil follows the recent approvals of Lemtrada in Mexico, Canada, Australia and the European Union (EU).

The company said that lemtrada is currently not approved in the US.

In December 2013, the company secured a complete response letter from the US FDA on its application for US approval of Lemtrada and announced its intent to appeal this decision. Marketing applications for Lemtrada are also under review in other countries.

Brazil's Hospital da Restauração head of Neurology Maria Lucia Brito Ferreira said lemtrada is an new treatment, with clinical trial data that support its potential to meaningfully address relapse rates and disability in patients with active MS.

"Lemtrada will provide physicians with a promising new option for their patients with active MS and could change the way this disease is managed," Ferreira said.

The company said that Lemtrada 12mg has a new dosing and administration schedule of two annual treatment courses.

The first treatment course of Lemtrada is administered through intravenous infusion on five consecutive days, and the second course is given on three consecutive days, 12 months later.

Most common side effects of Lemtrada are infusion associated reactions, infections, lymphopenia and leukopenia.

Source: © PBR 2010. Part of Progressive Digital Media Group Plc (24/03/14)

Lemtrada(R) approved in Mexico for treatment of Multiple Sclerosis(04/02/14)

Genzyme, a Sanofi company, announced today that Mexico's national regulatory authority, COFEPRIS, has approved Lemtrada (alemtuzumab) for the treatment of patients with relapsing remitting forms of multiple sclerosis (MS) to slow or reverse the accumulation of physical disability and reduce the frequency of clinical exacerbations.

"The approval of Lemtrada in Mexico is an important step forward for MS patients, who remain in great need of new treatment options that may offer greater efficacy," said Miguel A. Macias, M.D., Department of Neuroscience, University of Guadalajara. "The positive effect on disability progression demonstrated in clinical studies underscores Lemtrada's ability to address the course of disease in a potentially transformative way for patients with relapsing remitting MS."

Lemtrada is supported by a comprehensive and extensive clinical development program that involved nearly 1,500 patients and 5,400 patient-years of follow-up. Approval in Mexico follows the recent approvals of Lemtrada in Canada, Australia and the European Union. Lemtrada is currently not approved in the United States. In December, Genzyme received a complete response letter from the FDA on its application for U.S. approval of Lemtrada. Genzyme plans to appeal the agency's decision. Marketing applications for Lemtrada are also under review in other countries.

More than 2.5 million people worldwide have been diagnosed with MS, including approximately 15,000 people in Mexico. Lemtrada has been granted orphan drug designation in Mexico.

Lemtrada 12 mg has a novel dosing and administration schedule of two annual treatment courses. The first treatment course of Lemtrada is administered via intravenous infusion on five consecutive days, and the second course is administered on three consecutive days, 12 months later.

"The approvals in the European Union, Australia, Canada and now Mexico underscore Lemtrada's potential to have a positive impact on the lives of MS patients," said Genzyme President and CEO, David Meeker. "Genzyme remains committed to providing new hope for the MS community and plans this year to launch Lemtrada in more than 30 countries, and hopefully additional markets where the treatment is still under review."

The Lemtrada clinical development program included two randomized Phase III studies comparing treatment with Lemtrada to high-dose subcutaneous interferon beta-1a (Rebif(R) ) in patients with RRMS who had active disease and were either new to treatment (CARE-MS I) or who had relapsed while on prior therapy (CARE-MS II), as well as an ongoing extension study. In CARE-MS I, Lemtrada was significantly more effective than interferon beta-1a at reducing annualized relapse rates; the difference observed in slowing disability progression did not reach statistical significance. In CARE-MS II, Lemtrada was significantly more effective than interferon beta-1a at reducing annualized relapse rates, and accumulation of disability was significantly slowed in patients given Lemtrada vs. interferon beta-1a.

The most common side effects of Lemtrada are infusion associated reactions, infections (upper respiratory tract and urinary tract), lymphopenia and leukopenia. Autoimmune conditions and serious infections can occur in patients receiving Lemtrada. A comprehensive risk management program incorporating education and monitoring will support early detection and management of these identified risks.

Source: Wall Street Journal Copyright ©2014 Dow Jones & Company, Inc (04/02/14)

MS drug Lemtrada investigators protest FDA decision(17/01/14)

Several multiple sclerosis (MS) experts who were involved in the clinical development program for alemtuzumab (Lemtrada, Genzyme) have voiced their disappointment and frustration over the recent decision by the US Food and Drug Administration (FDA) not to approve the drug.

Stephen Krieger, MD, from Mount Sinai Medical Center, New York; Edward Fox, MD, from the Multiple Sclerosis Clinic of Central Texas, Round Rock; and Jeffrey Cohen, MD, from Cleveland Clinic, Ohio, all told Medscape Medical News that they strongly disagree with the FDA decision and are worried about the consequences, which could include patients sourcing the drug from other countries but not being monitored for the serious safety issues.

"It's a huge enterprise to do these trials," Dr. Krieger commented. "A lot of doctors are involved, and we have all seen patients do extremely well who probably wouldn't have done so without alemtuzumab. There is a lot of disappointment among MS specialists."

The company announced that FDA had declined approval for alemtuzumab on December 27, saying that Genzyme had not submitted evidence from adequate and well-controlled studies demonstrating that the benefits of alemtuzumab outweigh its serious adverse effects. The concerns are understood to relate to the open-label design of the 2 phase 3 Comparison of Alemtuzumab and Rebif Efficacy in Multiple Sclerosis trials, CARE-MS I and CARE-MS II.

The agency has also stated that 1 or more additional active comparator clinical trials of different design and execution are needed for approval.

"Genzyme strongly disagrees with the FDA's conclusions and plans to appeal the agency's decision," the company said in a statement released December 30 after the FDA issued a Complete Response Letter.

Prior to that, the drug was the subject of an advisory panel meeting of the FDA's Peripheral and Central Nervous System Drugs Advisory Committee, which did not reach a clear consensus. Dr. Krieger told Medscape Medical News that he thought the advisory panel was in favor of approval. "The spirit of the panel was that it was approvable," he said.

But chair of that advisory committee, Nathan Fountain, MD, University of Virginia, Charlottesville, defended the FDA's decision.

"It is likely that the FDA did not approve alemtuzumab for MS because some adverse events that occurred were potentially fatal; in addition, some of them did not occur for years after administration of the drug," Dr. Fountain told Medscape Medical News. "It would be very difficult to insure that patients would be monitored for these problems for an adequate duration."

Many MS researchers looking at the data to date with this drug had been hopeful that the dosing schedule of 2 treatments a year apart, with many patients apparently not requiring any further treatment, might represent an opportunity to move MS into the category of a controlled chronic disease. Others had even used the "C" word — cure — in discussions of its potential impact.

"If alemtuzumab were truly curative, then I imagine the benefits might outweigh the risks," Dr. Fountain said. "However, the clinical trials submitted did not allow determination of whether alemtuzumab altered the natural history of MS. Only through a longer, well-designed, double-blind, randomized, adequately powered study could it be determined whether alemtuzumab is curative.

"Although this type of study is not popular among industry or NIH [National Institutes of Health] sponsors, a curative therapy for MS would be worth the seemingly high risk of a drug like alemtuzumab and would represent a foundational breakthrough in MS therapy," Dr. Fountain concluded.

While the advisory panel did not appear to reach a clear consensus, the assessment of FDA's own reviewers, released before the meeting, was more clear, bordering on blunt. In documents provided to the panel in advance of the meeting, Billy Dunn, MD, acting deputy director of the Division of Neurology Products, summarizes 3 reports from reviewers: from Evelyn Mentari, MD, of the drug's safety; from John Marler, MD, of its clinical efficacy, and from Sharon Yan, PhD, of the clinical statistics supporting the supplemental Biologics License Application for alemtuzumab.

"As discussed by Drs. Mentari, Marler, and Yan, significant concerns exist regarding the safety profile of alemtuzumab and the adequacy of the efficacy data," Dr. Dunn says in the memo.

Playing Politics?

For his part, Dr. Fox believes the issue is more about Genzyme not following FDA requests.

"I knew the FDA had an issue regarding trial design," he said. "There had been a large amount of communication between Genzyme and the FDA which I thought had been resolved. But it appears that the FDA wanted a double-dummy design, Genzyme didn't do it, and now the FDA is putting its foot down. They appear to be making a point. But I don't believe they ever said the drug would not be approved with that trial design. They allowed it to be filed and fast tracked."

Dr. Fox says he "isn't taking no for an answer" at the moment, and he is organizing an open letter from the alemtuzumab investigators to the FDA explaining the reasons for the trial design and the need for the drug. "Individuals from the FDA made this decision, but they cannot dispute the overriding nature of the results. I want this drug available for patients with aggressive MS," he commented.

Dr. Fox was a member of the international steering committee for the development program for alemtuzumab and an investigator in the phase 2 and both phase 3 trials of the drug. He says the steering committee included specialists from many different countries, and there was international consensus on the study design.

Dr. Krieger, who was a site principal investigator for the CARE-MS 2 trial, takes a similar view. "The FDA did request several times that Genzyme conduct the studies with a double-dummy design, but Genzyme set a higher bar of efficacy by using an active comparator and that in itself made a double-dummy design difficult. But why then did the FDA let them go ahead and give them fast track status for the drug approval process?"

The FDA concerns revolve around the fact that the patients were not blinded, Dr. Krieger explains. But he maintains this was accounted for by other factors. "There were blinded evaluators and there were regular assessments to make sure the evaluators remained blinded."

Dr. Cohen echoes these opinions. "The open design of the study was very vigorously discussed on the steering committee of the CARE-MS program, of which I was a member. The hypothetical concerns about bias are well known," he said.

"I have 2 disagreements with the FDA," Dr. Cohen added. "I don't think the double-dummy design would have been feasible as patients would know which drug they were on because of the side effects. And there was enough evidence presented to assuage the concerns about bias."

Dr. Krieger made the point that alemtuzumab is given as an infusion over a few days, whereas interferon β-1a (Rebif, EMD Serono/Pfizer) is administered as an injection 3 times a week, so a double-dummy trial would involve patients being given sham injections 3 times a week for the 2-year trial period or undergoing a sham infusion for several days, which he said was "impractical and unethical."

"Every single person knows what drug they are on, even with a double dummy," Dr. Fox agreed. "With all the effort involved to make a double dummy happen, it would have been a sham."

He further pointed out that most other MS drugs have been tested against placebo, which makes blinding easier. "Because of safety concerns with alemtuzumab, it was felt that the only way to justify its use was to compare with an active drug routinely used for MS. This is a higher standard than applied to any other MS drug, but it is this that has caused them problems because of the lack of blinding. But alemtuzumab is very clearly more effective than Rebif on reducing relapses, slowing disability, and reducing MS lesions on MRI."

Accessing the Drug From Canada or Europe?

Alemtuzumab is already approved in the European Union, Canada, and Australia, and additional marketing applications are under review by other regulatory agencies.

All 3 experts were also uncomfortable about the situation that may now arise, with alemtuzumab being available in Canada and Europe but not in the United States. They point out that this could lead to unsupervised use and big safety concerns.

Dr. Krieger commented: "It is highly likely that some patients will travel to Canada to get it. It is relatively easy to undergo the 2 single administrations 1 year apart in a different country, but the problem is that there won't be any safety programs set up in the US to monitor patients. This drug has some dangerous side effects. Patients need to be monitored closely. This won't happen if they get the drug abroad. This is a very risky situation."

"This was never going to be a medicine for everyone but it is a powerful product for those with the most active form of MS," he added. "And there is a big unmet need here. But these are not the patients I would choose to send to another country for treatment. These are the patients I would want to keep close."

Recent experience with the use of angioplasty to address chronic cerebrospinal venous insufficiency (CCSVI) in MS shows without a doubt that knowledgeable, motivated patients with MS have been known to take matters into their own hands. After the first studies relating to a possible link between CCSVI and MS came out, hundreds of patients used their own resources to fly to centers, sometimes in different countries, to obtain treatment despite the lack of data to support efficacy or safety, and issues with follow-up care once they returned.

One possible scenario now would be that MS centers in the United States may collaborate with hospitals in Canada or Europe and together offer a program under which, if a patient obtains alemtuzumab treatment in a country where it is available, they will undergo monitoring back home at the US hospital.

Dr. Cohen says he has discussed this very possibility. "That is not an ideal situation but it would be feasible if it is the only option," he added.

The National Multiple Sclerosis Society, which describes itself as a "collective of passionate individuals who want to do something about MS now," testified before the FDA advisory committee on alemtuzumab, addressing "the need for more therapeutic options for people with MS and the importance of empowering people with MS to make their own informed treatment decisions," the society notes in a statement reacting to the FDA's decision not to approve alemtuzumab.

"This is disappointing news, given the need for more therapeutic options for people with MS living in the United States," Timothy Coetzee, PhD, chief advocacy, services and research officer at the National Multiple Sclerosis Society, said in the statement.

"The Society will continue to monitor this process and update its constituents of any news," the release concludes.

Dr. Fox reports receiving consultancy fees, honoraria, travel, and research support from Genzyme; Dr. Krieger has served as a consultant and on advisory boards for Genzyme; and Dr. Cohen reports research support paid to his institution from Genzyme.

Source: Medscape Multispeciality Copyright © 1994-2014 by WebMD LLC (17/01/14)

MS drug Lemtrada fails to win FDA approval(30/12/13)

Sanofi failed to win U.S. regulatory approval for its multiple sclerosis drug Lemtrada, denting the company’s ambitions of capturing a larger share of the $20 billion market for the disease.

The U.S. Food and Drug Administration said Sanofi’s Genzyme unit didn’t submit evidence from “adequate and well-controlled studies” showing that the benefits of Lemtrada outweigh its side effects, the Paris-based company said in a statement today. Sanofi disagrees with the conclusion and plans to appeal, the company said.

Lemtrada, which was approved in the European Union in September, and in Australia and Canada this month, was a key part of Sanofi’s $20.1 billion acquisition of Genzyme in 2011. The FDA indicated one or more additional trials of different design and execution comparing Lemtrada to another drug are needed for approval, Sanofi said.

“We are extremely disappointed with the outcome of the review and the implications for patients in the U.S. suffering with multiple sclerosis who remain in need of alternative therapies to manage a devastating disease,” Genzyme President David Meeker said in the statement.

The market for MS drugs will grow to $20.2 billion annually by 2017, according to a 2011 report by GlobalData, a London-based research company. Treatments against the disease totaled $11.6 billion last year, according to data compiled by Bloomberg Industries, in a market dominated by Teva Pharmaceutical Industries Ltd’s Copaxone, Biogen Idec Inc’s Tecfidera, Avonex and Tysabri, Novartis AG's Gilenya and Merck KGaA’s Rebif.

‘Safety Issues’ Sanofi submitted two late-stage clinical trials comparing treatment with Lemtrada to Rebif. An FDA advisory panel last month said Lemtrada was effective for preventing flare-ups, even as it said the trials weren’t conducted well enough to assess it. FDA staff determined in a Nov. 8 report that Lemtrada has “serious and potentially fatal safety issues” including risk of cancer and autoimmune and thyroid diseases.

“We strongly believe that the clinical development program, which was designed to demonstrate how Lemtrada compares against an active comparator as opposed to placebo, provides robust evidence of efficacy and a favorable benefit-risk profile,” Meeker said in the statement. “This evidence was also the basis for the approvals of Lemtrada by other regulatory agencies around the world.”

Neurological Function
The agency was scheduled to decide by Dec. 27 whether to approve Lemtrada for relapsing-remitting MS, the most common form of the disease.

Multiple sclerosis is a debilitating disease in which the immune system attacks the central nervous system, causing symptoms that can include coordination problems, sexual dysfunction and spasticity, according to the National MS Society. Relapses, or flare-ups, are episodes of worsening neurological function.

Lemtrada, also known as alemtuzumab, is a so-called monoclonal antibody given in two annual courses of infusions. The drug targets CD52, a protein on the surface of the immune system cells that are thought to cause the disease. It was approved as Campath in 2001 to treat a form of leukemia, but is no longer for sale. Sanofi also sells Aubagio, which like Tecfidera and Gilenya is an oral medicine for MS.

Development Goals
As part of its acquisition of Genzyme, Sanofi issued so-called contingent value rights, or CVRs, to the U.S. company’s investors. Holders of the rights, which are publicly traded, stood to receive payments of as much as $14 a share by the end of 2020 if Sanofi met certain goals, most of them tied to the approval and sale of Lemtrada. Sanofi said in 2011 it missed the first of the goals.

The rights plunged 62 percent on Nov. 8 after the FDA published a report saying the drug may not offer enough benefit to patients to outweigh risks including cancer. The CVRs closed Dec. 27 at 82 cents in Nasdaq Stock Market trading.

Sanofi said it doesn’t expect that one of the milestones that will trigger a payment, Lemtrada approval in the U.S. by March 31, 2014, will be met.

Source: Bloomberg ©2013 BLOOMBERG L.P (30/12/13)

Sanofi faces U.S. class action over MS drug Lemtrada(23/12/13)

A U.S. law firm is launching a class action against France's Sanofi over what it calls misleading statements on the safety and efficacy of its multiple sclerosis drug Lemtrada.

Sanofi acquired Lemtrada when it bought U.S. biotech firm Genzyme for $20.1 billion in 2011. The drug's prospects took center-stage in a drawn-out takeover battle and led to a deal in which Genzyme shareholders received listed contingent value rights (CVRs) linked to Lemtrada's future success.

Law firm Lieff Cabraser Heimann & Bernstein, LLP said on Friday it was bringing litigation on behalf of all purchasers of the CVRs between March 6, 2012 and November 7, 2013.

The firm alleged that over this period, Sanofi and some of its senior executives made false and misleading statements about its business and the prospects for Lemtrada, and misled investors over the design of its clinical trials on the drug.

A spokesman for Sanofi said the company does not comment on pending litigation.

The complaint comes after U.S. Food and Drug Administration (FDA) experts voiced concerns last month over Lemtrada's safety and the quality of its clinical studies, prompting CVRs on the drug to shed over 60 percent of their value in one day.

Lemtrada, an injectable treatment also known as alemtuzumab, is one of Sanofi's two new drugs for MS, an autoimmune disease that attacks the central nervous system and affects more than 2 million people worldwide.

The drug was approved by European regulators in September and the FDA is expected to rule by December 27 on whether to approve the treatment for marketing in the United States, the world's biggest pharmaceutical market.

Source: Reuters © Thomson Reuters 2013 (23/12/13)

MS drug Lemtrada approved in Australia (20/12/13)

Genzyme, a Sanofi company, has announced that the Australian Therapeutic Goods Administration (TGA) has approved Lemtrada (alemtuzumab) for the treatment of relapsing forms of multiple sclerosis for patients with active disease defined by clinical or imaging features to slow the accumulation of physical disability and reduce the frequency of clinical relapses.

"Multiple Sclerosis is a highly complex and often devastating disease that can lead to significant disability in patients, despite availability of standard therapies," said Associate Professor John King, Senior Neurologist, Royal Melbourne Hospital. "Lemtrada represents a significant advance in the way physicians and patients can think about treating multiple sclerosis. The efficacy data supporting Lemtrada highlight its strong potential to impact disease progression in patients with relapsing forms of MS."

Lemtrada is supported by a comprehensive and extensive clinical development program that involved nearly 1,500 patients and 5,400 patient-years of follow-up. Approval in Australia follows the recent approval of Lemtrada in Canada and the European Union. Marketing applications for Lemtrada are also under review in other countries.

More than 2.3 million people worldwide have been diagnosed with MS, including approximately 20,000 people in Australia.

Lemtrada 12 mg has a novel dosing and administration schedule of two annual treatment courses. The first treatment course of Lemtrada is administered via intravenous infusion on five consecutive days, and the second course is administered on three consecutive days, 12 months later.

"The approval of Lemtrada in Australia reinforces the significance of this treatment and is an important milestone in Genzyme's commitment to bring this potentially transformative therapy to patients globally," said David Meeker, President and CEO, Genzyme. "We're very pleased with the TGA's approval and look forward to working with the reimbursement authorities to make Lemtrada available to patients in Australia."

The Lemtrada clinical development program included two pivotal randomized Phase III studies comparing treatment with Lemtrada to high-dose subcutaneous interferon beta-1a (Rebif(R) ) in patients with RRMS who had active disease and were either new to treatment (CARE-MS I) or who had relapsed while on prior therapy (CARE-MS II), as well as an ongoing extension study. In CARE-MS I, Lemtrada was significantly more effective than interferon beta-1a at reducing annualized relapse rates; the difference observed in slowing disability progression did not reach statistical significance. In CARE-MS II, Lemtrada was significantly more effective than interferon beta-1a at reducing annualized relapse rates, and accumulation of disability was significantly slowed in patients given Lemtrada vs. interferon beta-1a.

The most common side effects of Lemtrada are infusion associated reactions, infections (upper respiratory tract and urinary tract), lymphopenia and leukopenia. Autoimmune conditions and serious infections can occur in patients receiving Lemtrada. A comprehensive risk management program incorporating education and monitoring will support early detection and management of these identified risks.

Source: The Wall Street Journal Copyright ©2013 Dow Jones & Company, Inc (20/12/13)

Lemtrada approved in Canada for treatment of Multiple Sclerosis(13/12/13

Genzyme, a Sanofi company, announced today that Health Canada has approved Lemtrada (alemtuzumab) for the management of adult patients with relapsing remitting multiple sclerosis (RRMS), with active disease defined by clinical and imaging features, who have had an inadequate response to interferon beta or other disease-modifying therapies.

"Lemtrada is an important new treatment option for Canadians with MS. It has impressive effectiveness following two treatment courses for those patients with active relapsing MS," said Dr. Anthony Traboulsee, Associate Professor of Neurology and Medical Director of the UBC Hospital MS Clinic of Vancouver Coastal Health. "Our own local experience in treating 35 patients through clinical trials with Lemtrada has been extremely positive."

Lemtrada is supported by a comprehensive and extensive clinical development program that involved nearly 1,500 patients and 5,400 patient-years of follow-up. Approval in Canada follows the recent approval of Lemtrada in the European Union. Marketing applications for Lemtrada are also under review in numerous other countries, including the US and Mexico.

More than 2.3 million people worldwide have been diagnosed with MS, including approximately 100,000 people in Canada.

Lemtrada 12 mg has a novel dosing and administration schedule of two annual treatment courses. The first treatment course of Lemtrada is administered via intravenous infusion on five consecutive days, and the second course is administered on three consecutive days, 12 months later.

"The approvals of Lemtrada in Canada and the EU are further recognition of its potential as a transformative treatment option for patients living with active MS," said Genzyme President and CEO, David Meeker. "Lemtrada is now approved in 30 countries, and we are very pleased that Canadian patients and physicians will have access to this important new treatment option."

The Canadian approval was based on data from the Lemtrada clinical development program comparing treatment with Lemtrada to high-dose subcutaneous interferon beta-1a (Rebif(R)) in patients with RRMS who had active disease.

The most common side effects of Lemtrada are infusion associated reactions (headache, rash, pyrexia, nausea, urticaria, pruritus, insomnia, chills and flushing) and infections (nasopharyngitis, urinary tract and upper respiratory tract). Autoimmune conditions, including thyroid disease, cytopenias, glomerulonephritis, and serious infections can occur in patients receiving Lemtrada. A comprehensive risk management program incorporating education and monitoring will support early detection and management of these identified risks.

About Lemtrada(TM) (alemtuzumab)

Alemtuzumab is a monoclonal antibody that selectively targets CD52, a protein abundant on T and B cells. Treatment with alemtuzumab results in the depletion of circulating T and B cells thought to be responsible for the damaging inflammatory process in MS. Alemtuzumab has minimal impact on other immune cells. The acute anti-inflammatory effect of alemtuzumab is immediately followed by the onset of a distinctive pattern of T and B cell repopulation that continues over time, rebalancing the immune system in a way that potentially reduces MS disease activity.

Genzyme holds the worldwide rights to alemtuzumab and has primary responsibility for its development and commercialization in multiple sclerosis. Bayer HealthCare holds the right to co-promote alemtuzumab in MS in the United States. Upon commercialization, Bayer will receive contingent payments based on global sales revenue.

Source: The Wall Street Journal Copyright ©2013 Dow Jones & Company, Inc. (13/12/13)

NICE asks Sanofi for more information on MS oral drug Lemtrada(05/12/13)

The U.K.’s health-cost regulator asked Sanofi for more information on its multiple-sclerosis drug Lemtrada, less than a month after U.S. regulatory advisers said the drug’s trials weren’t adequate to assess its efficacy.

The National Institute for Health and Care Excellence is seeking clarifications on the evidence Paris-based Sanofi’s Genzyme unit submitted on the drug, NICE said in draft guidance today. Sanofi has until Jan. 9 to submit the extra information, the institute said. NICE, which advises the U.K.’s National Health Service on which treatments provide value for money, didn’t specify what information it requested.

Lemtrada, approved in the European Union in September, was a key part of Sanofi’s $20 billion acquisition of Genzyme in 2011. The U.S. Food and Drug Administration is expected to decide by the end of this year whether to approve the drug, which will compete in an increasingly crowded market against treatments such as Novartis AG’s Gilenya and Teva Pharmaceutical Industries Ltd’s Copaxone.

Lemtrada may generate sales of 455 million euros ($619 million) in 2017, according to the average of eight analysts’ estimates compiled by Bloomberg. Bayer AG (BAYN) plans to co-promote the drug in the U.S. and will receive payments based on sales. Sanofi said in October it would charge 8,645 euros per vial for the drug in Germany.

Drug Infusions
Sanofi fell 0.3 percent to 74.71 euros as of 11:39 a.m. in Paris. The stock has gained 8 percent this year, including reinvested dividends, compared with a 25 percent advance in the Bloomberg Europe Pharmaceuticals Index.

The drug is a so-called monoclonal antibody administered to patients through two sets of infusions a year apart. Late-stage clinical trials showed the treatment slowed the progression of disability, though it also led to infections and an autoimmune thyroid-related side effect in some patients.

Sanofi’s first MS therapy, a tablet called Aubagio, won approval in the U.S. last year and in the EU this year. The drugmaker is counting on new products to offset revenue losses from generic competition to best-sellers including the blood-thinner Plavix.

Multiple sclerosis is a debilitating disease that attacks the central nervous system. Relapses, or flare-ups, are episodes of worsening neurological function. More than 100,000 people in the U.K. have the disease, according to the Multiple Sclerosis Trust.

An FDA advisory committee voted last month that Lemtrada is effective for preventing flare-ups of the disease, even as it decided the company’s trials weren’t adequate to assess the drug.

Source: Bloomberg ©2013 BLOOMBERG L.P.(05/12/13)

MS drug Lemtrada does not benefit patients, FDA panel says(14/11/13)

Lemtrada hasn’t proven to help against disability in the treatment of relapsing multiple sclerosis, said U.S. advisers who questioned whether the company’s studies were conducted well enough to assess the drug.

While the U.S. Food and Drug Administration advisory panel decided that potential safety risks don’t preclude approval of Lemtrada, its members voted 14-2 that the drug didn’t help improve a patient’s disability. The agency isn’t required to accept the recommendations of its advisers.

Lemtrada, approved in the Europe Union in September, was a key part of Paris-based Sanofi’s $20 billion acquisition of Genzyme Corp. in 2011. If cleared in the U.S., the drug would enter a crowded field in which relapsing MS patients now have 10 treatment options with varying degrees of efficacy, including Biogen Idec Inc.’s Tecfidera and Teva Pharmaceutical Industries Ltd.’s Copaxone.

“If the study is biased, then everything that flows from the study can’t be trusted,” Robert Clancy, a panel member and professor of neurology and pediatrics at the University of Pennsylvania School of Medicine, said at yesterday’s meeting. The panel voted 11-6, with one abstention, that the two large studies Sanofi submitted on the drug’s behalf weren’t adequate and well-controlled.

Contingent Value
The FDA is expected to decide whether to approve Lemtrada by Dec. 27. The agency will probably issue a so-called complete response letter that rejects the drug until the company does further work, said Jeffrey Holford, an analyst at Jefferies in New York.

“With the panel voting that the studies were not adequate and well-controlled, the FDA probably has to issue a CRL,” Holford wrote in a note today. “We continue to see the probability of approval at 20 percent to 30 percent.”

Lemtrada may generate sales of $672 million in 2017, according to the average of eight analysts’ estimates compiled by Bloomberg. Bayer AG plans to co-promote the drug and will receive payments based on sales.

Debilitating Disease
Multiple sclerosis is a debilitating disease that attacks the central nervous system. Relapses, or flare ups, are episodes of worsening neurological function, according to the National MS Society.

Several patients who said they had severe MS testified today in favour of Lemtrada’s approval, imploring panel members to leave the weighing of risk versus benefit to those who have the disease and their physicians.

“It’s critical that people with significant disease who stand to lose function and become disabled have the opportunity to access this drug,” said David Goldblatt, a neuroradiologist from Austin, Texas, who said he started taking Lemtrada 10 years ago.

Patients’ Responsibility
“I understand your issues with safety,” Goldblatt told the panel. “I think that nowadays patients are taking more responsibility and should be allowed to make the decision that they may decide that the risk/benefit ratio is adequate for them, that they would like to take this medication.”

The panel agreed, with many members saying they voted that the drug’s safety issues didn’t preclude its approval because patients should be allowed to make that decision with their doctor. While panelists said the drug appeared to be effective against the disease, they also voted unanimously that Lemtrada shouldn’t be a first option for patients, if approved.

“For the foreseeable future, I would rank it as a third-line drug,” said Justin Zivin, a panel member and professor emeritus at the University of California at San Diego.

Staff Report
FDA staff determined in a Nov. 8 report that Sanofi’s annual infusion has “serious and potentially fatal safety issues” including risk of cancer and autoimmune and thyroid diseases. FDA staff also questioned Sanofi’s claims the drug is effective.

The company’s decision not to keep secret which patients in clinical research were taking the medicine or an older treatment and the subjective nature of determining whether the drug was working may have skewed results, agency staff said. That was a main focus of yesterday’s FDA advisory panel discussions.

Sanofi said it was unable to keep that information confidential because of the differences in how the drugs, Lemtrada and an older treatment, were given, including annual dosing compared with three times a week. Follow-up data showed findings consistent with clinical trial results, Sanofi said in an e-mailed statement on Nov. 8.

“We are pleased that the advisory committee clearly recognized the effectiveness of Lemtrada and voted unanimously that the safety profile should not stand in the way of approval,” Jack Cox, a spokesman, said in an e-mailed statement today. “The committee vote did acknowledge FDA’s concerns around study design but this appears not to have had an impact on the committee’s votes of the effectiveness and safety profile of Lemtrada. We will work with the agency to support their completion of the review process.”

Lemtrada won European Union approval in September and the active ingredient alemtuzumab was cleared by the FDA in 2001 to treat a certain form of leukemia, though the drug is no longer for sale.

Other treatments for relapsing MS include another Sanofi drug Aubagio, Biogen’s Tecfidera and Tysabri and Teva’s Copaxone. Lemtrada is given through two courses of infusions given a year apart.

Source: Bloomberg ©2013 BLOOMBERG L.P. (14/11/13)

FDA staff says new oral MS drug Lemtrada may be too risky(08/11/13)

Sanofi’s multiple sclerosis treatment Lemtrada may not offer enough benefit to patients to outweigh risks including cancer, U.S. regulators said. The French drugmaker’s shares fell.

Lemtrada’s “serious and potentially fatal safety issues,” which include the risk of autoimmune and thyroid diseases, may make the medicine too dangerous to approve unless there is substantial clinical benefit shown, Food and Drug Administration staff said in a report today. Agency reviewers also questioned whether Sanofi conducted adequate trials to prove the annual infusion works.

“That’s like a death sentence,” said Fabian Wenner, an analyst with Kepler Cheuvreux in Zurich, “It isn’t what everyone expected, an issue with the safety. It seems to be a more fundamental issue here.”

Lemtrada, approved in Europe earlier this year, was at the center of Paris-based Sanofi’s $20 billion acquisition of Genzyme Corp. in 2011. Shareholders who have contingent value rights stand to receive cash payments if Lemtrada is approved. Bayer AG (BAYN) plans to co-promote Lemtrada and will receive payments based on sales.

“The certainty of the risks of potentially lifelong hypothyroidism, serious infusion reactions, melanoma and other malignancies, Graves’s ophthalmopathy and other autoimmune disorders, and prolonged increased susceptibility to infection may not be balanced by the uncertainty that exists in the limited evidence of the potential clinical benefits from clinical trials that were not well-controlled,” FDA drug reviewer John Marler wrote in the report.

Nov. 13 Panel

An advisory panel on Nov. 13 will discuss whether to recommend the drug’s approval. The FDA is expected to decide whether to clear the medicine for treating relapsing MS patients by the end of the year.

Sanofi did submit trial evidence that showed Lemtrada had a significant benefit, though the company’s decision to not keep secret which patients were taking the medicine and the subjective nature of determining whether the therapy was working may have skewed results, Marler said.

Sanofi said it was unable to keep that information confidential because of the differences in how the drugs, Lemtrada and an older treatment, were given, including annual dosing compared with three times a week. Follow-up data showed findings consistent with clinical trial results, Sanofi said in an e-mailed statement.

Sanofi Confident

“Our company is confident that Lemtrada offers an important step forward in the way physicians and patients will think about treating multiple sclerosis,” Sanofi said.

Sanofi fell less than 1 percent to 77.94 euros at 4:21 p.m. Paris time. The contingent value rights linked to the Genzyme acquisition dropped 68 percent to 65 cents in New York trading.

Genzyme stockholders had received one right entitling the owner to additional fees of as much as $14 per CVR by the end of 2020 if Sanofi met certain goals, most of them tied to the approval and sale of Lemtrada.

Lemtrada won European Union approval in September and the active ingredient alemtuzumab was cleared by the FDA in 2001 to treat a certain form of leukemia, though it is no longer for sale.

The drug may generate sales of $691 million in 2017, according to the average of eight analysts’ estimates compiled by Bloomberg.

Source: Bloomberg ©2013 BLOOMBERG L.P. (08/11/13)