The U.S. Food and Drug Administration has rejected Genzyme’s new multiple sclerosis drug Lemtrada (alemtuzumab) because the company failed to shows the benefits of the drug outweigh its “serious adverse effects.”
“The FDA has taken the position Genzyme has not submitted evidence from adequate and well controlled studies that demonstrate the benefits of Lemtrada,” the biotech firm said in a statement. Genzyme was acquired in 2011 by the French drug maker Sanofi (NYSE: SNY) for $20 billion.
The FDA said it wanted the company to conduct new clinical studies of Lemtrada on relapsing remitting MS patients, using different study designs and methods.
“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,” said Genzyme President and CEO, David Meeker, MD.
“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. This evidence was also the basis for the approvals of Lemtrada by other regulatory agencies around the world.”
Lemtrada is approved for use in treating MS in the European Union, Canada, and Australia.
Lemtrada is given via intravenous drip. The most common side effects of Lemtrada are IV related reactions, upper respiratory and urinary tract infections, lymphopenia and leukopenia. Serious autoimmune conditions can also occur in patients receiving Lemtrada.
MS is a chronic disease which attacks the body’s central nervous system and is characterized by the destruction of myelin, the membrane that protects nerves in the brain and spinal cord. Once damaged, it inhibits the nerves’ ability to transmit electrical impulses, causing cognitive impairment and poor mobility. About 400,000 Americans have the disease and more than 2 million worldwide.
For most people with MS, relapses are initially followed by recovery periods or remissions. Symptoms may be mild or severe, ranging from numbness in the limbs to paralysis or loss of vision. The progression, severity and symptoms of MS are unpredictable and vary from one person to another. There is no known cure.
Lemtrada is a monoclonal antibody that selectively targets CD52, a protein abundant on T and B cells. Treatment with Lemtrada results in the depletion of circulating T and B cells, which are thought to be responsible for the damaging inflammatory process in MS. The anti-inflammatory effect of Lemtrada is followed by the onset of T and B cell repopulation, which rebalances the immune system in a way that potentially reduces MS disease activity.
The increasingly crowded multiple sclerosis market is moving away from injectable treatments in favor of newer pills such as Novartis’ (NYSE: NVS) Gilenya and Biogen Idec’s (NASDAQ: BIIB) Tecfidera. Worldwide sales of MS drugs generate about $12 billion annually.
Analysts say the FDA’s request for additional studies of Lemtrada could delay its introduction in the U.S. for years.
“Even if approved we expected the drug’s profile would likely relegate it to a less competitive, late-line niche within MS therapy,” said Wells Fargo analyst Brian Abrahams in a note to clients.
“We view the CR (FDA’s) letter — and potential lengthy delay in the drug reaching the market – as incrementally positive for BIIB, as it removes an additional entrant from the U.S. market and lessens the comprehensiveness of competitior SNY’s (Sanofi) MS offerings in the U.S. We continue to believe BIIB’s valuation fairly reflects Tecfidera’s considerable long-term MS potential.”