Sanofi’s 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 Wednesday that the drug didn’t help improve a patient’s disability. The agency isn’t required to accept the recommendations of its advisory panels.
Lemtrada, approved in Europe earlier this year, was a key part of Paris-based Sanofi’s $20 billion acquisition of Genzyme Corp. in 2011. The drug if cleared in the U.S. 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 Wednesday’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.
The FDA is expected to decide whether to approve Lemtrada by Dec. 27. Genzyme stockholders had received rights to additional fees of as much as $14 a share, in the form of contingent value rights, or CVRs, at the end of 2020 if Sanofi meets certain goals, most of them tied to the approval and sale of Lemtrada.
Sanofi declined less than 1 percent to 77.78 euros earlier Wednesday at the close in Paris. The tradeable CVR was halted during the vote.
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.
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 Wednesday in favor 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.
“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. They also voted unanimously, though, that the drug should not 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.
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 the discussions Wednesday.
Sanofi said it couldn’t keep the treatments confidential because of the differences in the drugs, including annual dosing compared with three times a week.
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.
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