Sanofi (SAN)’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 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. (BIIB)’s Tecfidera and Teva Pharmaceutical Industries Ltd. (TEVA)’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.
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.”
The stock rose 1.2 percent to 78.73 euros at 9:28 a.m. in Paris. Before today, Sanofi had climbed 13 percent this year, including reinvested dividends, compared with a 25 percent advance in the Bloomberg Europe Pharmaceuticals Index.
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. The CVRs were halted from trading in New York yesterday.
Lemtrada may generate sales of $672 million in 2017, according to the average of eight analysts’ estimates compiled by Bloomberg. Bayer AG (BAYN) 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 today 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. 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.
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.
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