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Sanofi MS Drug’s Mixed Verdict at FDA Panel Puzzles Analysts

Sanofi’s multiple sclerosis drug Lemtrada is effective for preventing flare-ups of the disease, U.S. regulatory advisers said yesterday, even as they decided the company’s trials weren’t adequate to assess the drug.

A U.S. Food and Drug Administration advisory committee voted 12-6 that Sanofi provided substantial evidence of the drug’s effectiveness for treating patients with relapsing forms of MS, and 11-6 that the company’s trials weren’t conducted well enough to assess the drug.

“That seems contradictory,” Mark Clark, an analyst with Deutsche Bank AG in London, said by phone today. “Either they were adequate and proved something, or they were inadequate and proved nothing. We’re somewhat confused.”

Lemtrada, approved in the European 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.

The panel’s other verdicts added to the mixed picture. The committee voted 14-2, with two abstentions, that Lemtrada didn’t reduce a patient’s disability, and 17-0 that the drug’s risks don’t preclude approval.

Complete Response

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.”

Sanofi shares rose 1.4 percent to 78.85 euros 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 fell 2.2 percent to 68 cents in New York. The securities were halted from trading yesterday, after plunging 62 percent on Nov. 8, when the FDA published briefing documents for yesterday’s meeting that questioned whether the two key trials of the drug were valid.

Sales Forecast

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.

“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 Sanofi 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.”

Drug Access

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 yesterday 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.”

Drug’s Safety

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.

Annual Dosing

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 (SAN) said in an e-mailed statement on Nov. 8.

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.

To contact the reporters on this story: Simeon Bennett in Geneva at sbennett9@bloomberg.net; Meg Tirrell in New York at mtirrell@bloomberg.net

To contact the editor responsible for this story: Phil Serafino at pserafino@bloomberg.net

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