Top 10 for ’10: No. 6
No. 6: The First, Oral Disease-Modifying Drug for Multiple Sclerosis Is Approved
In September, the FDA approved fingolimod, trade name Gilenya, the first orally administered disease-modifying drug for the treatment of relapsing-remitting multiple sclerosis. The pill, at a dosage of 0.5 mg once daily, was approved approximately 9 months after drugmaker Novartis quietly submitted its NDA to the agency and nearly a year after Merck Serono trumpeted its first application for an oral competitor, cladribine.
Gilenya’s approval came with a number of safety caveats—including the institution of an FDA-mandated REMS (Risk Evaluation and Mitigation Strategy) program and a pregnancy registry. Uptake of the drug is also potentially hampered by its staggering cost—about $4000 per month, wholesale.* A company-sponsored program has been instituted to (slightly) ease the financial burden for patients. Despite these potential barriers to prescriptions, Novartis expects worldwide sales of Gilenya to reach $1.5 billion by 2017.
The final stretch of the approval horse race for the first, disease-modifying pill for MS was significant for a bad stumble taken by Merck Serono, after the FDA refused to file its application in December of last year. Speculation: the submission was incomplete. Merck Serono filed a new application last June, and the company received a priority review.
FDA approval for cladribine was anticipated at the end of this year; however, in November, the company announced that the agency was extending its original 6-month priority review by another 3 months. Why the FDA requested the extra review time is unclear, but a company spokesperson told Bloomberg in November that the FDA was not requesting additional clinical trials.
The prospect for cladribine’s US approval remains murky, given a thumbs-down opinion from the European Medicines Agency in September. However, the pill is currently available for the treatment of MS in Russia and Australia.
NDA = new drug application.
* The web site destinationrx.com offers 30, 0.5-mg capsules for $4268.16.