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Posted by on Dec 26, 2010 in FDA, Neurology, Pharma

Top 10 for ’10: No. 6

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 caveatsincluding 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 costabout $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 offers 30, 0.5-mg capsules for $4268.16.

bmartin (1130 Posts)

A native East Tennessean, Barbara Martin is a formerly practicing, board-certified neurologist who received her BS (psychology, summa cum laude) and MD from Duke University before completing her postgraduate training (internship, residency, fellowship) at the Hospital of the University of Pennsylvania in Philadelphia. She has worked in academia, private practice, medical publishing, drug market research, and continuing medical education (CME). For the last 3 years, she has worked in a freelance capacity as a medical writer, analyst, and consultant. Follow Dr. Barbara Martin on and Twitter.