FDA Greenlights First Drug for Primary Progressive MS

By Jonathan Springston, Editor, AHC Media

The FDA on Wednesday approved ocrelizumab, a drug for patients suffering from multiple sclerosis (MS) and primary progressive MS (PPMS). This is the first drug for PPMS the agency has ever approved.

Researchers demonstrated the efficacy of ocrelizumab in a pair of clinical trials that featured more than 1,600 patients treated for 96 weeks. Patients were randomized to ocrelizumab and another MS drug. In both studies, those who took ocrelizumab demonstrated fewer relapse episodes and experienced reduced disability progression compared to the other drug. Because of these promising results, the FDA granted breakthrough designation, fast-track designation, and priority review.

“Multiple sclerosis can have a profound impact on a person’s life,” Billy Dunn, MD, director of the division of neurology products in the FDA’s Center for Drug Evaluation and Research, said in a statement. “This therapy not only provides another treatment option for those with relapsing MS, but for the first time provides an approved therapy for those with primary progressive MS.”

More than 1 million people worldwide suffer from MS, with about 85% of those suffering from a relapsing-remitting course. Approximately 15% of MS patients suffer from PPMS, whereby the condition grows worse gradually and is not associated with relapses.

The National Multiple Sclerosis Society hailed Wednesday’s news as “a significant milestone,” calling the drug “an encouraging new option.”

“We are grateful that there is finally a disease-modifying treatment for people with primary progressive MS, and also an important new option for people with relapsing MS,” Bruce Bebo, PhD, executive vice president, research, at the National MS Society, said in a statement. “We hope this is just the first of many new treatments approved for people with progressive MS.”

In the March issue of Neurology Alert, Jai S. Perumal wrote extensively about the two trials that tested the efficacy of ocrelizumab.

“The results of ocrelizumab in [relapsing-remitting MS] demonstrates high efficacy, making it one of the treatment options for patients with highly active disease. It also offers convenience of administration and less difficulty with compliance, as it will be administered in infrequent infusions every six months,” Dr. Perumal concluded.

Look for more information about ocrelizumab in upcoming issues of Pharmacology Watch and Internal Medicine Alert. For even more summaries of the latest clinical neurology, be sure to check out past issues of Neurology Alert.