FDA Grants Priority Review to Experimental Multiple Sclerosis Drug

The FDA granted priority review to ocrelizumab (Ocrevus) for the treatment of relapsing multiple sclerosis (RMS) and primary progressive multiple sclerosis (PPMS) with a targeted action date of December 28, 2016.

Ocrevus is an investigational, humanized monoclonal antibody that selectively targets CD20-positive B cells. The drug binds to CD20 cell surface proteins expressed on certain B cells, but not plasma or stem cells, which may help preserve important functions of the immune system.

The grant was based on positive results from three phase 3 studies: OPERA I, OPERA II, and ORATORIO. These studies met their primary and key secondary endpoints.

OPERA I and II were identical, randomized, double-blind, double-dummy, global multi-center, phase 3 studies that evaluated the safety and efficacy of Ocrevus in patients with relapsing forms of MS. There were 1656 patients enrolled in the study, who received either 600-mg Ocrevus intravenously every 6 months or 44-mcg interferon beta-1a (Rebif) administered by subcutaneous injection 3 times per week.

In the 2 studies, Ocrevus demonstrated superior efficacy in reducing annualized relapse rates and disability progression sustained for at least 3 months and for at least 6 months compared with Rebif.

ORATORIO is a randomized, double-blind, global multi-center, phase 3 study that evaluated the safety and efficacy of 600-mg Ocrevus administered by intravenous infusion every 6 months compared with placebo in 732 PPMS patients.

The results of the ORATORIO study showed that PPMS patients had significant reductions in disability progression sustained for at least 3 months, and for at least 6 months, as well as in other measures of progressive disease compared with placebo.

“Ocrevus is the first investigational medicine to significantly reduce disability progression in people with relapsing and primary progressive forms of MS,” said Sandra Horning, MD, chief medical officer and head of Global Product Development at Genentech. “We are pleased by the FDA’s decision to classify their review of the BLA as priority because we believe Ocrevus has the potential to help people living with either of these 2 forms of MS. We will continue to work closely with the FDA and EMA to bring this investigational medicine to people with MS as quickly as possible.”

The most common adverse events for Ocrevus were infusion-related reactions and infections, with a majority being mild to moderate in severity.