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FDA Grants Breakthrough Therapy for PPMS

February 18, 2016

Genentech announced that the FDA has granted its investigational medicine Ocrelizumab as a Breakthrough Therapy Designation judging from its positive Phase 3 results.  Ocrelizumab is a potential treatment of primary progressive multiple sclerosis (PPMS).

This designation is designed to speed up the process of reviewing the medicines treating serious/life-threatening diseases. Ocrelizumab is the first medicine to show positive pivotal trial results in both PPMS and relapsing MS, a more commonly seen form of the disease.

This therapy is a much needed one, as there are currently no approved treatments for PPMS, which plagues about 10% of the MS population.

“Ocrelizumab is the first investigational medicine for MS to be granted Breakthrough Therapy Designation by the FDA,” Sandra Horning, MD, chief medical officer and head of Global Product Development at Genentech, said in a press release. “With no approved treatments for primary progressive MS, ocrelizumab has the potential to address an important unmet need. We are committed to working with the FDA to bring ocrelizumab to people with primary progressive MS as quickly as possible.”

The pivotal, multicenter and double-blinded Phase 3 clinical trial, called ORATORIO, is investigating the efficacy and safety of ocrelizumab compared with placebo in 732 people with PPMS. Participants randomly received ocrelizumab intravenously in two infusions of 300 mg every 14 days in each treatment cycle, or received placebo, with the primary endpoint being time to onset of confirmed disability progression (CDP) sustained for at least 12 weeks. This trial is scheduled to be completed in November 2017.

Genentech said in its announcement that it plans to pursue marketing authorization for ocrelizumab as both a PPMS and relapsing MS treatment, and will submit data from its three Phase 3 studies to the FDA by June 2016.