FDA Accepts Review of Application for Ocrelizumab
On June 27, 2016, Genentech, a member of the Roche group, announced that the Biologics License Application (BLA) for ocrelizumab has been accepted for review by the United States Food and Drug Administration (FDA). This is the first time that an investigational medication is being reviewed for the treatment of two types of multiple sclerosis (MS): relapsing forms of MS (RMS) and primary-progressive MS (PPMS).
The FDA has also granted Priority Review Designation of the application, with a targeted action date of December 28, 2016. This means that the review of the application is expected to be completed by this date, as well as a decision on whether or not this investigational medication is approved. Genentech has submitted the brand name Ocrevus™ for use with ocrelizumab.
If approved, ocrelizumab would be the first medication available for both RMS and PPMS. Presently, 14 disease-modifying therapies are available for the treatment of relapsing forms of MS, but no treatments have been approved by the FDA for PPMS, which is a less-common form of the disease. PPMS is characterized by a steady accumulation of symptoms, versus sudden flare-ups and remissions.
In February 2016, the FDA granted “Breakthrough Therapy Designation” for ocrelizumab (for the treatment of PPMS only), which expedites the review process. Please note that the Breakthrough Therapy Designation does not apply to this investigational medication when used as a treatment for relapsing forms of MS. For more information, please see MSAA’s online news article, “FDA Expedites Review of Ocrelizumab for the Treatment of PPMS.”
Ocrelizumab is an investigational, humanized monoclonal antibody designed to selectively target CD20-positive B cells. These are a specific type of immune cell that is an important contributor to the MS-disease process. In Phase III trials, 600 mgs of ocrelizumab were given via intravenous (IV) infusion every six months. For the first dose in the OPERA studies for people with RMS, and throughout the ORATORIO study (not including the extension study) for people with PPMS, the 600-mg dose was divided into two 300-mg doses, given via IV infusion, and separated by two weeks.
Positive results were seen in three Phase III trials with ocrelizumab. OPERA I and OPERA II were studies with relapsing forms of MS. Compared to treatment with Rebif® (interferon beta-1a), ocrelizumab showed greater efficacy in reducing annualized relapse rates and reducing disability progression that was sustained for time periods of at least three and at least six months. The ORATORIO study was with PPMS and compared treatment with placebo. The trial showed significant reductions in disability progression sustained for time periods of at least three and at least six months. Reductions in other measures of progressive disease were also seen.
Adverse events in all three of the Phase III studies were similar between those given ocrelizumab and those given either interferon beta-1a in the RMS studies or the placebo in the PPMS study. Genentech reports that the most common adverse events seen with this medication were mild to moderate infusion-related reactions and infections.
In other news regarding ocrelizumab, this experimental medication will soon be available to eligible individuals with PPMS through an Expanded Access Program (EAP). An EAP enables participants to receive an investigational medication that has not yet been approved by the Unites States Food and Drug Administration (FDA). This program is a nontraditional study that has no placebo group, so all those enrolled are given the active medication. It follows a strict protocol that has been developed through consultation with the FDA. Please note that this program is limited to only eligible people with PPMS and not for anyone with a relapsing form of MS. For more information, please see MSAA’s article, “Eligible People with PPMS May Receive Experimental Medication.”
For general information or to speak with a trained Client Services Specialist, please call MSAA’s Helpline at (800) 532-7667, extension 154. Questions to MSAA’s Client Services department may also be emailed to MSquestions@mymsaa.org.
Written by Susan Courtney, MSAA Senior Writer
Reviewed by Barry A. Singer, MD; Member of MSAA’s Board of Directors
