Research News: FDA Approval and Review of MS Medications
By Susan Wells Courtney
Reviewed by Dr. Jack Burks
Portions reviewed by Barry A. Singer, MD
FDA Approves Zinbryta™ (Daclizumab) for Relapsing Forms of Multiple Sclerosis
On May 27, 2016, the United States Food and Drug Administration (FDA) announced the approval of Zinbryta™ (daclizumab) for adults with relapsing forms of multiple sclerosis (RMS). This monoclonal antibody is self-administered subcutaneously (under-the-skin) once per month and has been shown to reduce the number of relapses as well as new or newly enhancing lesions, as compared to Avonex® (interferon beta-1a) and to placebo, in two separate studies. Zinbryta is co-promoted in the United States by Biogen and AbbVie.
While the FDA approved Zinbryta because they determined that the benefits outweigh the potential for adverse events, this medication does carry safety risks, which include liver injury and immune conditions. Monthly liver-function tests are required with Zinbryta, and it is available only through a restricted distribution program under a Risk Evaluation and Mitigation Strategy.
Daclizumab (Zinbryta) is a genetically engineered monoclonal antibody that binds to CD25, a receptor on T cells that is thought to become activated in response to MS. Daclizumab is believed to work by selectively targeting these activated T cells without causing general T-cell depletion. For more information, please contact Biogen’s Above MS™ program at (800) 456-2255 or visit www.zinbryta.com.
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 had been accepted for review by the FDA. This is the first time that an investigational medication is being reviewed for the treatment of both relapsing forms of MS (RMS) and primary-progressive MS (PPMS). A decision for approval will be made on or before December 28, 2016. Genentech has submitted the brand name Ocrevus™ for use with ocrelizumab.
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.
Positive results were seen in three Phase III trials with ocrelizumab, including reductions in annualized relapse rates and reductions in disability progression that was sustained for time periods of at least three and at least six months. Genentech reports that the most common adverse events were mild to moderate infusion-related reactions and infections.
Eligible People with PPMS May Receive Ocrelizumab
Also announced in late June, the experimental medication ocrelizumab is available for eligible individuals with PPMS through an Expanded Access Program (EAP). Please note that only people with PPMS, not with RMS, are able to participate in this program. This program is a nontraditional study that has no placebo group and follows a strict protocol developed through consultation with the FDA.
To take part in ocrelizumab’s EAP, individuals must be 18 to 55 years of age, have a PPMS diagnosis, and have an Expanded Disability Status Score (EDSS) of 2.0 to 6.5 points. Women of child-bearing potential will need to check with their program doctor about birth-control options. For more information please call Genentech’s Trial Information Support Line at (888) 662-6728.
Experimental SPMS Medication Shows Positive Results
On August 25, 2016, Novartis announced that siponimod, an experimental oral treatment for MS, is showing positive results for individuals with secondary-progressive MS (SPMS). Novartis, the pharmaceutical company developing siponimod, announced that the Phase III EXPAND study met its primary endpoint, which was to reduce the risk of confirmed disability progression at three months, versus a placebo.
Siponimod, also known as BAF312, is in a class of immunomodulatory drugs called “S1P-receptor modulators,” which also includes Gilenya® (fingolimod). The structure of these medications is similar to a naturally occurring component of cell-surface receptors on white blood cells, and block potentially damaging T cells from leaving lymph nodes. They may reduce damage to the central nervous system (CNS) and enhance the repair of damaged nerves within the brain and spinal cord.
For more information on these and other topics, please go to mymsaa.org/news.
For any MS-related questions, please contact MSAA’s Client Services Specialists via email at MSquestions@mymsaa.org, via phone at (800) 532-7667, extension 154, or via our interactive one-on-one chat feature, at mymsaa.org/chat.