Eligible People with PPMS May Receive Experimental Medication
Ocrelizumab, an experimental medication presently under investigation for the treatment of primary-progressive multiple sclerosis (PPMS), will be available for 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 United 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.
While 14 disease-modifying therapies are available for the treatment of relapsing forms of MS, no treatments have been approved by the FDA for PPMS, a less-common form of MS that is characterized by a steady accumulation of symptoms versus flare-ups and remissions. To take part in ocrelizumab’s EAP, individuals must meet the following specific study criteria:
- 18 to 55 years of age
- PPMS diagnosis
- 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
Medical records from one’s treating physician will be required. Exclusion criteria include a history of any other form of MS, a history of certain medical conditions, and a history of certain treatments. These and other study details may be found by referencing ClinicalTrials.gov identifier NCT02807285.
Through ocrelizumab’s EAP, enrolled participants will receive the study medication, given every six months via two infusions, which are separated by two weeks. The study sponsor will cover the cost of program-specific medical procedures performed during this program outside the costs for standard-of-care medical procedures. However, if and when ocrelizumab is approved by the FDA, participants wishing to continue treatment would need to convert to obtaining the treatment through their doctor’s prescription and their insurance coverage or other method of purchase, following a limited transition period.
Genentech, a member of the Roche group, is the company developing ocrelizumab and conducting its EAP. They note that about 50 study sites across the country will be participating in this program, although not all have been identified as of this time. Genentech’s support staff is available to take calls from anyone interested in learning more about this program. Their Trial Information Support Line may be reached by calling (888) 662-6728. While members of the MS community are invited to contact this support line for more information, their physicians will need to use this line to begin the application process.
Ocrelizumab is an investigational, humanized monoclonal antibody designed to selectively target CD20-positive B cells. Genentech is also studying this medication for the treatment of relapsing forms of MS. 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.”
In other news regarding ocrelizumab, on June 27, 2016, Genentech announced that the Biologics License Application (BLA) for ocrelizumab – to treat both relapsing forms of MS as well as PPMS – has been accepted for review by the United States Food and Drug Administration (FDA). 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. For more information, please see MSAA’s article, “FDA Accepts Review of Application for Ocrelizumab.”
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