FDA Approves First Treatment for Primary-Progressive Forms of Multiple Sclerosis

Genentech announced on March 28, 2017 the landmark approval of its monoclonal antibody (mAb)-based therapy Ocrevus (ocrelizumab), the first treatment approved by the agency that targets progressive forms of multiple sclerosis. The medication was also approved to treat relapsing-remitting forms of the disease, which affect a larger population of patients than the primary progressive form.  In clinical trials, results of which were published in the New England Journal of Medicine in December 2017, ocrelizumab demonstrated superior efficacy to Rebif (interferon beta-1a), a widely used treatment for relapsing forms of the disease. In two trials, OPERA I and OPERA II, the humanized mAb cut annualized relapse rates almost in half when compared with Rebif, and significantly helped improve measurements of walking speed, upper-limb movements, and cognition. Despite its benefits, infusion-related reactions occurred in approximately 34% of patients, and FDA warns that the drug can lead to malignancies in some patients.

Most of the existing treatments for relapsing multiple sclerosis (MS) and primary progressive MS (PPMS) work to manage the symptoms of the immune-mediated condition, which is characterized by damage to myelin surrounding nerve fibers of the central nervous system. Symptoms are associated with gradual CNS breakdown, and include fatigue, weakness, spasticity, weakness, numbness, problems with gait, vision problems, and gradual cognitive decline. Approximately 60,000 people are estimated to suffer from the progressive form of the disease, which is about 15% of the total population of people with MS, according to the National Multiple Sclerosis Society. 

Ocrevus is to be administered by intravenous infusion every six months, and Roche, the arm of Genentech commercializing the medication, says it will be available within two weeks. The drug is genetically engineered to selectively bind to CD20-positive B cells and block their action. Because ocrelizumab does not bind to stem cells or plasma cells, Roche says the immune system will remain somewhat intact following the elimination of mature B cells. The approval comes a bit later than previously expected, as FDA extended review of the application for Ocrevus so that Roche could provide extra data surrounding the commercial manufacture of the drug.

According to numerous news outlets, Roche reportedly said it would charge $65,000/year for the drug, which is approximately 24% less than the current price of Rebif ($86,000/year).

Source: Genentech, FDA