The U.S. Food and Drug Administration has approved ocrelizumab, a new drug to treat adult patients with relapsing forms of multiple sclerosis (MS) and primary progressive multiple sclerosis (PPMS). This is the first drug approved by the FDA for PPMS. Marketed under the name Ocrevus, the drug is an intravenous infusion given by a health-care professional.
“Multiple sclerosis can have a profound impact on a person’s life,” said Billy Dunn, M.D., director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “This therapy not only provides another treatment option for those with relapsing MS, but for the first time provides an approved therapy for those with primary progressive MS.”
MS is a chronic, inflammatory, autoimmune disease of the central nervous system that disrupts communication between the brain and other parts of the body. It is among the most common causes of neurological disability in young adults and occurs more frequently in women than men. For most people with MS, episodes of worsening function (relapses) are initially followed by recovery periods (remissions). Over time, recovery may be incomplete, leading to progressive decline in function and increased disability. Most people experience their first symptoms of MS between the ages of 20 and 40.
PPMS is characterized by steadily worsening function from the onset of symptoms, often without early relapses or remissions. The U.S. Centers for Disease Control and Prevention estimates that approximately 15 percent of patients with MS have PPMS.
Dr. Stephen Hauser of the University of California-San Francisco, whose research led to the drug’s development, told STAT News: “This is a big deal for people with MS.”
The efficacy of Ocrevus for the treatment of relapsing forms of MS was shown in two clinical trials in 1,656 participants treated for 96 weeks. Both studies compared Ocrevus to another MS drug, Rebif (interferon beta-1a). In both studies, the patients receiving Ocrevus had reduced relapse rates and reduced worsening of disability compared to Rebif.
In a study of PPMS in 732 participants treated for at least 120 weeks, those receiving Ocrevus showed a longer time to the worsening of disability compared to placebo.
“Until now, no FDA-approved treatment has been available to the primary progressive MS community, and some people with relapsing forms of MS continue to experience disease activity and disability progression despite available therapies,” says Dr. Sandra Horning, chief medical officer of Genentech, a member of the Roche Group. “We believe Ocrevus, given every six months, has the potential to change the disease course for people with MS, and we are committed to helping those who can benefit gain access to our medicine.”
Genentech says the medication will be available in the U.S. within two weeks.