FDA Approves Multiple Sclerosis Therapy

(commonfund.nih.gov)

29 March 2017. The Food and Drug Administration approved a synthetic targeted antibody to treat multiple sclerosis in people with relapsing or steadily worsening forms of the disease. The therapy approved by FDA for marketing in the U.S. is ocrelizumab, marketed as Ocrevus and developed by Genentech in South San Francisco, California, a division of pharmaceutical company Roche.

Multiple sclerosis is an autoimmune condition where the immune system attacks the central nervous system and damages myelin, the fatty, protective substance around nerve fibers, as well as nerve cells themselves. Scar tissue from the damaged myelin, known as sclerosis, distorts the nerve signals sent to and from the brain and spinal cord, causing symptoms ranging from mild numbness to loss of vision or paralysis.

FDA approved ocrelizumab for people whose multiple sclerosis relapses, as well as primary progressive multiple sclerosis, a form of the disorder marked by steady deterioration in neurological functioning, without periods of remission or relapse. A 2010 study estimates 15 percent of Americans with multiple sclerosis have the primary progressive form of the disease.

Ocrelizumab is a synthetic humanized antibody designed specifically to address immune system cells known as CD20-positive B cells. B cells are white blood cells produced in the bone marrow that produce antibodies. CD20-positive B cells are considered contributors to damage in myelin and nerve cells associated with multiple sclerosis. Ocrelizumab binds to proteins emitted on the surface of CD20-positive B cells, but not on other immune system cells, and thus can avoid damaging other immune functions.

FDA based its approval in part on three late-stage clinical trials evaluating ocrelizumab against other treatments or a placebo. Two of the trials tested ocrelizumab against another biologic therapy, interferon beta-1a, among a total of 1,656 participants with relapsing multiple sclerosis. In these studies, ocrelizumab reduced the number of relapses and brain lesions revealed by MRI scans, as well as slowed the deterioration leading to disability, compared to interferon beta-1a, over 2 years

A separate clinical trial tested ocrelizumab against a placebo among 732 individuals with primary progressive multiple sclerosis, tracking participants for at least 120 weeks. Results show fewer participants receiving ocrelizumab in that period experienced declining indicators of disability and brain lesions from MRI scans, compared to placebo recipients.

Ocrelizumab is given as an intravenous infusion by a clinician. Most adverse reactions in the trials were related to infusions, but in the primary progressive multiple sclerosis trial, more recipients of ocrelizumab experienced upper respiratory tract and oral herpes infections than placebo recipients. Genentech says rates of serious adverse effects in the trials were similar for ocrelizumab participants as those in the comparison groups.

FDA used several of its special programs to accelerate the review process for ocrelizumab. The experimental therapy received a breakthrough designation, fast-track status, and priority review during its evaluation by the agency.

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