FDA Grants Breakthrough Designation to Multiple Sclerosis Treatment

The FDA has granted Breakthrough Therapy Designation for an investigational antibody drug for the treatment of primary progressive multiple sclerosis (PPMS).

PPMS is a debilitating form of multiple sclerosis that affects 1 in 10 patients with MS. It is characterized by worsening symptoms that don’t have distinct relapses or periods of remission.

The investigational humanized monoclonal antibody ocrelizumab (Ocrevus) was manufactured by Genentech to target immune cell CD20-positive B cells, which are thought to be a key contributor to myelin and axonal damage that can lead to disability for MS patients.

“Ocrelizumab is the first investigational medicine for MS to be granted Breakthrough Therapy Designation by the FDA,” said head of Global Product Development, Sandra Horning, MD. “With no approved treatments for primary progressive MS, ocrelizumab has the potential to address an important unmet need. We are committed to working with the FDA to bring ocrelizumab to people with primary progressive MS as quickly as possible.”

The designation followed positive results from a phase 3 double-blind global multi-center study that showed a significant reduction in disability progression and other markers of disease activity in patients administered Ocrevus compared with placebo.

In prior preclinical studies, it was found that Ocrevus binds to CD20 cell surface proteins, which are expressed on certain B cells, but not on plasma cells or stem cells. This allows important functions to be preserved in the immune system.

In the first half of 2016, Genentech plans to submit data from 3 pivotal phase 3 studies to the FDA for marketing authorization for PPMS and relapsing multiple sclerosis.