FDA Grants Breakthrough Therapy Status for Roche’s MS DrugBy
The US Food and Drug Administration has granted breakthrough therapy designation for Roche’s investigational medicine, ocrelizumab, for treating people with primary progressive multiple sclerosis (PPMS). There are currently no approved treatments for PPMS, a debilitating form of MS characterized by steadily worsening symptoms and typically without distinct relapses or periods of remission.
Roche plans to pursue marketing authorization for both PPMS and relapsing multiple sclerosis (RMS), a more common form of the disease, and will submit data from three Phase III studies to global regulatory authorities in the first half of 2016.
Ocrelizumab is an investigational, humanized monoclonal antibody designed to selectively target CD20-positive B cells. CD20-positive B cells are a specific type of immune cell thought to be a key contributor to myelin (nerve cell insulation and support) and axonal (nerve cell) damage, which can result in disability in people with MS. Based on preclinical studies, ocrelizumab binds to CD20 cell surface proteins expressed on certain B cells, but not on stem cells or plasma cells, and therefore important functions of the immune system may be preserved.