Multiple sclerosis (MS) is a chronic inflammatory demyelinating disease of the central nervous system. MS affects about 400,000 patients in the United States, and the disease commonly begins in patients between the ages of 20 to 40 years. While the etiology of MS is unknown, it is thought to be immune-related and the result of genetic and environmental factors, such as cigarette smoking and UV light exposure. There are four disease courses: clinically isolated syndrome, relapsing-remitting MS, secondary progressive MS (SPMS), and primary progressive MS (PPMS). More common symptoms that patients may experience include muscle weakness, visual disturbances, trouble with coordination and balance, numbness or tingling, and cognitive changes. Currently, there is no cure for the disease, but the following treatment approaches are available: treatment of exacerbations (or relapses), management of symptoms, and treatment to modify the disease course.

Not all exacerbations require treatment, but depending on the severity and nature of the exacerbation, a short course of high-dose corticosteroids may be used to shorten the time to recovery from the relapse. Other treatment options for exacerbations include corticotropin therapy. There are also various treatments for the management of MS symptoms. The long-term goal of treatment is to slow or modify the course of the disease by implementing disease modifying therapy (DMT). There are currently 15 DMT agents that are approved in the United States. DMT slows down disease progression and reduces disease activity. DMT is used in patients who suffer from relapsing forms of MS, as well as those who have the progressive form of the disease with relapses.

The industry is making strides in the development of additional therapeutic options for the treatment of MS, including several new medications in the pipeline. An agent that was granted breakthrough therapy designation, fast track designation, and priority review by the FDA, Ocrevus (ocrelizumab), was approved on March 28, 2017 for the treatment of relapsing forms of MS and PPMS. Ocrevus is the first medication to be approved for the treatment of PPMS. Another advancement in treatment includes agents called sphingosine-1-phosphate receptor immunomodulators (ponesimod, siponimod, ozanimod), which are all in phase 3 trials for the treatment of MS. One of these drugs, siponimod, is being studied for the treatment of SPMS; there is currently only one drug that is specifically approved for SPMS, mitoxantrone. Another agent in a phase 3 trial is masitinib. Masitinib is a tyrosine kinase inhibitor that is being studied for PPMS and relapse-free SPMS. If approved, these agents could offer additional treatment options to patients, especially for the progressive forms of MS.

Please update or register your profile to receive email alerts and other critical drug information updates from PDR. You can also stay current by using the official PDR app, available now for free from your favorite app stores.