Decision Resources forecasts the launch of four new agents to treat DMD by 2021

Decision Resources, one of the world's leading research and advisory firms focusing on pharmaceutical and healthcare issues, finds that interviewed experts unanimously identify a disease-modifying therapy as the highest unmet need in the treatment of Duchenne muscular dystrophy (DMD). Duchenne is the most common form of muscular dystrophy in children.    

"Glucocorticoid treatment is the only available therapy for the treatment of DMD," said Decision Resources Analyst Richa Mukherjee, M.Sc., M.Phil., Ph.D. "It's a symptomatic therapy that does not target the cause of the disease and its use is often accompanied by concerning side effects. The lack of a disease-modifying therapy to treat DMD presents a high unmet need for drug developers."

According to Niche Markets and Rare Diseases: Muscular Dystrophy, Decision Resources forecasts the launch of four new agents to treat DMD by 2021. These therapies include GlaxoSmithKline/Prosensa's GSK-2402968/PRO-051/drisapersen, Santhera/Takeda's Catena/Sovirma's idebenone, AVI BioPharma's eteplirsen/AVI-4658 and Prosensa's PRO-044. Of these therapies, GSK-2402968, eteplirsen and PRO-044 are disease-modifying therapies that will heavily saturate their target patient segments however, given the small size of these segments, the market will still be very receptive to novel disease-modifying agents.

"The DMD market will grow as a result of polytherapy as patients will be treated with mutation-specific disease-modifying drugs in addition to symptomatic treatments like idebenone," said Dr. Mukherjee. "Symptomatic therapies will continue to play a major role for a majority of DMD patients because the new disease-modifying agents target only a subset of the DMD patient population."