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."