Scholar Rock announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead antibody product candidate, SRK-015, for the treatment of spinal muscular atrophy (SMA). SRK-015 is an inhibitor of the activation of myostatin, which Scholar Rock believes has the potential to be the first muscle-directed therapy to reverse or prevent muscle atrophy in SMA patients and could be used both as a monotherapy or in conjunction with the current standard of care.
“We are very pleased that the FDA granted Orphan Drug Designation to SRK-015 for the treatment of patients suffering from SMA, and we appreciate that the agency’s decision came much earlier than anticipated,” said Nagesh Mahanthappa, PhD, President and Chief Executive Officer of Scholar Rock. “This designation is an important milestone in the development of our lead product candidate along the path towards a first-in-human Phase 1 clinical trial in the second quarter of 2018.”
Under the U.S. Orphan Drug Act, FDA's Office of Orphan Products Development (OOPD) grants orphan drug designation to drugs and biologic products that are intended for the treatment of rare diseases or disorders that affect fewer than 200,000 people in the U.S. Orphan drug status is intended to facilitate drug development for rare diseases and may provide several benefits and incentives to drug developers, including assistance with clinical study design and drug development, tax credits for qualified clinical trials costs, and seven years of market exclusivity upon regulatory product approval.
SRK-015 is a selective first-in-class inhibitor of the activation of myostatin. Myostatin, a member of the TGF-beta superfamily of growth factors, is expressed primarily in skeletal muscle cells and the absence of its gene is associated with an increase in muscle mass and strength in multiple animal species. Scholar Rock believes the inhibition of the activation of myostatin with SRK-015 may promote a clinically meaningful increase in muscle mass and strength. Scholar Rock is advancing SRK-015 into clinical development for the treatment of patients with SMA. SRK-015 is an investigational drug candidate. The effectiveness and safety of SRK-015 have not been established and SRK-015 has not been approved by the FDA or any other regulatory agency.