Scholar Rock, a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced that the European Medicines Agency’s (EMA’s) Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending SRK-015 for designation as an orphan medicinal product for the treatment of SMA.
SRK-015 was previously granted orphan drug designation by the U.S. Food and Drug Administration (FDA) in March 2018.
“We are delighted with the EMA COMP’s adoption of a positive opinion for SRK-015 orphan drug designation, which represents another important milestone for this clinical program,” said Nagesh Mahanthappa, PhD, President and CEO of Scholar Rock. “We believe SRK-015 has the potential to be the first muscle-directed therapy to improve muscle function in patients with SMA and look forward to working with the EMA as we progress the program, including our intention to initiate a Phase 2 proof-of-concept study in the first quarter of 2019.”
Orphan Medicinal Product Designation by the European Commission is available to companies developing products intended to treat a life-threatening or chronically debilitating condition that affects fewer than five in 10,000 people in the EU, and has the potential to be of significant benefit. This designation could allow for a number of incentives, including protocol assistance, access to the centralised authorisation procedure, reduced regulatory fees and a 10-year period of marketing exclusivity in the EU after product approval.
SRK-015 is a selective inhibitor of the activation of myostatin and is an investigational product candidate for the treatment of patients with SMA. Myostatin, a member of the TGF-beta superfamily of growth factors, is expressed primarily by 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. A Phase 1 clinical trial in healthy volunteers is ongoing. The effectiveness and safety of SRK-015 have not been established and SRK-015 has not been approved by regulatory agencies.