Scholar Rock Announces Positive Opinion by the European Medicines Agency

Scholar Rock (NASDAQ:SRRK), 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 (human anti-promyostatin monoclonal antibody) for designation as an orphan medicinal product for the treatment of Spinal Muscular Atrophy (SMA).

As quoted in the press release:

“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 centralized authorization procedure, reduced regulatory fees, and a 10-year period of marketing exclusivity in the EU after product approval.

Click here to read the full press release.