Scholar Rock Experimental Drug for SMA Granted Orphan Drug Status

03 May 2018

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to SRK-015 for the treatment of SMA. SRK-015 is the lead drug candidate of pharmaceutical company Scholar Rock. Orphan Drug Designation gives incentives to companies to pursue therapies intended to treat rare conditions in order to facilitate the drug development process for conditions like SMA.

SRK-015 is an SMN-independent therapy, meaning that it is not designed to increase the levels of the SMN protein that is lacking in SMA patients. Instead, SRK-015 is a muscle-directed therapy that aims to reverse or restrict the muscle atrophy and weakness experienced by SMA patients. The drug is an antibody that very specifically targets an important protein called myostatin.

When myostatin is present in muscles, it can restrict their growth and function. By targeting myostatin for removal, SRK-015 can potentially release the breaks on muscle cell growth leading to an increase in muscle size and function. If this is successful, this could benefit SMA patients.

Orphan Drug Designation should help to expedite the development of SRK-015 as a potential therapeutic option for SMA. Scholar Rock hope to test SRK-015 in a first-in-human Phase 1 clinical trial in the second quarter of 2018.


Further Information

Scholar Rock Press Release