Scholar Rock announced that the Food and Drug Administration (FDA) has granted Orphan Drug Designation to SRK-015, a drug candidate for the treatment of spinal muscular atrophy

Spinal muscular dystrophy is a rare genetic disorder that commonly manifests in young children. Patients experience loss of motor neurons, atrophy of the voluntary muscles of the limbs and trunk as well as progressive muscle weakness. 

SRK-015 is a selective first-in-class inhibitor of the activation of myostatin, a growth factor found mostly in skeletal muscle cells to inhibit muscle growth. By inhibiting the activation of myostatin, SRK-015 may promote a clinically meaningful increase in muscle mass and strength. SRK-015, given as monotherapy or in combination with standard of care, may serve as the first muscle-directed therapy to reverse or prevent muscle atrophy in patients who have spinal muscular dystrophy. 

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The Company anticipates a first-in-human Phase 1 clinical trial in the 2nd quarter of 2018. 

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