US FDA approves Scholar Rock drug for rare muscle-wasting disease

Sept 11 — The U.S. Food and Drug ​Administration approved Scholar Rock's therapy on Friday to treat ‌a rare genetic condition that causes muscle ⁠weakness.

The therapy, ​sold as Isembyld, ⁠is the first muscle-targeted ‌therapy designed to ‌improve motor function for adults ⁠and children ⁠with spinal muscular atrophy who have been treated with existing therapies for the condition.

Scholar Rock shares were halted in aftermarket ‌trading.

Spinal muscular atrophy ​is a genetic disorder that affects motor neurons, the nerve cells that control voluntary muscle movement. It causes progressive weakness in the arms and legs ​and difficulty walking, breathing ‌or swallowing.

The ‌condition ⁠is estimated to affect roughly 10,000 children and adults in the United States, according to the ‌Muscular Dystrophy Association.

(Reporting ​by Sneha ‌S K and ⁠Christy ​Santhosh in Bengaluru; Editing by Tasim ​Zahid)

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