Capricor Therapeutics' Becker Muscular Dystrophy Drug Gets Orphan Drug Designation in U.S.

Dow Jones

2025/06/17

By Adriano Marchese

Capricor Therapeutics has received an orphan drug designation for its drug aimed at potentially treating Becker Muscular Dystrophy.

The biotechnology company said Tuesday that the Food and Drug Administration granted the special status to Deramiocel, the company's lead cell therapy candidate.

Becker Muscular Dystrophy, much like Duchenne Muscular Dystrophy, is a type of genetic disorder that causes progressive muscle weakness and degeneration over time, primarily in males.

Chief Executive Linda Marban said the drug has show to have the potential to treat the cardiac and skeletal muscle complications of Duchenne Muscular Dystrophy and "based on the overlap in disease pathology, may also offer benefit to patients with Becker Muscular Dystrophy."

Write to Adriano Marchese at adriano.marchese@wsj.com

(END) Dow Jones Newswires

June 17, 2025 09:23 ET (13:23 GMT)

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