FDAnews Drug Daily Bulletin
Pharmaceuticals / Regulatory Affairs

PTC Therapeutics Wins Expanded Label for Duchenne Muscular Dystrophy

June 19, 2019
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PTC Therapeutics has won FDA approval of Emflaza (deflazacort) to treat children between two years and five years old for Duchenne muscular dystrophy.

The corticosteroid was originally approved in 2017 for patients five years and older.

Emflaza is currently the only drug of its kind for patients two years and older with Duchenne muscular dystrophy, a rare genetic disorder that causes progressive muscle deterioration.

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