FDA Grants Accelerated Approval for Sarepta's $3.2M Gene Therapy for Duchenne Muscular Dystrophy
TL;DR Summary
Peter Marks, the senior FDA official overseeing gene therapies, overrode agency reviewers' decision to reject Sarepta Therapeutics' Duchenne muscular dystrophy gene therapy after disagreeing with parts of their interpretation of the limited efficacy data. The FDA granted accelerated approval to Sarepta's gene therapy, marketed as Elevidys, in boys aged 4 and 5 years old.
Topics:business#duchenne-muscular-dystrophy#fda#gene-therapy#healthcare#peter-marks#sarepta-therapeutics
- FDA's Peter Marks overrode reviewers' call to reject Sarepta's new Duchenne gene therapy Endpoints News
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- Cambridge-based Sarepta Therapeutics to charge $3.2M for new Duchenne muscular dystrophy treatment WCVB Boston
- FDA approves first gene therapy for muscular dystrophy WQAD News 8
- Sarepta’s Duchenne muscular dystrophy gene therapy granted FDA accelerated approval PMLiVE
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