BioMarin's Drisapersen 'Compete Response' Shows FDA Flexibility Still Limited

FDA wants another clinical trial for Duchenne muscular dystrophy treatment; focus moves to Sarepta's candidate, which gets committee review next week.

It appears that FDA's celebrated approval flexibility for rare disease treatments has its limits, especially when efficacy is questioned.

Agency officials issued a "complete response" letter for BioMarin Pharmaceutical Inc

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