A Controversial Approval for a Duchenne Drug and What's Ahead

The controversial approval of Sarepta Therapeutics eteplirsen to treat a certain form of Duchenne muscular dystrophy has been viewed as a major victory for patient advocates. Advocates aggressively lobbied the U.S. Food and Drug Administration to grant approval for the drug despite a weak data package presented by the company. Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research overrode staff to greenlight the drug. We spoke to Debra Miller, president, CEO, and co-founder of CureDuchenne, about the significance of the approval, her organization’s venture philanthropy model, and what other hope the Duchenne drug pipeline may hold for patients.