RARECast: A Controversial Approval for a Duchenne Drug and What Lies 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.

Daniel S. Levine is an award-winning business journalist who has reported on the life sciences, economic development, and business policy issues throughout his 25-year career. He founded Levine Media Group in 2013 to provide strategic communications to life sciences companies. He is host of The Bio Report and RARECast podcasts, a senior fellow at the Center for Medicine in the Public Interest, and a member of the advisory board of the California Biotechnology Council.

What do you think about the approval of Eteplirsen?