How the FDA Approved a $300,000-a-Year Drug Its Own Experts Didn’t Believe Worked – By Susan Pulliam and Brody Mullins


After trial data for a new medicine proved inconclusive, Sarepta Therapeutics joined with the parents of sick boys to persuade officials it helped

Jennifer McNary, a stay-at-home mother, was desperate to find a medicine that might spare her two sons an early death from a rare form of muscular dystrophy.

Chris Garabedian, the chief executive of a pharmaceutical firm, was desperate to find a profitable drug that would reverse his company’s slow fall.

They met in June 2012 at a conference on Duchenne muscular dystrophy and joined forces, often behind the scenes and with little public disclosure, in a yearslong mission to push the government to approve a drug to treat DMD, as it is known.

At the time, it wasn’t at all clear the firm’s drug worked. Faced with government skepticism, a consultant to Mr. Garabedian’s company helped Ms. McNary and other parents prepare slickly packaged testimony to convince the Food and Drug Administration that the drug was helping their sick children, according to parents, company managers and the consultant.

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