Parents Push One Rare-Disease Drug Through FDA, Not Its Successor
The agency’s rejection of a new therapy for treating a deadly condition may signal a tougher FDA, and more risks for makers of rare-disease drugs.
A more flexible FDA? Not so fast.
Photographer: Congressional Quarterly/CQ-Roll Call GroupFor a small biotechnology firm, Sarepta Therapeutics Inc. has made a lot of waves at the Food and Drug Administration. The 2016 approval of its first drug, Exondys 51 – which targets the deadly muscle-wasting disease Duchenne Muscular Dystrophy (DMD) – caused a schism at the agency. A highly organized and vocal group of parents and patients saw hope for boys otherwise resigned to a short and challenging life, helping it gain green-light status, while an agency detractor called the drug an “elegant placebo.”
Sarepta’s second drug-approval attempt didn’t go so well. On Monday, the FDA rejected the company’s follow-up drug Vyondys 53, intended for another subset of DMD patients. According to the surprised company, the agency’s principal concerns were with infections related to ports used to infuse the drug, and kidney toxicity observed in pre-clinical models but not seen in the actual trial.