888-633-4029

Despite many disagreements within the agency, the FDA recently approved the first drug for Duchenne muscular dystrophy. This decision was largely based on a single study of Sarepta Therapeutics Inc.’s drug, Eteplirsen, featuring 12 patients with this crippling disease. This small study as well as the strong, emotional responses from parents of children with muscular dystrophy, swayed the opinions of those on the FDA advisory panel.

Although, with still a great lack of data and information, many FDA experts argued that Eteplirsen was not fit for approval. Against their judgment, a powerful figure in the FDA, Dr. Woodcock, decided to push the accelerated approval causing a greater uproar.

In an article, “FDA Approves Sarepta’s Muscular Dystrophy Drug”, Thomas M. Burton quotes the director of Public Citizen Health Research Group, Michael A Carome:

“The decision by Dr. Woodcock to approve eteplirsen, against the strong objections of FDA experts who reviewed the drug and the advice of its advisory committee, represents a disturbing disregard for the agency’s legal standards for approving new drugs. In particular, such action eviscerates the FDA’s longstanding requirement that there be substantial evidence of effectiveness for new drugs—even drugs for serious rare diseases—before they are marketed.”

This accelerated approval of Eteplirsen showcases the broken drug pipeline. The FDA advisory panel’s job is to look at the bigger picture of the drug’s effectiveness, as well as the unintended consequences, to determine if a drug is fit for approval. In this case, many members on the board were easily swayed by emotion and the power of a controversial figure going outside of the given requirements.

Read the full article, FDA Approves Sarepta’s Muscular Dystrophy Drug, from the Wall Street Journal for more information about this unique situation.