FDA Grants Accelerated Approval to Controversial Muscular Dystrophy Drug

After months of advocacy and speculation, the Food and Drug Administration today granted accelerated approval to the first treatment for a rare form of muscular dystrophy. The decision pitted the passionate testimony of patients and families against an FDA advisory committee and internal reviewers who weren’t convinced the drug worked.

The approval of Sarepta Therapeutics’ drug, eteplirsen, was a huge, emotionally fraught victory for families with Duchenne muscular dystrophy — one of the most vocal and involved patient communities since the days when HIV patients pushed the agency to approve more drugs. The approval specifies that the clinical benefit of the drug “has not been established” and is contingent on a follow-up clinical trial. The process has been closely watched by parents, patient advocates and biotech investors, and has been seen as an important test case for the effort to integrate the patients’ point of view more deeply into the approval process. […]

The agency usually follows the recommendation of its advisers, whose split vote against approval was met with angry outbursts at the conclusion of a long and contentious meeting in April. The FDA missed its initial deadline to decide on whether to approve the drug and requested more data from the company, fueling a rollercoaster of rumors about whether the drug would ultimately be approved. […]

The study supporting approval was a small trial with a dozen boys who carry a gene mutation that occurs in 13 percent of the 9,000 to 12,000 boys in the U.S. with Duchenne. FDA deemed that the trial did not show improvement on a walking test, but the study did show a measurable increase in dystrophin, the protein that is missing in the disease. There was significant dispute internally at the FDA over whether that increase was likely to be a predictor of a benefit. Internal documents released as part of the approval show that, in an unusual move, the decision to approve the drug was appealed to the highest levels of the FDA — to commissioner Robert Califf. […]

But critics of the decision worry that the approval of a drug — when internal scientists and an external advisory committee have found the evidence unconvincing — could be a slippery slope.

“If this drug can be approved under those conditions, is there any drug that FDA won’t approve?” said Diana Zuckerman, president of the National Center for Health Research, a nonprofit research organization. “This drug was based on the strong lobbying of patients and the company, and time will tell whether it will really help these boys or not, and that has always been the question.”

At a meeting in April, the agency walked a difficult high-wire act, visibly struggling to strike the balance between respecting the views of parents and boys who attributed their health to the drugs while also pointing out that the data showed scant evidence of the positive effects reported. […]

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