FDA Approves First Drug to Treat Rare Form of Muscular Dystrophy

The Food and Drug Administration Monday approved the first drug to treat Duchenne muscular dystrophy, a rare and lethal muscle weakening disorder that affects about 15,000 Americans.

The FDA’s approval of the drug, Exondys 51, also known generically as eteplirsen, came over the objections of its own advisory committee, which voted not to approve the medication earlier this year. Patients and their families had lobbied hard for the drug, made by Sarepta Therapeutics of Cambridge, Massachusetts, noting that people with the disease have few treatment options. […]

The FDA’s decision speeds up the approval process for Exondys 51, allowing it onto the market based on preliminary data that suggests the drug will strengthen children’s muscles, even though the company has not yet produced clear proof that the medication will delay paralysis or improve symptoms.

In clinical trials, some patients treated with Exondys 51 had more dystrophin in their skeletal muscles, which people use to move their arms and legs. The FDA will require Sarepta to launch another clinical trial to show whether it actually improves patients’ symptoms. If the drug doesn’t help, the FDA could withdraw approval. […]

Some health advocates criticized the FDA’s decision.

Overruling the advisory committee shows “a disturbing disregard for the agency’s  legal standards for approving new drugs,” said Michael Carome, director of Public Citizen’s Health Research Group, a nonprofit that studies drug safety. “In particular, such action eviscerates the agency’s long-standing requirement that there be substantial evidence of effectiveness for new drugs — even drugs for serious rare diseases — before they are marketed.”

Diana Zuckerman, president of the National Center for Health Research, a nonprofit research group, noted that the Exondys 51 clinical trial was poorly done. Doctors leading the trial didn’t compare patients who received the drug with a “control group” of untreated patients.

“It sets a dangerous precedent if the FDA is going to start approving drugs that aren’t compared to anything,” Zuckerman said. “Why would a company choose to do a careful, well-designed study that might show that its product isn’t particularly safe or effective if it can get away with doing a tiny, poorly designed study with ambiguous results?” […]

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