Robert Langreth, Fiona Rutherford, Immanual John Milton and Madeline Campbell, Bloomberg News, May 12, 2023
When Exondys 51 was approved to treat Duchenne muscular dystrophy, a deadly disease that puts kids in wheelchairs by the time they are teenagers, there was no proof the drug actually slowed the disease. That was seven years ago. The company still hasn’t provided conclusive data to this day. The drug’s maker, Sarepta Therapeutics Inc., has so far reaped more than $2.5 billion in sales from Exondys 51 and two related drugs. All three were cleared via a US regulatory shortcut called accelerated approval that allows companies to market medications before completing definitive trials in cases where patients have few or no treatment options.
In the case of Exondys, Sarepta’s confirmatory study didn’t even begin until four years after it was approved and won’t be completed until 2024, eight years after it started selling the drug.
This is not uncommon. A Bloomberg News analysis of Food and Drug Administration databases found 19 drugs with accelerated approvals whose confirmatory trials are still listed as delayed as of April.
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What started out as a relatively obscure way for drugmakers to get new medicines to patients, accelerated approval is now a business model for some companies. Critics argue the FDA hasn’t done enough to force companies to produce conclusive data on drugs they’re already selling.
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Sarepta sought approval for Exondys in Europe, but regulators rejected it in 2018 saying the studies “were not satisfactory for showing that the medicine was effective.” The company appealed that decision, but the regulators held firm.
Since then, Sarepta has gotten two more drugs for muscular dystrophy approved in the US through the accelerated pathway. “Sarepta is a poster child for an absolutely outrageous and inexcusable lack of any kind of oversight or enforcement,” said Diana Zuckerman, president of the National Center for Health Research, a nonprofit think tank. Sarepta said it is “fully committed” to completing the trial for Exondys and attributes the delays to a variety of factors including some requirements imposed by the FDA. A confirmatory trial for the company’s other two drugs is enrolled and expected to be complete in 2025.
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In December, with support of the FDA, Congress passed a new law that says the FDA “may” require confirmatory studies be underway at the time of an accelerated approval. It spells out a process the FDA can use to withdraw accelerated approvals, too. “Congress gave us the teeth,” FDA Commissioner Robert Califf said in January.
Not everyone is so sure. The new law is subject to interpretation and doesn’t eliminate the possibility of delayed trials, says Thomas Hwang, a physician at Harvard Medical School.
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