Jennifer Hill Blair recognized the Cincinnati number on her buzzing phone and slipped out of the school meeting, excited to finally learn when her son Cayse would receive a therapy she believed would change his life. Blair, a second-grade teacher in southern Mississippi, and her husband had been fighting the state insurer since April, calling senators and representatives to lobby on their behalf.
Insurance approval finally came on July 9, Cayse’s fifth birthday. Doctors at Cincinnati Children’s, the closest treatment center, just had to give Blair a date to come in.
She answered. And she started to break down. It was their nurse, calling to say the Food and Drug Administration had asked the company behind the treatment to pull it off the market due to safety concerns. “We’re not really sure as to what’s going on,” the nurse said. “Just keep watching the news.”
Since then, regulators’ call for Sarepta Therapeutics to halt shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy, and the company’s subsequent decision to do so, have stoked confusion, fear, and heartbreak for Duchenne families across the country. Parents preparing to have their children infused after months fighting insurers, talking to doctors, and undergoing preparatory tests have had their appointments postponed indefinitely. The vast majority of these patients are young boys.
The unrelenting pace of the news alone has exacerbated the emotional strain. Since just last Thursday, parents, children, and young adults have received a whiplash-inducing string of updates, from media reports at all hours of the day: that another patient died from a related product, a death that Sarepta had failed to publicly disclose; that Sarepta initially refused the FDA’s request on Elevydis; that Sarepta reversed course and acceded to the agency’s call; and, on 2:30 p.m. Tuesday, that Elevidys, according to a senior FDA official, faces an “arduous” and “treacherous” path back to market.
“I think we have to remind ourselves that it is kids’ lives and families’ well-beings at stake,” said Stephen Chrzanowski, co-director of the the Duchenne Program at UMass Chan Medical School, in an email. “This is not a trivial decision, and the emotional rollercoaster for families is unfair and vicious.”
Elevidys, intended to slow a neurodegenerative disease that is ultimately fatal, had previously been a source of hope for many families, and remains so for many of them.
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