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After a teen who had received Sarepta Therapeutics’s Elevidys for Duchenne muscular dystrophy (DMD) died of liver failure last month, pediatricians and families with children who got the drug are speaking out about its risks versus benefits.
Sarepta and Roche, the company’s partner outside the US, temporarily halted three clinical trials of the therapy in Europe at regulatory body the European Medicines Agency’s request, though on April 4, a group of independent trial monitors recommended they resume. But as pediatricians and families affected by Duchenne grapple with the tragedy, some are also standing firmly behind the drug.
Omer Abdul Hamid, a pediatric neurologist with Nemours Children’s Hospital, told Healthcare Brew the hospital is still offering Elevidys to all eligible patients, and that despite its risks, it “has shown promise for DMD patients.” Acute liver injury was already a documented risk associated with Elevidys and similar treatments, and the teen’s doctor said it’s possible a recent virus could have also contributed to his death, according to Sarepta.
That being said, “this incident is certainly a sobering reminder of the broad spectrum of potential outcomes with many therapies, especially those that are relatively new,” he added.
Rewinding
Duchenne is a terminal muscle-wasting disease, affecting about 1 in every 3,500 male births worldwide, according to the National Organization for Rare Disorders. Most patients require wheelchairs by their teenage years and typically die around age 30.
Though other companies are testing their own therapies, Elevidys is currently the only FDA-approved gene therapy for Duchenne, leaving families scrambling for it and putting Sarepta in a powerful position. More than 800 patients have received the drug in clinical trials and post-authorization prescriptions. Its sales made up $384.2 million of Sarepta’s total $638.2 million net product revenue in Q4 2024, according to a February earnings report.
Getting treatment and recognition for Duchenne has been an uphill battle propelled by parent-led lobbying and fundraising. Parent Project Muscular Dystrophy (PPMD) drafted community-led FDA treatment development guidance finalized in 2018. CureDuchenne, another parent-founded group, has raised and invested more than $3 billion to fund treatment research, including contributions to Sarepta, according to the group’s website.
Hope vs. risk
However, some FDA reviewers questioned whether Elevidys had sufficient evidence to be approved in the first place. Peter Marks, the FDA’s former director of the Center for Biologics Evaluation and Research, overruled his staff to give the drug accelerated approval in June 2023, then full approval in June 2024.
Lakeland, Florida, resident Stephanie Farley, whose 14-year-old son, Logan, has Duchenne and received the one-time Elevidys infusion in December, said she is “so thankful” the drug was approved to give her son at least a chance to halt or slow the disease’s progression.
She said her “heart breaks” over the recent death, but it has not made her regret her decision.
“You know when you’re signing up for it that there is risk to it, but there’s also ‘known risk’ to not doing anything,” Farley said.
If Farley’s son hadn’t gotten Elevidys, the family might have turned to a clinical trial.
“There was a clinical trial we were considering where Logan would have been one of the first humans to take it, and that’s way more risky,” she said.