() An Illinois family is urging the Food and Drug Administration to restore access to Sarepta Therapeutics’ Elevidys, the only FDA-approved treatment for Duchenne’s muscular dystrophy.
Two of Alison and William Small’s three sons, Hunter and Noah, are diagnosed with Duchenne’s. Noah, 9, received the treatment in March. After three months of close monitoring and with no complications, the family said he’s made progress, including being able to swim and ride a bike.
But a recent FDA investigation has paused further distribution of Elevidys, following reports of at least three patient deaths, including an 8-year-old boy who died June 7 in Brazil.
The FDA said the deaths appear to have been a result of acute liver failure in those treated with Elevidys. However, Roche, which markets the drug outside the U.S. in partnership with Sarepta, confirmed the boy who died in Brazil was not enrolled in a clinical trial.
Sarepta voluntarily and temporarily halted distribution on July 22 to allow time to address FDA safety labeling updates and respond to regulatory inquiries.
‘We’re looking for clarity and transparency’: Father
The Smalls had been working through the insurance approval process for their 10-year-old son, Hunter, to receive the same treatment. That process is now on hold.
Speaking to ‘s “Morning in America” on Monday, the Smalls called for transparency and a clear path forward from federal regulators. They said families affected by Duchenne’s deserve to understand the risks and that many believe the treatment is worth fighting for.
“Whether it’s through the administration or working with the FDA, what we’re looking for is some clarity and transparency … especially talking to the DMD community and families to understand where the risks are, why we believe this drug is worth fighting for and worth dosing to our son and the sons of other people in the community,” William Small said.
The family’s personal connection to Duchenne’s led them to establish the Small Heroes Foundation, which supports families affected by the disease. They’re now among a growing group of advocates calling on federal regulators to allow continued access to Elevidys for families who are willing to accept the risks.
Neither the FDA nor Sarepta has provided a timeline for when the pause may be lifted.
What is Duchenne’s muscular dystrophy?
Duchenne’s is a rare genetic condition that primarily affects boys, causing progressive muscle degeneration and premature death, according to the Muscular Dystrophy Association. It occurs in 1 in 3,500 to 5,000 male births.
Without treatment, most patients lose the ability to walk in their early teens and often do not survive into adulthood.
Elevidys is the only gene therapy approved by the FDA for the treatment of this disease.
