FDA allows distribution of muscular dystrophy drug again after public criticism

After blocking a new muscular dystrophy therapy in order to investigate patient deaths, the Federal Drug Administration reversed course after criticism and restored hope to parents seeking a treatment to aid children affected by the debilitating disease. 

The FDA’s initial decision to freeze distribution of the therapy, marketed as Elevidys, following at least three patient deaths potentially connected to the therapy, stirred criticism from advocates for muscular dystrophy patients and from health policy experts. Added to the mix of criticism about the initial decision were the attacks and defenses of Dr. Vinay Prasad, the agency’s top biologics official, who supported progressive candidates and criticized several of President Trump’s key health policies in the past. 

The company that produced the therapy, Sarepta Therapeutics, initially resisted the freeze, but eventually paused shipments of its innovative product, opening its stocks up to a hammering that threatened to put the company's future in serious jeopardy. According to financial analytics website companiesmarketcap.com, Sarepta's market cap fell yesterday to $1.56 billion from $14 billion a year ago. Market capitalization, commonly called market cap, is the total market value of a publicly traded company's outstanding shares — held by institutional investors and the public at large — and is commonly used to measure how much a company is worth.

The "Right-to-try"

The reversal of the pause aligns with President Donald Trump’s longtime advocacy for the right-to-try. During his first term, the president signed a new law giving terminally ill patients the opportunity to seek new or experimental treatments without obtaining the approval of the FDA, which had been required in the past. 

The FDA demanded on July 18 that Sarepta pull Elevidys from the market and halt all shipments to patients after reports of two deaths connected to the treatment. Elevidys is not an experimental drug, and was approved by the FDA under certain circumstances in 2024. It is a prescription gene therapy designed to treat patients with Duchenne Muscular Dystrophy, a genetic disorder that leads to the breakdown and decay of muscles over time and mostly impacts male children. 

Despite initially refusing to voluntarily pause shipments of the therapy, Sarepta eventually agreed to a “temporary pause” to allow “the necessary time to respond” to the FDA and to finish the “safety labeling supplement process.” 

The reaction was immediate. After the pause was announced, the Parent Project Muscular Dystrophy, a nonprofit organization dedicated to ending the disease by supporting research and advocating for care, criticized the decision. 

“These reports are profoundly upsetting and raise serious concerns for our entire community,” PPMD said in a statement. “Families who have fought tirelessly for access to this therapy, those who have already received it, and those who are in line to receive it are now left with more questions than answers.” 

The organization said the agency’s original approval of Elevidys was important for researchers to continue innovating new therapies for rare genetic diseases. 

“Elevidys was the first gene therapy approved for Duchenne and set an important precedent for the development and evaluation of future therapies,” PPMD said. “Its approval signaled both scientific and regulatory readiness to explore genetic approaches to treating Duchenne and underscored the importance of continued investment and collaboration in the rare disease space.”

Families worried about lack of information about deaths

The FDA’s initial demand to pull Elevidys from the market came after concerns about the therapy and its effectiveness began to percolate among the muscular dystrophy community. Some parents of children with the disease who were receiving the gene therapy were not as optimistic, reportedly raising concerns about the patient deaths. The FDA would later determine that those deaths were not connected to the therapy, informing its recent decision to reverse course. 

“I got worried about the silence that was around these deaths,” Christine McSherry, who filed a citizen’s petition to the FDA about the drug, told Bloomberg News. "All these families are looking for answers and no one is giving answers.” McSherry is the founder of the Jett Foundation, which is dedicated to the funding of muscular dystrophy research and named after her son, who passed away from the condition. 

Sarepta’s Elevidys was originally approved by the FDA and access was expanded despite the initial clinical trials failing to meet their main goal and concerns among FDA reviewers about the therapy’s effectiveness. The company had used the FDA’s "fast-track" approval process, which permits drugmakers to submit less data to get approval and promise more comprehensive evidence after the drug was already in use. The Biden-era director overruled concerns from his own FDA staff to maintain agency approval of Elevidys, Bloomberg News reported. 

Sarepta did not respond to a request for comment from Just the News. 

The company insists through media relations that though the initial trials show Elevidys treatment failed to elicit statistically significant improvement for a key effectiveness measurement, the recipients did improve on a slew of secondary measurements. The company also says that evidence from subsequent studies indicates that patients may experience greater benefits over time after the therapy. 

Ambulatory patients will receive shipments again

After the FDA’s decision, the therapy will be made available again to ambulatory patients, a population with no recorded deaths after receiving the therapy. But, those patients who can no longer walk will still be prevented from receiving the therapy. 

Nevertheless, Sarepta said it was satisfied by the FDA’s quick action. “We are very pleased that the FDA chose to rapidly and comprehensively complete that review and to recommend that we remove our voluntary pause and resume shipment of ELEVIDYS for ambulatory patients,” the company’s CEO Doug Ingram said in a statement. “The FDA’s swift review evinces a commitment to the Duchenne population, a commitment shared by Sarepta.” 

Prasad vindicated

The move is likely to alleviate criticisms pointed at the FDA official responsible for the pause, Dr. Vinay Prasad, who heads the Center for Biologics Evaluation and Research at the FDA. After the decision threatened to interrupt treatments for muscular dystrophy patients and roiled the biotech markets, he faced blistering assessments from conservatives over the policy, who pointed out his long history of support for progressive Democrats and heavier regulation in medicine and the pharmaceutical approval process.

“Vinay Prasad Is a Bernie Sanders Acolyte in MAHA Drag,” an op-ed in the pages of The Wall Street Journal declared, using the common acronym for the “Make America Healthy Again” movement that emerged during the 2024 presidential campaign. 

“I am a progressive. I favor a strong regulatory state (please read my books and you will see!), and I support Bernie Sanders,” Prasad wrote in January 2023 in a post to his online blog. 

Prasad has also long been skeptical of new treatments that have less supporting evidence for their effectiveness, even if they could be the last chance for someone with a debilitating disease. He has also argued for a policy of rationing—where the government would direct spending away from cancer drugs because their cumulative benefits do not outweigh spending on more “cost-effective” healthcare that targets heart health, basic surgeries, and antibiotics. 

Prasad, as the Chair of Oregon’s Health Technology Assessment Subcommittee, also led an effort to block Medicaid funding for genomic testing that might inform cancer treatments. “Genome-informed cancer medicine has some benefit, but is mostly hype,” he wrote, per RealClear Health. 

This generated a sharp reaction from oncologists. “This is potentially an assault on the treatment of underrepresented populations,” said Dr. John Stewart of the University of Illinois Cancer Center. The proposed policy was eventually withdrawn.

The Department of Health and Human Services, which oversees the FDA, did not respond to a request for comment from Just the News about the decision to reverse its demands and whether the speed of its review had anything to do with public pressure.