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.”
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