You’ve no doubt seen many mentions of “Sarepta” and “Elevidys” in media headlines as of late. Last week, the situation culminated in the EMA declining to recommend the therapy for approval in Europe, and Vinay Prasad, head of the FDA’s Center for Biologics Evaluation and Research, resigning from his post – allegedly because of controversy involving Sarepta.
How did we end up here?
Breakthrough approval in 2023 and 2024
Elevidys is an AAV-vector based gene therapy for Duchenne muscular dystrophy (DMD). Approved by the FDA in 2023 for ambulatory patients aged 4 and 5 years, it was the first gene therapy for the disease and considered groundbreaking. It became one of the most expensive drugs in the world – with a price tag of over $3 million for a one-time dose. In 2024, its indication was expanded to include non-ambulatory patients aged 4 years and over.
As is the case with any therapy, true success can only be measured in the real world. Gene therapies are often hyped as miracle cures, but they can have serious side effects. In particular, therapies that use AAV vectors are known to potentially cause liver toxicity.
2025: patient deaths and investigations
In March 2025, a teenage non‑ambulatory boy with DMD died less than two months after receiving Elevidys. The cause was acute liver failure. A second teenage non‑ambulatory patient also died from acute liver failure in June.
Sarepta took action quickly by suspending shipments of the therapy for non-ambulatory patients “while an enhanced immunosuppressive regimen is evaluated, discussed with regulatory bodies, and put in place.”
On June 24, the FDA announced that it was investigating. The agency statement noted that although the prescribing information mentioned the risk of liver toxicity, it did not include warnings about liver failure and death.
July: a third death and a flurry of action
In July, media reports began to surface claiming that an 8‑year‑old boy in Brazil had also died in June after taking Elevidys. However, it was also reported that the death may have been unrelated.
Sarepta announced a label update to Elevidys on July 16: a black box warning was to be added for acute liver injury and acute liver failure. The company was also planning to present a new protocol to the FDA that could open the way forward for dosing to be re-established in non-ambulatory patients.
Shortly after, reports of another death began circulating in the media, prompting Sarepta to disclose that a 51-year old, non-ambulatory limb-girdle muscular dystrophy patient had died after receiving the experimental gene therapy, SRP-9004, which uses the same AAVrh74 delivery vector as Elevidys.
On July 18, the FDA placed a clinical hold on Sarepta’s clinical trials for limb girdle muscular dystrophy and revoked the company’s platform technology designation for the AAV vector AAVrh74.
The agency also asked Sarepta to voluntarily stop all Elevidys shipments for both ambulatory and non-ambulatory patients – even though the reported deaths had only been in non-ambulatory individuals. “Protecting patient safety is our highest priority, and the FDA will not allow products whose harms are greater than benefits,” Prasad said in a July 18 statement. “The FDA will halt any clinical trial of an investigational product if clinical trial participants would be exposed to an unreasonable and significant risk of illness or injury.”
Sarepta initially refused to comply with the suspension of shipments. The company acknowledged that pausing shipments for non-ambulatory patients was the right decision, but argued: “Based on our comprehensive scientific interpretation of the data, which shows no new or changed safety signals in the ambulant patient population, we will continue to ship ELEVIDYS to the ambulant population.”
The company pivoted on July 21 – agreeing to a voluntary pause to keep the peace with the FDA. A statement from the company Sarepta said: “It is important for the patients we serve that Sarepta maintains a productive and positive working relationship with FDA, and it became obvious that maintaining that productive working relationship required this temporary suspension while we address any questions that FDA may have and complete the ELEVIDYS label supplement process.”
Meanwhile in Europe
There was also trouble in Europe for Sarepta and its partner Roche. On July 25, the EMA’s Committee for Medicinal Products for Human Use (CHMP) declined to recommend Elevidys for approval in the EU. Roche – which has a licensing and commercialization deal with Sarepta for the therapy – says it will work with the EMA to find a path forward.
A statement from Roche says: “The CHMP opinion is based on data from the largest and broadest gene therapy clinical programme in DMD to date, including results from the pivotal Phase III EMBARK study that showed treatment with Elevidys provided sustained stabilisation or slowing of disease progression, and a consistent and manageable safety profile in ambulatory patients. To date, more than 900 individuals with DMD, 760 of whom are ambulatory, have been treated with Elevidys in clinical and real-world settings.”
Flipflopping at the FDA
Moving back to the US – there was still more to come. On July 25, the FDA launched a formal investigation into the death of the 8-year-old boy. After seeing the press release, Sarepta released its own statement saying that the death was unrelated to the treatment and that all procedures had been followed in reporting the death to the relevant authorities.
On July 28, the FDA decided that the death of the 8-year-old was, indeed, unrelated to Elevidys and that the voluntarily pause on Elevidys shipments for ambulatory patients could be lifted.
Two days later, Prasad resigned from his role at the FDA – having spent less than 3 months in the role. No details about the departure were given, but it’s been widely reported that he “did not want to be a distraction to the great work of the FDA in the Trump administration and has decided to return to California and spend more time with his family.”
There are also other rumblings in media reports – including that Prasad was accused of being a “progressive leftist saboteur undermining President Trump’s FDA.”
Politics aside, the public exchange between the FDA and Sarepta over Elevidys was far from ideal.
Understanding DMD
DMD affects around 1 in 5000 boys
It is caused by a gene mutation that disrupts the production of functional dystrophin protein and leads to muscle weakness
DMD patients eventually lose the ability to walk, along with upper limb, lung, and cardiac function
Life expectancy is 28 years
Elevidys remains the only approved gene therapy for the disease
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