Eyes on Sarepta & the FDA

Lately, we've kept our Eyes On Sarepta Therapeutics and their discussions with the US FDA regarding their gene therapy Product Elevidys, their pipeline platform. All of this, plus the management shuffles at the FDA itself, has created quite a stir in the industry.

Sarepta Therapeutics has been standing by its licensed treatment for Duchenne muscular dystrophy (DMD) as the debate over the safety of its gene therapies reached a climax. The FDA has been requesting that the business remove the medication from distribution. Citing its own thorough scientific analysis of the data, which indicates no new or altered safety signals in the ambulant patient population, Sarepta stated that it will continue to ship its gene therapy Elevidys to DMD patients who are still able to walk on their own. In order to further the common goal of ensuring patient safety and informed access to care, Sarepta is enthusiastic about ongoing conversations and information exchanges with the FDA.

Sarepta took a stand soon after the FDA requested that the business voluntarily halt all shipments of Elevidys, a medication under investigation by the U.S. pharma regulator after two patients passed away earlier in 2025 after receiving it. Simultaneously, the FDA put Sarepta’s exploratory gene therapy trials in limb-girdle muscular dystrophy on clinical hold and revoked the company’s platform technology designation, which can speed up the FDA review process for subsequent products originating from the same platform. The limb-girdle program had already been weakened by significant layoffs in an attempt to save $400 million per year. A number of Sarepta gene therapy assets are impacted by the new clinical hold, but after the clinical hold is removed, the firm intends to pursue a biologics license application for SRP-9003.

In a phase 1 trial, a 51-year-old man with limb-girdle muscular dystrophy type 2D/R3 was the third patient who died while receiving a Sarepta therapy. Instead of Elevidys, Sarepta’s licensed medicine, he had been given SRP-9004, an experimental gene therapy. The SRP-9004 patient passed away from acute liver failure, just like the previous patients who had taken Elevidys. Before news of the most recent death, Sarepta announced that it was halting the development of SRP-9004 and numerous other gene therapy initiatives. The business emphasised that the investigational gene therapy was designed to treat an alternative disease to Elevidys, was administered with another type of dose, and employed a contrasting manufacturing technique.

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22 July 2025 - Unexpectedly, Sarepta announced that, starting at the end of business on July 22nd, it would stop shipping any of its Duchenne muscular dystrophy gene therapy Elevidys to the U.S. After patient deaths, including two that occurred after using Elevidys, the FDA requested that distribution be suspended. Following Sarepta’s first denial of the FDA’s request, the firm stated that it will use this time to address any information demands and finish revising Elevidys’s safety labelling in accordance with an agreement with the FDA. The FDA and the firm are collaborating to revise the drug’s risk management plan and add a black box warning for acute liver injury to the label.

Elevidys will not be available to Duchenne patients for an extended amount of time due to the marketing suspension. Sarepta had maintained that patients must receive prompt therapy for Duchenne because it is an irreversible, degenerative illness. By adhering to this suspension, Sarepta wants to continue having a fruitful and constructive working relationship with the FDA, despite possible disappointment from individuals in need of the treatment.

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July 28, 2025 - FDA Informs Sarepta That It Recommends That Sarepta Remove Its Pause and Resume Shipments of ELEVIDYS for Ambulatory Individuals With Duchenne Muscular Dystrophy

Key takeaways from the company press release:

• Company commends FDA for swift and comprehensive review of available information.

• Shipments to sites of care will resume imminently.

• Sarepta and FDA will continue dialogue on next steps in the safety labeling process and risk-mitigation approach for non-ambulatory patients.

See the full press release here:

August 13, 2025 - Sarepta provides new Elevidys safety data at a virtual event hosted by the Center for Duchenne Muscular Dystrophy at UCLA. The data show no deaths in over 1,000 ambulatory patients with Duchenne muscular dystrophy treated with the drug.

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In FDA News - Less than two weeks after the White House pressured the FDA’s chief to fire Dr. Vinay Prasad from his numerous agency leadership positions, Makary asked him to return to his CBER position. Following a review of Prasad’s previous comments brought to light by right-wing provocateur Laura Loomer last month, the White House determined the agency could reinstate Prasad. For Makary and HHS Secretary Robert F. Kennedy Jr., who fought Prasad’s termination, his reinstatement represents a personnel victory. Makary urged Prasad to change his mind about leaving the agency in the days following his removal.

The move to rehire Prasad was quickly criticised by Loomer, who called him a progressive and brought up his previous criticism of President Donald Trump. What Prasad’s return means for Sarepta Therapeutics is uncertain. In the days leading up to his dismissal last month, the company clashed with the FDA centre that oversees Elevidys, a therapy for Duchenne muscular dystrophy.

FDA employees alternated between sadness and concern in response to the decision to bring Prasad back, expressing incredulity. Although about 25% of the 3,500 agency workers who were first issued reduction-in-force notices earlier this year have been restored, staff morale is still low despite Makary’s efforts to address a higher-than-normal number of vacancies among scientific reviewers.

As an important ally of Makary, Prasad was instrumental in creating the FDA’s regulatory strategy for the upcoming approval of Covid-19 booster doses. Prasad was also the top medical and scientific officer of the agency prior to his dismissal, although it is uncertain if he will return to that position.

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