Second Child's Death Raises Alarms Over Gene Therapy Approval Process by FDA

Gene Therapy Under Fire Following Child Fatalities

The recent deaths of two children after receiving Elevidys, an experimental gene therapy intended for Duchenne muscular dystrophy (DMD), has triggered widespread alarm regarding the oversight process of the Food and Drug Administration (FDA). On June 24, 2025, Florida attorney Sean C. Domnick publicly criticized the FDA for what he describes as regulatory shortcuts that jeopardize patient safety. Both fatalities involved a vulnerable population that had not been adequately included in clinical trials, leading to questions about the ethical implications of rapid drug approvals.

Elevidys, developed by Sarepta Therapeutics, was initially fast-tracked for use in ambulatory children aged four and older, aimed at improving the quality of life for those diagnosed with DMD. Yet, following the drug's approval, Sarepta began administering it to non-ambulatory patients, which raises significant medical concerns given that these children present vastly different health risks. The tragic outcome of two deaths has prompted outrage and scrutiny from the medical community, demanding a thorough investigation into the circumstances surrounding these incidents.

In Domnick's critical assessment, he stated, "Medical innovation is essential—but so is humility. When we lower the bar for evidence, vulnerable patients pay the price." His statement underscores the dire need for a refined and more cautious approach towards gene therapies that target children—particularly those who fall outside the originally tested demographics.

After the second child’s death, Sarepta paused the therapy's use in non-ambulatory patients amidst an internal review. Preliminary investigations suggest potential liver failure, a known risk with Elevidys, may have contributed to the fatalities despite clear warning labels about this danger. This troubling revelation has led to growing calls within the medical community for reform and enhanced scrutiny of FDA approval processes. Notably, even FDA officials expressed internal skepticism about the drug's approval before these incidents transpired, further illuminating critical gaps in regulatory oversight.

Domnick argues that these deaths are indicative of broader systemic flaws within the FDA's fast-track approval processes, asserting that families often remain uninformed about the full extent of risks associated with unproven treatments. He emphasizes, "These are not isolated incidents. They're a warning. We need to reexamine how therapies are being pushed through before we fully understand their consequences—especially for children."

As families continue to seek innovative solutions for rare genetic disorders, and biotech companies race to deliver breakthroughs, the equilibrium between expediency and safety has never been more crucial. Domnick and his firm, Rafferty Domnick Cunningham Yaffa, are advocating for greater transparency and stronger informed consent practices in clinical trials, defending the rights of families and patients who might become collateral damage in the biotech industry's rush for innovation.

While the devastation resulted from these tragic fatalities is immeasurable, it serves as a pivotal moment for the FDA and the biotech community to reconsider existing practices and protocols to ensure that patient safety remains at the forefront. In the face of medical progress, the call for accountability and strict adherence to safety measures echoes louder than ever, inviting critical discourse on how to safeguard the health of the most vulnerable.

In Conclusion

As the scrutiny grows, many in the healthcare profession urges for reformed guidelines that emphasize safety and thorough research, particularly for new treatments involving children. The time for reform is now, as the price of negligence can be paid in lives lost rather than lives saved. Keeping the well-being of patients, especially children, in mind must be paramount in the race for medical advancements.