Novel gene therapy kills four, but the FDA approves anyway

Earlier this summer, The U.S. Food and Drug Administration (FDA) marked a significant, yet unusual milestone for patients with Duchenne muscular dystrophy (DMD). After an initial decision to halt Sarepta Therapeutics’ gene therapy for the condition, experts are now encouraging to proceed with the treatment once again.

This decision follows just 10 days after the death of an eight-year-old child, who was treated with Elevidys—the DMD gene therapy developed by Sarepta Therapeutics—but died in late June. Prior to this suspension, there were an additional three deaths tied to this case.

When gene controversy surfaces the world of healthcare and pharmaceuticals, companies need the proper support to overcome the barriers. Clinical research organizations (CROs) are made for instances like this. By providing outsourced research services, CROs help companies manage clinical trials, offering data-driven expertise to streamline the drug development process.

Dinkar Sindhu, CEO of AXIS Clinicals, agrees CROs are the answer to optimal success, providing the flexibility and convenience when drug problems arise.

“The FDA’s updated stance on Elevidys demonstrates the complexity of evaluating gene therapy safety as new data emerges. As regulatory decisions evolve, companies need CROs that are agile, responsive, and grounded in scientific rigor,” he says.

According to Fierce Pharma, the FDA reversed protocols for the administration of Elevidys after determining that the deaths were caused by reasons separate from the gene therapy. Now, Sarepta Therapeutics has resumed shipping the therapy to treatment sites with ambulatory patients, and the drug remains an option for treating DMD.

But despite this quick turnaround, it’s still important for CROs to actively partake in FDA changes in order to meet vulnerable patients where they are at.

Sindhu continues, “An effective CRO must be ready to adjust protocols, address safety concerns in real time, and support sponsors through regulatory engagement. This kind of adaptability is essential for advancing innovative therapies while maintaining patient safety.”

DMD is a severe genetic muscular disorder primarily affecting boys that leads to muscle degeneration and culminates in paralysis, respiratory complications, and premature death. The illness arises from a deficiency of dystrophin, a crucial protein for muscle integrity. While traditional approaches to alleviating DMD involve palliative relief, such as physical therapy and orthopedic interventions, these conventional therapies have not provided a definitive solution for treatment, at least not until the Elevidys drug came to fruition.

In essence, the Elevidys treatment offers hope by delivering a functional copy of the DMD gene, enabling production of dystrophin. The treatment is administered as a single intravenous infusion and requires careful monitoring to protect patients.

But regardless of the notable impacts the gene therapy provides, Elevidys is the only one of its kind for adults and children, and therefore, it’s common for many to be reluctant at first. 

Plus, drug hesitancy is nothing new. As Assist RX explains, nearly 50% of patients do not take their medications as prescribed, due to initial concerns like the potential side effects, cost and access, and a lack of information. 

Of course, the journey to Elevidys is also not without its challenges. It was initially granted approval in 2023 for children aged 4-5, but another decision in 2024 expanded the use of the treatment for individuals aged 4 and older with the disease. Neither approval was supported by favorable FDA scientific reviews or appropriate clinical trial results, but proceeded anyway because an FDA director overruled the agency’s scientific staff.

Looking ahead, while Elevidys is intended to help those affected by DMD, its story reflects the broader challenges and responsibilities of introducing a new therapy to the market. Ultimately, the collaboration between physicians, pharmaceutical companies, and CROs will continue to be pivotal in navigating the controversy head on.

For stakeholders in the clinical research space, the life of DMD patients awaits. Because when all else fails, the power of CROs can make a game-changing difference.