The US Food and Drug Administration (FDA) is facing intense scrutiny over its approval process for rare disease treatments. The agency’s decision to grant accelerated approval for Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy, a genetically devastating disorder, has sparked controversy. The treatment, which costs $3.2m per patient, was initially approved despite concerns about limited clinical data. However, three patients on the treatment have since died from acute liver failure, prompting the FDA to ask Sarepta to suspend distribution.
The incident highlights the politicization of rare disease oversight at the FDA. The agency’s director at the time, Peter Marks, faced criticism for his decision. The controversy has also led to a significant decline in Sarepta’s stock value. This incident serves as a reminder of the delicate balance between expediting new treatments and ensuring patient safety.
As the FDA navigates this complex issue, it must also contend with growing tensions within its own ranks and across party lines. The agency’s oversight of rare disease treatments has become increasingly politicized, with implications for the treatment landscape and patients’ lives.
Source: https://www.economist.com/united-states/2025/08/10/americas-drug-regulator-is-in-turmoil