FDA Advisory on Barth Syndrome Drug Accelerated Approval

In a tumultuous journey marked by setbacks and delays, Stealth’s drug for Barth syndrome faced initial denial by the FDA, only to later gain momentum with a favorable vote from an advisory committee. Despite this positive development, subsequent missed deadlines by the FDA have raised concerns about the impact of job cuts on the approval process. The agency’s handling of this case highlights the complexities and challenges of navigating the regulatory pathway for rare disease treatments, underscoring the importance of efficient communication and decision-making in drug approval timelines.