FDA Advisory Committee Skeptical of Stealth BioTherapeutics' Barth Syndrome Drug Application

Key Insights

• Stealth BioTherapeutics faced scrutiny from the FDA's Cardiovascular and Renal Drugs Advisory Committee regarding its drug application for Barth syndrome.
• Committee members questioned the feasibility of conducting a new randomized trial, citing challenges like potential functional unblinding.
• Despite concerns, the committee acknowledged that inherent difficulties could compromise the integrity of a new study.

Stealth BioTherapeutics encountered significant skepticism from the FDA's Cardiovascular and Renal Drugs Advisory Committee concerning its application for a new drug intended to treat Barth syndrome. The committee members repeatedly challenged Stealth's assertion that a new randomized trial in Barth syndrome was not possible.

Concerns Over Trial Feasibility

The advisory committee voiced concerns about the feasibility of conducting a new randomized trial. Members pointed out potential functional unblinding and other challenges that could compromise the integrity of a new study. Functional unblinding occurs when patients or investigators can deduce the treatment assignment based on observed effects, potentially biasing the results.

Impact on Approval Prospects

The skepticism from the advisory committee introduces uncertainty regarding the potential approval of Stealth BioTherapeutics' drug. While acknowledging the difficulties inherent in studying rare conditions like Barth syndrome, the committee's reservations highlight the need for robust evidence to support drug efficacy and safety. The FDA will consider the advisory committee's feedback when making its final decision on the drug application.