Disc setback deepens questions about FDA’s accelerated approval commitment, value of new vouchers

FDA’s complete response letter for Disc Medicine’s bitopertin adds to a growing list of cases in which the agency has declined to grant biomarker-based accelerated approvals of orphan drugs. Like other recent setbacks, the agency’s decision raises questions about whether FDA’s actions are consistent with promises its leaders have made to approve drugs for rare conditions based on a “plausible mechanism.”

The Disc Medicine Inc. (NASDAQ:IRON) CRL also add to scrutiny of FDA’s new commissioner’s national priority voucher (CNPV) program, and of the role of Vinay Prasad, director of the agency’s Center for Biologics Evaluation and Review and FDA’s CSO and CMO. ...