All articles tagged with #disc medicine
The FDA rejected Disc Medicine's bitopertin for porphyria, citing uncertainties that the biomarker used as the efficacy endpoint may not translate to real patient benefit, marking the first setback for a drug in Commissioner Makary's fast-track review program and sending Disc's shares lower.
The FDA declined Disc Medicine’s bitopertin for erythropoietic protoporphyria, citing that the phase 2 trials used a surrogate endpoint (a ~40% drop in PPIX) whose link to meaningful clinical benefit—sun tolerance—remains unproven. The company plans to press on with the APOLLO phase 3 trial, which uses sun-exposure outcomes, while the broader regulatory environment and the CNPV priority voucher program continue to draw scrutiny.
Reuters reports the U.S. FDA has delayed reviews of two drugs selected for the National Priority Voucher fast-track program after agency scientists flagged safety and efficacy concerns, including a treatment-related death linked to one drug. Disc Medicine's experimental therapy for a rare blood disorder was pushed back due to questions about trial data and abuse risk, while Sanofi's Tzield for late-stage type 1 diabetes was delayed over adverse events, including seizures and a death. Two other program drugs—Boehringer Ingelheim's zongertinib and Lilly's weight-loss pill—were also delayed by weeks. The delays underscore the program’s aim to accelerate reviews only when warranted by robust safety/efficacy data, amid ongoing scrutiny of how political factors may influence drug decisions.