All articles tagged with #bitopertin
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.