Disc Medicine’s bitopertin—rejected by the FDA in February—significantly reduced levels of a key disease biomarker and significantly increased the time patients with erythropoietic protoporphyria were able to spend in the sun and artificial light.

Original title: “Disc’s rebuffed rare disease drug sets up FDA redo after ‘clean’ Phase 3 sweep”

perioperative management of a child with x linked erythropoietic protoporphyria undergoing dental surgery in preparation for liver transplantation

Original title: “Perioperative management of a child with X-linked erythropoietic protoporphyria undergoing dental surgery in preparation for liver transplantation.”

new and currently investigated pharmacotherapies for the erythropoietic protoporphyrias spotlight on dersimelagon and bitopertin

Original title: “New and currently investigated pharmacotherapies for the erythropoietic protoporphyrias: spotlight on dersimelagon and bitopertin.”

The FDA in October 2025 granted bitopertin its Commissioner’s National Priority Voucher but after a shortened review time has decided the data did not support regulatory approval for treating erythropoietic protoporphyria.

Original title: “FDA Rejects Disc’s Rare Blood Disease Drug Amid Reports of Prasad’s Skepticism”