A pill brings the shade down: sunlight stops hurting in a late-stage trial

For people with erythropoietic protoporphyria, a rare blood disorder, ordinary daylight can mean burning pain — many spend their lives in shade, covered up, watching summer through a window. Disc Medicine's pill bitopertin has now passed a late-stage trial: among 183 patients aged 12 and older, it cut the build-up of the light-sensitive substance protoporphyrin IX in the blood by 41.4% compared with placebo, met every main and secondary goal, and was generally well tolerated with no new safety concerns. The US Food and Drug Administration had earlier declined to approve the drug, asking for a clearer link between lowering the substance and tolerating sunlight; this trial was built to answer exactly that. The only approved treatment today is an implant under the skin — a working pill would be a first.

SDG 3 · Good health and well-being — Mechanism: tech innovation