A new drug for rare diseases: pozelimab for CHAPLE disease.

Competing Interests: AB reports consulting fees from Kabi, Roche Chugai, GlaxoSmithKline, AbbVie, Novartis, and Boehringer Ingelheim; research grants from Boehringer Ingelheim, ANR, and Horizon Europe outside the scope of this Comment; and is an unpaid co-chair of the French National Network on Rare Autoimmune and Autoinflammatory Diseases. All other authors declare no competing interests.