FDA clears Ascendis’ once‑weekly achondroplasia drug—new option for rare growth disorder

The FDA approved Ascendis Pharma’s once‑weekly TransCon CNP (navepegritide) for achondroplasia, marking a new injectable option for the genetic short‑limb growth disorder. Ascendis and competitors now face a potential three‑way market for disease‑modifying therapies in pediatric achondroplasia. Achondroplasia is the most common cause of dwarfism caused by FGFR3 mutations; approved therapies aim to modify growth plate signaling to improve height outcomes rather than only treating symptoms.