BridgeBio Pharma, Inc., a commercial-stage, multi-product biopharmaceutical company focused on developing medicines for genetic conditions, has announced the FDA has accepted for filing its New Drug Application (NDA) for encaleret for the treatment of individuals living with autosomal dominant hypocalcaemia type 1 (ADH1).
The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of 8 May 2027, and BridgeBio is prepared to launch encaleret upon approval, representing a potential blockbuster opportunity for the Company.
The FDA also notified the company that it is not currently planning to hold an advisory committee meeting to discuss the application.
A phase 3 clinical trial of encaleret in ADH1 successfully achieved all pre-specified primary and key secondary efficacy endpoints, supporting encaleret’s potential as a disease-modifying therapy by targeting the underlying genetic cause of ADH1.
Result data showed simultaneous restoration of blood and urine calcium, as well as restoration of physiologic parathyroid hormone (PTH) production.
As of April 2026, over 2,100 individuals have been diagnosed in the U.S. with autosomal dominant hypocalcaemia since October 2023 based on claims data, suggestive of a growing marketplace and elevated diagnostic suspicion.
The company also intends to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the use of encaleret in ADH1 in the second half of 2026.
