BridgeBio's encaleret NDA accepted for autosomal dominant hypocalcemia type 1
- Sponsor
- BridgeBio Pharma $BBIO
- Drug
- encaleret
- Indication
- autosomal dominant hypocalcemia type 1 (ADH1)
FDA accepted BridgeBio's New Drug Application for encaleret, an oral calcium-sensing receptor antagonist, in autosomal dominant hypocalcemia type 1 (ADH1), with a PDUFA target action date of May 8, 2027. FDA is not currently planning to hold an advisory committee. If approved, encaleret would become the first therapy specifically indicated for ADH1, a rare genetic form of hypoparathyroidism driven by gain-of-function CASR mutations.
What this means
Encaleret is an oral drug designed to treat a rare genetic disorder that causes low blood calcium levels by blocking a faulty calcium-sensing receptor. Today the FDA accepted BridgeBio's application for review, setting a decision date for later this year. If approved, encaleret could become the first targeted therapy for patients with this specific inherited condition.