BridgeBio wins FDA filing acceptance for rare calcium disorder drug

BridgeBio wins FDA filing acceptance for rare calcium disorder drug

BridgeBio Pharma (BBIO) said the US Food and Drug Administration has accepted its New Drug Application for encaleret, an oral therapy for autosomal dominant hypocalcemia type 1, with a target action date of May 8, 2027.

The FDA indicated it does not currently plan to convene an advisory committee to review the application.

ADH1 is a form of genetic hypoparathyroidism caused by variants in the calcium-sensing receptor gene, a condition that disrupts calcium regulation and can trigger muscle cramps, seizures, and kidney complications. More than 2,100 individuals have been diagnosed with the disease in the US since October 2023, based on claims data.

The NDA is supported by results from CALIBRATE, a Phase 3 trial in which encaleret met all pre-specified primary and key secondary efficacy endpoints. Data were presented at the European Congress of Endocrinology 2026 and the Endocrine Society's 2026 annual meeting.

Encaleret is a small molecule designed to modulate the calcium-sensing receptor and has received Fast Track and Orphan Drug designations from the FDA, as well as orphan status in the European Union and Japan.

BridgeBio said it plans to submit a Marketing Authorization Application to the European Medicines Agency in the second half of 2026. The company is also enrolling CALIBRATE-PEDS, a Phase 2/3 pediatric study, and intends to launch RECLAIM-HP, a Phase 3 trial in chronic hypoparathyroidism, later this summer.