PALO ALTO, Calif. — BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage, multi-product biopharmaceutical company focused on developing medicines for genetic conditions, today announced the submission of its New Drug Application (NDA) to the FDA for encaleret as a potential targeted treatment for autosomal dominant hypocalcemia type 1 (ADH1).
CALIBRATE, the 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. The topline results can be found here. Additional positive results were presented at the European Congress of Endocrinology (ECE) 2026 in an oral presentation, with data showing comprehensive normalization of mineral homeostasis.
Primary results of the study include:
- 76% of participants randomized to encaleret achieved both target serum calcium and urine calcium levels compared to 4.4% of those same individuals while on standard of care (p<0.0001)
- Rapid and sustained improvements in calcium metabolism for participants randomized to encaleret, with increases in serum calcium observed by Day 3 and reductions in urine calcium by Week 3, maintained through Week 24
- At Week 24, more participants randomized to encaleret achieved both target serum and urine calcium levels than participants who remained on standard of care (76% on encaleret vs. 19% on standard of care; p < 0.0001)
- Encaleret was observed to restore endogenous parathyroid hormone (91.1% on encaleret vs. 0% of participants on standard of care at Week 24)
- Favorable safety and tolerability profile, with no discontinuations in the encaleret arm and low rates of serious adverse events with frequency similar between treatment arms
“These Phase 3 findings are a landmark moment for the autosomal dominant hypocalcemia type 1 community,” said Filomena Cetani, M.D., Ph.D. of the University of Pisa, Italy. “Encaleret not only has the potential to become the first-ever approved therapy for this rare disease, but it does so by addressing the root cause, restoring normal calcium regulation and lowering the risk of renal complications that individuals on current treatment face every day. Together, these findings exemplify what a first-in-class therapy should look like.”
BridgeBio anticipates a U.S. launch in early 2027. Nearly 2,000 individuals have been diagnosed in the U.S. with autosomal dominant hypocalcemia (ADH) 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.
BridgeBio is currently enrolling CALIBRATE-PEDS (NCT07080385), a global registrational Phase 2/3 study of encaleret in pediatric ADH1. The Company also plans to initiate RECLAIM-HP, a global Phase 3 study of encaleret in chronic hypoparathyroidism in Summer 2026, building on the Phase 2 proof-of-concept findings of PTH-independent effects of encaleret on renal calcium handling and expanding the potential applications of encaleret beyond ADH1.
About Encaleret
Encaleret is an investigational, orally administered small molecule under investigation to treat ADH1 and chronic hypoparathyroidism, that is designed to selectively negatively modulate the calcium sensing receptor. Encaleret has been granted Fast Track Designation by the U.S. FDA and Orphan Drug Designation in the U.S., European Union, and Japan.
About BridgeBio
BridgeBio exists to develop transformative medicines for genetic conditions. Millions of people worldwide living with genetic conditions lack treatment options, often because drug development for small patient populations can be commercially challenging. We aim to bridge the gap between advancements in genetic science and meaningful medicines for underserved patient populations. Our decentralized, hub-and-spoke model is designed for speed, precision, and scalability. Autonomous and empowered teams focus on individual conditions, while a central hub provides the clinical, regulatory, and commercial capabilities needed to bring innovation to market. For more information, visit bridgebio.com and follow us on LinkedIn, X, Facebook, Instagram, YouTube, and TikTok.
BridgeBio Media Contact
Bubba Murarka, Executive Vice President
[email protected]
(650)-789-8220
BridgeBio Investor Contact
Chinmay Shukla, Senior Vice President, Strategic Finance
[email protected]