Encaleret Sets New Standard with 76% of Patients Reaching Calcium Targets
BridgeBio Pharma has announced landmark results from its global Phase 3 CALIBRATE trial evaluating encaleret for autosomal dominant hypocalcemia type 1 (ADH1). An impressive 76% of patients treated with encaleret achieved both blood and urine calcium targets at Week 24—a figure that dramatically outpaces the mere 4% seen on conventional therapy (calcium supplements and vitamin D). These results mark a pivotal moment in the treatment landscape for this rare genetic disease.
Major Efficacy Gains and Improved PTH Levels Highlighted
Notably, encaleret did more than stabilize calcium levels. In a key secondary measure, 91% of participants had endogenous parathyroid hormone (PTH) levels above the reference range’s lower limit by Week 24, compared to only 7% with conventional therapy. This suggests that encaleret may address underlying disease mechanisms—rather than simply patching symptoms.
| Endpoint | Encaleret | Conventional Therapy | P-Value |
|---|---|---|---|
| Patients with Both Calcium Targets Met | 76% (34/45) | 4% (2/45) | <0.0001 |
| Patients with Intact PTH Above Lower Limit | 91% | 7% | <0.0001 |
| Serum Calcium in Target Range at Week 20 | 98% (44/45) | 33% (7/21) | - |
Safety Profile Remains Strong, with Nearly All Patients Continuing
Encaleret demonstrated a favorable safety profile. No patients discontinued due to drug-related events, and the majority elected to continue in the study's long-term extension phase—reflecting both physician and patient confidence in the therapy’s tolerability. Reported side effects were consistent with known ADH1 biology and the mechanism of the drug.
Forward Plans: Regulatory Submissions and Expanded Indications Ahead
Based on these results, BridgeBio plans to submit a New Drug Application (NDA) to the FDA in the first half of 2026. A registrational trial for encaleret in pediatric ADH1 and another for adults with chronic hypoparathyroidism are slated for 2026. The strong results may pave the way for encaleret to become the first targeted therapy approved for ADH1, representing a significant leap forward in genetic disease management.
Takeaway: A Transformative Option for a Difficult Disease
While regulatory hurdles remain, encaleret’s Phase 3 performance positions it as a potential new standard for patients struggling with ADH1. Its ability to normalize both serum and urine calcium, restore PTH, and maintain a strong safety profile are encouraging signs for clinicians and families awaiting better solutions.
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