BridgeBio's Double Victory: Phase 3 Successes Pave Way for FDA Submissions

BridgeBio Pharma, a Bay Area-based biopharmaceutical company, has reported significant advancements in its drug development pipeline, marking a pivotal moment for the company and potential new treatment options for patients with rare diseases.

Encaleret Triumphs in Phase 3 Trial for Calcium Deficiency

BridgeBio's encaleret, an oral small molecule designed to treat autosomal dominant hypocalcemia type 1 (ADH1), has demonstrated remarkable efficacy in its global phase 3 Calibrate trial. The study showed that 34 out of 45 patients treated with encaleret achieved healthy blood and urine calcium levels, compared to only two patients under standard-of-care treatment.

ADH1, a rare genetic disorder, causes disrupted calcium sensing, leading to abnormally low blood calcium and parathyroid hormone levels, coupled with excessive calcium in urine. Dr. Michael Mannstadt, chief of the endocrine unit at Massachusetts General Hospital, highlighted the potential of encaleret to become "an important new standard of care for this patient community."

The success of encaleret extends beyond ADH1, with plans to initiate registrational studies in chronic hypoparathyroidism and pediatric ADH1 in 2026. This expansion follows promising results from a small phase 2 study in post-surgical hypoparathyroidism patients, conducted in collaboration with the National Institutes of Health's National Institute of Dental and Craniofacial Research.

Muscular Dystrophy Candidate BBP-418 Hits Key Endpoints

Adding to BridgeBio's recent successes, the company announced that its limb-girdle muscular dystrophy candidate, BBP-418, met all primary and secondary endpoints in an interim phase 3 analysis. This achievement, coming just days before the encaleret results, underscores BridgeBio's growing strength in the rare disease space.

Strategic Plans and Future Outlook

BridgeBio is positioning itself for significant regulatory milestones in the coming years. The company plans to submit FDA approval applications for both encaleret and BBP-418 in the first half of 2026. Following the FDA submissions, BridgeBio intends to seek approval from the European Medicines Agency for encaleret.

The company's pipeline continues to show promise beyond these two candidates. BridgeBio's dwarfism treatment, infigratinib, is set to complete its phase 3 trial in achondroplasia by the end of the year. Notably, infigratinib has already outperformed BioMarin's approved therapy, Voxzogo, in a phase 2 achondroplasia trial.

These developments come at a crucial time for BridgeBio, following the earlier setback and subsequent comeback of its cardiovascular asset, acoramidis. After initial disappointing results in late 2021, acoramidis rebounded to secure FDA approval for transthyretin amyloid cardiomyopathy in November 2024.

As BridgeBio advances its diverse portfolio of rare disease treatments, the pharmaceutical industry watches closely. The company's recent successes not only validate its research approach but also offer hope to patients with limited treatment options for these challenging genetic conditions.