BridgeBio Pharma’s Encaleret Gains Fast-Track Review for ADH1

BridgeBio Pharma has secured FDA Fast-Track designation for encaleret as a treatment for autosomal dominant hypocalcemia (ADH1), a condition in which there are lower-than-average levels of calcium in the blood.

Encaleret works by correcting defects in the calcium-sensing receptor gene that affects calcium-level regulation in the parathyroid glands, kidneys and bones.

The designation was supported by results from a phase 2 trial in which the investigational drug normalized blood and urine calcium in six ADH1 participants over five days.

Encalerat could become the first approved therapy specifically indicated for treating ADH1, which can cause a variety of symptoms, including muscle cramps, confusion, and tingling in the lips and fingers.