BridgeBio Pharma Receives FDA Fast Track Designation for Encaleret for the Treatment of Autosomal Dominant Hypocalcemia Type 1

BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company founded to discover, create, test and deliver meaningful medicines for patients with genetic diseases and cancers with clear genetic drivers, today announced that the U.S. Food and Drug Administration (FDA) granted Fast Track designation for encaleret for the treatment of autosomal dominant hypocalcemia (ADH1). The FDA Fast Track designation program is designed to facilitate the development and to expedite the review of new therapies hoping to treat or prevent serious conditions and fill an unmet medical need. BridgeBio announced the Fast Track designation on World Hypoparathyroidism Awareness Day, an annual global awareness and education event designed to spotlight and support people living with ADH1 and other types of hypoparathyroidism.

ADH1 is a rare, genetic form of hypoparathyroidism caused by pathogenic variants in the calcium-sensing receptor gene (CASR). It is estimated that about 12,000 individuals in the United States carry such variants in CASR.¹ The calcium-sensing receptor gene encodes the receptor, CaSR, which senses the level of calcium in the body and regulates the amount of calcium in the blood through its effects on the parathyroid glands, the kidney, and bone. Gain-of-function variants in CASR result in sensing low calcium as normal. As a result, patients with ADH1 have low blood calcium (hypocalcemia), low or low-normal parathyroid hormone levels, and excess urinary excretion of calcium (hypercalciuria). Hypocalcemia can cause severe muscle cramping and seizures, while hypercalciuria can lead to impaired kidney function and kidney stone formation. The current standard-of-care for ADH1 patients consists of oral calcium supplements in excess of typical dietary requirements for people with normal CaSR function, and activated vitamin D, which can partially correct hypocalcemia but typically worsens both hypoparathyroidism and hypercalciuria.

“Balancing near-normal blood and avoiding excess urinary calcium is a daily struggle for patients with ADH1 as the range of symptoms produced by the highs and lows of the condition cannot adequately be addressed by current standard-of-care,” said Jonathan Fox, M.D., Ph.D., Chief Medical Officer of the cardio-renal affiliates at BridgeBio, including Calcilytix, which is focused on developing encaleret. “With respect to the mechanism of disease, which is driven by gain-of-function variants in CASR, encaleret, as an allosteric negative modulator of the receptor’s calcium sensing activity, has the potential to correct the disease mechanism at its source. It is encouraging to receive Fast Track designation from the FDA as it recognizes the seriousness of ADH1 and the potential for encaleret to address this profound unmet medical need.”

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