BridgeBio Pharma Inc. (BBIO), a commercial-stage biopharmaceutical company, on Wednesday announced acceptance of the new drug application (NDA) for encaleret in treating autosomal dominant hypocalcemia type 1 by the U.S. Food and Drug Administration (FDA).
Autosomal dominant hypocalcemia type 1 (ADH1) is a form of hyperparathyroidism, that arises due to variations in the CASR gene that controls the calcium sensing receptor. This receptor controls blood calcium levels by regulating parathyroid hormone secretion and calcium reabsorption in the kidneys.
Symptoms of hypocalcemia may include severe muscle cramps, muscle spasms (tetany), a burning or prickling sensation in the hands or feet (paresthesia), brain fog, fatigue, and seizures. From October 2023 to April 2026, about 21,000 people were diagnosed with ADH1 in the U.S. alone.
Encaleret is an investigational, orally administered small molecule developed to treat ADH1 and chronic hypoparathyroidism by negatively modulating the calcium sensing receptor. The drug has been granted Fast Track Designation in the U.S., European Union, and Japan.
The NDA was supported by results of the Phase 3 CALIBRATE trial, which met all its primary and key secondary endpoints to prove that encaleret addressed the underlying genetic cause of ADH1. Data indicated that treated the therapy showed simultaneous restoration of blood and urine calcium, as well as restoration of physiologic parathyroid hormone (PTH) production.
The Phase 3 CALIBRATE-PEDS trial is currently evaluating the efficacy of encaleret in pediatric patients of ADH1.
The FDA has set a prescription drug user fee act (PDUFA) target action date on May 8, 2027, at which point the regulatory body may release its verdict on encaleret. No further advisory committee meetings to discuss the application are scheduled at this point.
The company also plans to submit a marketing authorization application (MAA) to the European Medicines Agency (EMA) for encaleret in the second half of 2026.BBIO is currently trading at $81.56, down 1.08%.
For comments and feedback contact: editorial@rttnews.com
Economic News
What parts of the world are seeing the best (and worst) economic performances lately? Click here to check out our Econ Scorecard and find out! See up-to-the-moment rankings for the best and worst performers in GDP, unemployment rate, inflation and much more.
September 04, 2026 15:08 ET The job report and foreign trade data were the main economic news from the U.S. in the first week of September. Elsewhere in North America, the Canadian central bank announced its latest policy decision. Inflation data from Eurozone was the highlight in Europe. The latest interest rate decision by the Reserve Bank of New Zealand drew attention in Asia.