encaleret

Encaleret is an investigational, orally administered small molecule that acts as a negative allosteric modulator (antagonist) of the calcium-sensing receptor (CaSR). It is being developed primarily for the treatment of autosomal dominant hypocalcemia type 1 (ADH1), a rare genetic disorder caused by gain-of-function mutations in the *CASR* gene. These mutations make CaSR overly sensitive to extracellular calcium, resulting in low blood calcium levels (hypocalcemia), high urinary calcium excretion (hypercalciuria), and abnormally low parathyroid hormone levels. By selectively antagonizing CaSR, encaleret aims to restore normal parathyroid hormone secretion and normalize blood and urine calcium levels. Clinical studies have shown that encaleret can rapidly and sustainably correct hypocalcemia and hypercalciuria in patients with ADH1 without significant safety concerns. The drug has received Orphan Drug designation in both the US and EU[1][3][5][6][8].

Other names

Encaleret Sulfate