MBX 2109, an experimental hormone replacement therapy that MBX Biosciences is developing for hypoparathyroidism, has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). The FDA awards this designation to therapies intended to treat rare diseases, defined as those that affect less than 200,000 people…

The European Commission has granted orphan drug designation to AZP-3601, Amolyt Pharma’s investigational therapy to treat hypoparathyroidism. Orphan designation is given to promising treatment candidates for life-threatening and debilitating rare diseases, those affecting not more than five in every 10,000 people in the European Union. The designation will…

Entera Bio is developing a better formulation of EB612, its add-on therapy for maintaining normal blood calcium levels in people with hypoparathyroidism, and expects to launch a clinical trial next year. The biotechnology company shared its plans while announcing the publication of an earlier Phase 2a study of…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AZP-3601, an investigational therapy for hypoparathyroidism by Amolyt Pharma. The designation is given to treatment candidates with the potential to be safe and effective in rare diseases, defined in the U.S as those affecting fewer than…

The European Commission (EC) has granted orphan drug designation to Ascendis Pharma’s experimental hormone replacement therapy TransCon PTH, currently in development for the treatment of hypoparathyroidism. Medicines…

The U.S. Food and Drug Administration has granted orphan drug designation to TransCon PTH, an investigative therapy by Ascendis Pharma to treat hypoparathyroidism. Hypoparathyroidism is a rare endocrine disorder characterized by insufficient levels of parathyroid hormone (PTH), resulting in low calcium and elevated phosphate levels in the blood. TransCon…