Ascendis Pharma will request to meet with the U.S. Food and Drug Administration (FDA) in an effort to gain approval of TransCon PTH, its experimental hormone replacement therapy for hypoparathyroidism. “Based on our clinical experience with TransCon PTH to date, we have seen the improvement in the…

Online enrollment of an expanded access program for TransCon PTH, an experimental hormone replacement therapy for hypoparathyroidism, is now open to physicians who want to access the therapy for their patients, the treatment’s developer Ascendis Pharma has announced. Ascendis announced last month that the U.S. Food…

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 U.S. Food and Drug Administration (FDA) has rejected Takeda Pharmaceutical’s proposed changes to address the risk of rubber fragments detaching into Natpara’s cartridge that led to the therapy’s U.S. recall in 2019. This treatment for hypoparathyroidism involves puncturing the medication’s cartridge rubber septum — a disk…

The U.S. Food and Drug Administration has granted fast track designation to encaleret, an investigational therapy for autosomal dominant hypocalcemia type 1, called ADH1, a rare genetic form of hypoparathyroidism. Encaleret is designed to help normalize the calcium levels of ADH1 patients by blocking the calcium-sensing receptor…

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…

There currently is no cure for hypoparathyroidism, and moreover, few therapy options are available — particularly for patients for whom the standard treatment of calcium and active vitamin D is not very effective in controlling the disease. Participating in clinical trials can help researchers develop new and better treatments.

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, the FDA these days relies more and more on patients to…

Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…