Ascendis Seeks FDA Approval of TransCon PTH for Adults

Ascendis Pharma has submitted an application to the U.S. Food and Drug Administration (FDA) seeking approval of its treatment candidate TransCon PTH for adults with hypoparathyroidism, the company announced. “Our data support that TransCon PTH, if approved, could become the first-in-class therapy to address the underlying cause of…

Hormone Replacement Therapy MBX 2109 Named FDA Orphan Drug

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…

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.