Ascendis Pharma Announces Orphan Drug Designation Granted for TransCon PTH as Treatment for Hypoparathyroidism

Ascendis Pharma (Nasdaq:ASND), a biopharmaceutical company that utilizes its innovative TransCon technology to address significant unmet medical needs, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to TransCon PTH, a long-acting prodrug of parathyroid hormone (PTH) in development for hypoparathyroidism.

As quoted in the press release:

“We are pleased about the ODD for TransCon PTH, which reflects the need for a true PTH replacement therapy that more fully addresses all aspects of hypoparathyroidism,” said Jonathan Leff, M.D., Ascendis Pharma’s Chief Medical Officer. “We have recently completed a phase 1 trial in healthy adults, which reinforced the potential of TransCon PTH to restore PTH to physiological levels for 24 hours per day. We are committed to developing innovative new therapies that improve patients’ lives and look forward to advancing TransCon PTH.”

The FDA grants orphan status to drugs that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The ODD program provides a drug developer with certain benefits and incentives, including a seven-year period of U.S. marketing exclusivity from the date of marketing authorization, waiver of FDA user fees, and tax credits for clinical research.  The granting of an orphan drug designation does not alter the FDA’s regulatory requirements to establish safety and effectiveness of a drug through adequate and well-controlled studies to support approval and commercialization.

Click here to read the full press release.