Ascendis Pharma A/S Receives Orphan Drug Designation for TransCon hGH as Treatment for Growth Hormone Deficiency in the United States

COPENHAGEN, Denmark, April 15, 2020 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to address unmet medical needs, today announced that the United States (U.S.) Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to TransCon hGH (lonapegsomatropin), as a treatment for growth hormone deficiency (GHD).

TransCon hGH is an investigational long-acting prodrug of somatropin (human growth hormone or hGH) that is being developed as a once-weekly treatment for GHD. TransCon hGH is designed to maintain the same mode of action as daily hGH therapies by releasing the same growth hormone molecule, somatropin. Currently, there is no approved long-acting growth hormone treatment on the market in the U.S. or Europe. TransCon hGH received Orphan Designation (OD) for the treatment of GHD in Europe from the European Commission (EC) in October 2019.

“TransCon hGH has now been granted orphan designation in both the U.S. and Europe, which we believe is an important acknowledgement of the global need for a long-acting therapy to address GHD and overall endocrine health,” said Dana Pizzuti, M.D., Ascendis Pharma’s Senior Vice President of Development Operations. “As the only long-acting growth hormone product in development that delivers somatropin, we believe TransCon hGH has significant potential to improve patients’ lives. We remain on track to file our marketing applications for TransCon hGH in the U.S. and Europe, as planned, in the second and fourth quarters of this year, respectively.”

The FDA grants orphan designation 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, and potentially may be safer or more effective than already approved products. Orphan designation 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 orphan 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.