Ascendis Pharma announced on April 15, 2020 that the US Food and Drug Administration (FDA) has granted TransCon Human Growth Hormone (lonapegsomatropin) orphan drug designation to treat growth hormone deficiency (GHD).
TransCon Human Growth Hormone is a research long-acting prodrug of growth hormone, which has been developed into a weekly GHD treatment drug. TransCon Human Growth Hormone maintains the same mode of action as daily human growth hormone treatment by releasing the same growth hormone molecules. Currently, there is no approved long-acting growth hormone therapy in the US or European market. TransCon Human Growth Hormone has been certified by the European Commission (EC) for the treatment of GHD in Europe in October 2019.
Drugs approved by the FDA for orphan drugs refer to those that can safely, effectively treat, diagnose, or prevent rare diseases or conditions affecting less than 200,000 US citizens, and may be safer or more effective than approved products. The orphan drug designation provides certain benefits and incentives for drug developers, including a seven-year US market monopoly period from the date of market authorization, exemption from FDA user fees and tax credits for clinical research. Granting orphan drug designations will not change FDA ’s regulatory requirements to determine the safety and effectiveness of drugs through appropriate and well-controlled research to support approval and commercialization.