– Prescription Drug User Fee Act (PDUFA) target action date of June 25, 2021 –
– FDA indicated that it is currently not planning to hold an Advisory Committee Meeting –
COPENHAGEN, Denmark, Sept. 04, 2020 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (NASDAQ:ASND), a biopharmaceutical company that uses its novel TransCon technologies to address unmet medical needs, today announced that the FDA has accepted the company’s BLA for TransCon hGH (lonapegsomatropin), an investigational long-acting prodrug of somatropin (human growth hormone or hGH) for the treatment for pediatric GHD. A PDUFA date is set for June 25, 2021. The FDA also has indicated that it is currently not planning to hold an Advisory Committee Meeting to discuss the application at this time.
“We look forward to engaging with the FDA during its review of our BLA submission for TransCon hGH in pediatric GHD,” said Dana Pizzuti, M.D., Ascendis Pharma’s Senior Vice President of Development Operations. “Based on data from our clinical development program, we believe once-weekly TransCon hGH has the potential to expand treatment options for clinicians and children with GHD.”
TransCon hGH is designed to release somatropin with the same mode of action and distribution as once-daily somatropin products, but with a once-weekly injection.1 The BLA for pediatric GHD is supported by the results a clinical development program that included eight clinical trials evaluating safety and efficacy in more than 400 subjects with GHD.
TransCon hGH is an investigational therapy that is not approved for use in any country. There is no long-acting growth hormone treatment approved by the FDA for the treatment of pediatric GHD. TransCon hGH has received orphan designation in both the U.S. and Europe as a treatment for GHD. The company plans to submit a Marketing Authorisation Application for TransCon hGH in pediatric GHD to the European Medicines Agency in the third quarter of 2020.