Benzinga | Sep 8, 2020 04:45PM EDT

Ascendis Pharma A/S Submits Marketing Authorization Application To European Medicines Agency For TransCon hGH For Treatment Of Pediatric Growth Hormone Deficiency

COPENHAGEN, Denmark, Sept. 08, 2020 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (NASDAQ:ASND), a biopharmaceutical company that uses its novel TransCon technologies to address unmet medical needs, announced today the submission of a MAA to the EMA seeking approval for TransCon hGH (lonapegsomatropin), an investigational long-acting once-weekly prodrug of somatropin (human growth hormone or hGH), for the treatment of pediatric patients who are diagnosed with growth hormone deficiency (GHD).



"This MAA submission, which follows the approval of our Paediatric Investigation Plan (PIP) from EMA, is an important next step for extending the global reach of TransCon hGH," said Dana Pizzuti, M.D., Ascendis Pharma's Senior Vice President of Development Operations. "If approved, TransCon hGH could provide clinicians and patients with a once-weekly treatment option."

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 MAA submission includes data fromthe clinical development program for TransCon hGH that included eight clinical trials evaluating safety and efficacy in more than 400 subjects with GHD.

A Biologics License Application seeking approval for TransCon hGH for the treatment for pediatric GHD is under review by theU.S. Food and Drug Administration(FDA), and a PDUFA date is set for June 25, 2021.

TransCon hGH is an investigational therapy that is not approved for use in any country. There is no long-acting growth hormone treatment currently approved by the EMA for the treatment of pediatric GHD. TransCon hGH has received orphan designation in both the U.S. and Europe as a treatment for GHD.