Ascendis Pharma, a Danish biopharma company, said that the US Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for TransCon hGH (lonapegsomatropin) for the treatment for pediatric growth hormone deficiency (GHD).
TransCon hGH is an investigational long-acting prodrug of somatropin (human growth hormone or hGH).
The FDA has set a Prescription Drug User Fee Act (PDUFA) date for 25 June 2021 to take a decision on the investigational biologic’s approval.
Ascendis Pharma said that the FDA also indicated to it that it presently does not intend to hold an advisory committee meeting to discuss the BLA at this time.
Dana Pizzuti – Ascendis Pharma Senior Vice President of Development Operations said: “We look forward to engaging with the FDA during its review of our BLA submission for TransCon hGH in pediatric GHD.
“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.”
According to Ascendis Pharma, TransCon hGH releases somatropin with the same mode of action and distribution as daily once somatropin products, however with a weekly once injection.
The Danish biopharma company said that the BLA for pediatric GHD is backed by the data from a clinical development program that included eight clinical trials, which assessed safety and efficacy in over 400 subjects with GHD.