US FDA Nods Ascendis Pharma's sBLA for TransCon hGH in Adult GHD

Ascendis Pharma A/S, a worldwide pharmaceutical firm utilizing its cutting-edge TransCon technology platform to develop a prominent and fully integrated biopharma business dedicated to significantly improving patients' lives, reported that the US Food & Drug Administration (FDA) has approved its supplemental Biologics License Application (sBLA) for evaluation to address adult growth hormone deficiency (GHD) with TransCon hGH (lonapegsomatropin-tcgd; sold as Skytrofa for pediatric GHD). The US FDA established a goal date of July 27, 2025, for the Prescription Drug User Fee Act (PDUFA).

“This marks another step towards achieving our objective to expand Skytrofa’s label beyond paediatric GHD and expand its reach to address new groups of patients,” said Jan Mikkelsen, Ascendis Pharma’s president and chief executive officer. “Adult GHD is an undertreated condition associated with significant comorbidities and higher annual healthcare costs compared to the 5-10% of patients who receive treatment, indicative of the high unmet need.”

The sBLA submission relies on findings from foresiGHt, a phase 3 randomized, parallel-group, placebo-controlled (double-blind) and active-controlled (open-label) trial that assessed the efficacy and safety of weekly TransCon hGH compared to weekly placebo and daily human growth hormone (hGH) in adults with GHD. The clinical trial assessed 259 adults with GHD, aged 23 to 80 years, who were randomized in a 1:1:1 manner, titrated to receive a set fixed dose of TransCon hGH, placebo, or daily hGH according to age and oral estrogen consumption, with roughly comparable hGH mg/week for both TransCon hGH and daily hGH.