Ascendis Pharma submits sBLA for TransCon hGH to treat GHD

Ascendis Pharma (NASDAQ:ASND) announced it has submitted a supplemental Biologics License Application (sBLA) to the U.S. Food & Drug Administration for its product TransCon hGH, marketed as SKYTROFA® (lonapegsomatropin-tcgd) in the U.S., seeking approval for the treatment of adults with growth hormone deficiency (GHD).

The company's application is supported by data from the foresiGHt trial, a robust Phase 3 study which included 259 adults with GHD, aged between 23 and 80 years.

This randomized, parallel-arm trial employed both placebo-controlled (double-blind) and active-controlled (open-label) methodologies to compare the effects of weekly TransCon hGH against a placebo and daily hGH injections.

Results from the trial have shown that TransCon hGH significantly outperforms the placebo in reducing trunk fat and increasing total body lean mass at the 38-week mark, fulfilling its primary and key secondary efficacy endpoints.

Additionally, TransCon hGH has demonstrated a safety profile comparable to daily hGH, with no study drug-related discontinuations reported, highlighting its tolerability and safety for adult patients.

The successful trial outcomes could potentially lead to expanded treatment options for adults suffering from growth hormone deficiency, pending FDA review and approval