Ascendis Pharma Says FDA Approves SKYTROFA For Adults With Growth Hormone Deficiency

Ascendis Pharma

141.00 EUR -1.40%

• Charts
• News
• Analysen

Kaufen Verkaufen

(RTTNews) - Swiss biopharmaceutical company Ascendis Pharma A/S (ASND) announced Monday that the U.S. Food & Drug Administration (FDA) has approved SKYTROFA (lonapegsomatropin-tcgd; developed as TransCon hGH) for the replacement of endogenous growth hormone in adults with growth hormone deficiency (GHD).

Lonapegsomatropin (approved by the FDA in 2021 for the treatment of pediatric GHD) is a prodrug of somatropin (human growth hormone, or hGH) administered once weekly, providing sustained release of active, unmodified somatropin.

The FDA's approval of SKYTROFA for adult GHD was based on results from foresiGHt, a Phase 3 randomized, parallel-arm, placebo-controlled (double-blind) and active-controlled (open-label) clinical trial that compared the efficacy and safety of weekly TransCon hGH with weekly placebo and daily somatropin in adults with GHD.

The company said this is the first of many planned label expansions supporting Vision 2030 goal to become the leading endocrinology rare disease company.

The company is on track to initiate basket trial for ISS, SHOX deficiency, Turner syndrome, and SGA, as well as combination therapy trials in achondroplasia and hypochondroplasia, in the fourth quarter of 2025.