TransCon hGH Aims for FDA Label Expansion for Adult Growth Hormone Deficiency

Credit: US Food and Drug Administration

Ascendis Pharma has submitted a supplemental Biologics License Application (sBLA) to the US Food and Drug Administration (FDA) for TransCon hGH (lonapegsomatropin) for the treatment of adults with growth hormone deficiency (GHD).¹

Announced on September 30, 2024, Ascendis indicated the sBLA submission was based on results from the Phase 3 foresiGHt trial, demonstrating the superiority of weekly TransCon hGH on primary efficacy and key secondary efficacy endpoints compared with placebo.

“Growth hormone plays a vital role in human health at all ages, and, with research showing that the majority of adults living with growth hormone deficiency are not currently treated for this condition, we believe once weekly TransCon hGH could provide a new potential treatment option to address this unmet medical need,” said Jan Mikkelsen, president, and chief executive officer of Ascendis Pharma.¹

Adult GHD is a well-recognized medical condition where an individual’s body does not produce enough growth hormones, causing abnormalities in substrate metabolism, bone remodeling, body composition, and psychological symptoms, including cognitive impairment, social isolation, lack of motivation, and depression.²

The randomized, parallel-arm, placebo-controlled (double-blind) and active-controlled (open-label) foresiGHt trial compared the efficacy and safety of weekly Transcon hGH with weekly placebo and daily hGH in adults with GHD.¹

The trial randomized 259 adults with GHD aged 23 to 80 years old 1:1:1, titrated to receive a target fixed dose of TransCon hGH, placebo, or daily hGH. Randomization was based on age and oral estrogen intake, with approximately equivalent hGH mg/week for TransCon hGH and daily hGH.

Upon analysis, TransCon hGH achieves superiority on primary and key secondary efficacy endpoints at Week 38, demonstrating a statistically significant reduction from baseline in trunk fat and an increase in total body lean mass at Week 38 versus placebo. Safety results showed TransCon hGH appeared safe and well-tolerated, without discontinuations related to the study drug and a similar safety profile to daily hGH.

In August 2021, the US Food and Drug Administration approved Ascenis Pharma’s lonapegsomatropin-tcgd (SKYTROFA®) for the treatment of pediatric GHD. Children aged ≥1 year who weighed ≥11.5 kg and had growth failure due to inadequate secretion of endogenous growth hormone could receive the once-weekly treatment.³

“The sBLA submission for TransCon hGH for adult GHD supports our strategy to develop [lonapegsomatropin] into a blockbuster product through label expansion and build upon our value leadership position in the US growth hormone market,” Mikkelsen added.¹

References

1. _{Ascendis Pharma announces submission of Supplemental Biologics License Application to FDA for TransConTM HGH for the treatment of adults with growth hormone deficiency: Ascendis Pharma. Ascendis Pharma Announces Submission of Supplemental Biologics License Application to FDA for TransConTM hGH for the Treatment of Adults with Growth Hormone Deficiency | Ascendis Pharma. September 30, 2024. Accessed September 30, 2024. https://investors.ascendispharma.com/news-releases/news-release-details/ascendis-pharma-announces-submission-supplemental-biologics.}
2. _{Feldt-Rasmussen U, Klose M. Adult Growth Hormone Deficiency- Clinical Management. [Updated 2022 May 23]. In: Feingold KR, Anawalt B, Blackman MR, et al., editors. Endotext [Internet]. South Dartmouth (MA): MDText.com, Inc.; 2000-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK425701/}
3. _{Ascendis Pharma A/S announces U.S. Food and Drug Administration approval of SKYTROFA® (Lonapegsomatropin-tcgd), the first once-weekly treatment for pediatric growth hormone deficiency: Ascendis pharma. Ascendis Pharma A/S Announces U.S. Food and Drug Administration Approval of SKYTROFA® (lonapegsomatropin-tcgd), the First Once-weekly Treatment for Pediatric Growth Hormone Deficiency | Ascendis Pharma. August 25, 2021. Accessed September 30, 2024. https://investors.ascendispharma.com/news-releases/news-release-details/ascendis-pharma-announces-us-food-and-drug-administration.}