Ascendis Pharma Submits Supplemental Biologics License Application to FDA for Adult Growth Hormone Deficiency Treatment

Ascendis Pharma A/S, a biopharmaceutical company headquartered in Copenhagen, Denmark, has recently submitted a supplemental Biologics License Application (sBLA) to the U.S. Food & Drug Administration (FDA) for their product, TransCon hGH. Known as SKYTROFA® (lonapegsomatropin-tcgd) in the U.S., this drug is currently used to treat pediatric growth hormone deficiency. The new application seeks approval for its use in adults with growth hormone deficiency (GHD).

Jan Mikkelsen, President and CEO of Ascendis Pharma, expressed that the sBLA submission aligns with the company's strategy to expand the SKYTROFA label and strengthen their market leadership in the U.S. growth hormone sector. Mikkelsen highlighted the critical role of growth hormone in overall health at all stages of life. He noted that many adults with GHD remain untreated, and a weekly dose of TransCon hGH could offer a valuable new treatment option.

The sBLA submission is anchored on findings from the "foresiGHt" clinical trial, a Phase 3 study designed to evaluate the efficacy and safety of TransCon hGH in adults aged 23 to 80 with GHD. This randomized, placebo-controlled (double-blind), and active-controlled (open-label) trial included 259 participants divided into three groups: those receiving TransCon hGH, a placebo, or daily hGH. The study compared the effects of weekly doses of TransCon hGH with daily hGH and a placebo, considering factors such as age and oral estrogen intake to determine the appropriate dosage.

At Week 38 of the trial, TransCon hGH demonstrated significant efficacy. Patients treated with TransCon hGH showed a notable reduction in trunk fat and an increase in total body lean mass compared to those given a placebo. The results, considered statistically significant, also suggested that TransCon hGH was generally safe and well tolerated, showing similar safety and tolerability profiles to daily hGH, with no discontinuations related to the study drug.

Ascendis Pharma uses its innovative TransCon technology platform to develop potential best-in-class therapies and aims to make a substantial impact on patients' lives. The company, guided by core values of Patients, Science, and Passion, operates facilities in both Europe and the United States.

Growth hormone deficiency in adults is a condition where the body does not produce sufficient growth hormone, affecting overall health. Growth hormone is crucial for protein production, fat utilization, muscle mass enhancement, and blood sugar regulation. Adults with GHD may experience a range of symptoms, including central obesity, metabolic syndrome, reduced bone density, altered lipid profiles, cardiovascular risks, fatigue, muscle weakness, and psychological issues such as cognitive impairment, social isolation, lack of motivation, and depression.

With the submission of the sBLA for TransCon hGH, Ascendis Pharma aims to address the unmet medical needs of adults with GHD, offering a potentially new and effective treatment option. The company's efforts highlight its commitment to advancing healthcare through innovative biopharmaceutical solutions.