U.S. Food and Drug Administration Approval of SKYTROFA® (lonapegsomatropin-tcgd), the First Once-weekly Treatment for Pediatric Growth Hormone Deficiency

2021-09-22

COPENHAGEN, Denmark, Aug. 25, 2021--Ascendis Pharma announced that the U.S. Food and Drug Administration (FDA) has approved SKYTROFA ® (lonapegsomatropin-tcgd, China’s trade name is under registration) for the treatment of pediatric patients one year and older who weigh at least 11.5 kg (25.4 lb) and have growth failure due to inadequate secretion of endogenous growth hormone (GH).

 

As a once-weekly injection, SKYTROFA is the first FDA approved product that delivers somatropin (growth hormone) by sustained release over one week.

 

 VISEN Pharmaceuticals, which is committed to becoming an expert in the field of endocrine-related therapy, is an exclusively authorized enterprise for development, manufacture and commercialization of SKYTROFA in Greater China and is currently carrying out domestic phase III clinical trials of the drug.

 

The approval includes the new SKYTROFA® Auto-Injector and cartridges which, after first removed from a refrigerator, allow families to store the medicine at room temperature for up to six months. With a weekly injection, patients switching from injections every day can experience up to 86 percent fewer injection days per year.  1

 

The FDA approval of SKYTROFA was based on results from the phase 3 heiGHt Trial, a 52-week, global, randomized, open-label, active-controlled, parallel-group trial that compared once-weekly SKYTROFA to daily somatropin (Genotropin®) in 161 treatment-naïve children with GHD. The primary endpoint was, AHV at 52 weeks for weekly SKYTROFA and daily hGH treatment groups. Other endpoints included adverse events, injection-site reactions, incidence of anti-hGH antibodies, annualized height velocity, change in height SDS, proportion of subjects with IGF-1 SDS (0.0 to +2.0), PK/PD in subjects < 3 years, and preference for and satisfaction with SKYTROFA.  1

 

At week 52, the treatment difference in AHV was 0.9 cm/year (11.2 cm/year for SKYTROFA compared with 10.3 cm/year for daily somatropin) with a 95 percent confidence interval [0.2, 1.5] cm/year. The primary objective of non-inferiority in AHV was met for SKYTROFA in this trial and further demonstrated a higher AHV at week 52 for lonapegsomatropin compared to daily somatropin, with similar safety, in treatment-naïve children with GHD.  1

 

Professor Luo Xiaoping, Vice Chairman of Pediatric Branch of Chinese Medical Association and Director of Department of Pediatrics, Tongji Hospital, Huazhong University of Science and Technology, indicated that GHD in children is a disease caused by the inability of adenohypophysis to produce enough growth hormone, which is usually manifested as short stature with normal proportion and inability to achieve ideal height. Patients have increased body fat and abnormal distribution ratio, dyslipidemia, decreased bone mineral density, and even accompanied by psychosocial disorders, cognitive deficits and decreased overall quality of life. Epidem iology data show that the prevalence of GHD in children is about 1 in 5,000. 2

 

The main purpose of growth hormone therapy for children with GHD is to accelerate growth velocity, promote growth and normalize height in childhood, and achieve normal adult height in line with the genetic potential of children. 3

 

" Growth hormone treatment is a long-term process. “Professor Luo Xiaoping indicated, "In recent decades, the conventional treatment has been once-daily subcutaneous injection of growth hormone for improving height and metabolic abnormalities. However, the heavy burden of daily injections often results in poor compliance, making it difficult to achieve the desired treatment goals. “Clinical studies have shown that as the number of missed injections per week increases in patients, the growth improvement effect of daily dosage also decreases.  4

 

SKYTROFA uses the " Transient conjugation." technology to release unmodified human growth hormone (hGH) at a predictable rate, resulting in the same tissue distribution as daily growth hormone therapy. As early as October 2019 and April 2020, SKYTROFA was granted Orphan Drug Designation (ODD) by the European Commission (EC) and the US FDA, respectively.

 

Pony Lu, CEO of VISEN Pharmaceuticals indicated that: "This approval provides a brand-new treatment option for children with GHD and their families. This once-weekly treatment is expected to improve the compliance of patients and prolong the duration of treatment so as to achieve better treatment outcomes. We are very pleased to see that SKYTROFA is approved in the United States and looking forward to the results of the Chinese phase III study. At VISEN,  we always put patients first. Innovation brings light to life, as we believe and are committed to help endocrine patients make the dreamed life possible. ”

 

About VISEN Pharmaceuticals

 

VISEN Pharmaceuticals is committed to the treatment of endocrine-related diseases, introducing the world's leading treatment methods and drugs into the China market, and hoping to provide more Chinese patients quick access to the world's most advanced and reliable treatment solutions.

 

In 2018, VISEN Pharmaceuticals was formed by Ascendis Pharma A/S (Nasdaq: ASND) and an investor syndicate led by Vivo Capital (along with participation by Sofinnova Investments). In January 2021, VISEN Pharmaceuticals completed series B financing, led by Sequoia China with participation from OrbiMed, Sherpa Healthcare Partners, Cormorant, HBM Healthcare Investments, Pivotal bioVenture Partners China, Logos Capital, and CDG Capital, to develop and commercialize endocrinology therapies in Greater China, which includes mainland China, Hong Kong, Macau, and Taiwan.

 

Reference

1. 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. Retrieved August 25, 2021, from  https://investors.ascendispharma.com/news-releases/news-release-details/ascendis-pharma-announces-us-food-and-drug-administration

2. Ranke MB, et al. Nat Rev Endocrinol. 2018;14(5):285-300.

3. Grimberg A, et al. Horm Res Paediatr. 2016;86(6):361-397.

4. Cutfield WS,et al. PLoS One. 2011;6(1):e16223.