2024-12-09
- Innovative long-acting fusion protein regimen offers a safe, efficacious and more convenient weekly treatment option for children with growth hormone deficiency in China
HANGZHOU, China – December 9, 2024 – TJ Biopharma (“TJ Bio” or “Company”), a fully integrated biotech company in China that develops, manufactures, and commercializes through partnership on innovative biologics for autoimmune diseases, oncology and metabolic diseases, today announced jointly with Jumpcan Pharmaceutical (“Jumpcan”) that the Biologics License Application (BLA) for eftansomatropin alfa has been accepted for review by China’s National Medical Products Administration (NMPA) for the treatment of pediatric growth hormone deficiency (PGHD). As the first and only fusion protein long-acting growth hormone among the marketed products in China, this innovative therapy is expected to offer a safe, efficacious and more convenient treatment option to pediatric patients with gowth hormone defficiency.
In China, pediatric patients under lengthy growth hormone treatment are burdened by frequent daily injections of the existing short-acting regimens. Multiple studies have shown that over 30% of patients using daily injections of growth hormone miss treatment doses every week, which significantly impacts patient compliance and treatment effectiveness.
Eftansomatropin alfa, developed through the innovative hyFc® fusion protein technology, combines human growth hormone with a proprietary engineered human immunoglobulin to achieve enhanced drug stability and extended half-life. While keeping the natural biological activity of growth hormone, this patented technology enables sustained therapeutic levels in the body, making eftansomatropin alfa the first long-acting growth hormone fusion protein that can be administered once-weekly. The Phase 3 registrational study (CTR20202305) demonstrated a compelling efficacy and safety profile:
- The study met its primary endpoint of annualized height velocity (AHV) at week 52, demonstrating non-inferiority compared to Norditropin®, a marketed daily growth hormone in China
- Eftansomatropin alfa, administered once-weekly, achieved an AHV of 10.76 cm/year vs. 10.28 cm/year for daily Norditropin®, with a between-group difference of 0.47 (95% CI: -0.06, 1.00, p<0.0001)
- Eftansomatropin alfa was well-tolerated with no treatment-related adverse events leading to discontinuation, demonstrating a safety profile comparable to Norditropin®
“Eftansomatropin alfa represents a significant advancement in growth hormone therapy through protein engineering technology to deliver a safe and efficacious long-acting weekly regimen,” said Professor Xiaoping Luo, Leading Principal Investigator of the Phase 3 study, Director of Pediatrics at Tongji Hospital, Tongji Medical College of Huazhong University of Science and Technology. “The Phase 3 clinical study results demonstrated that the desired height velocity outcomes accompanied by a favorable safety profile are consistent with current daily treatments. Notably, its fusion protein technology enabling weekly dosing has the advantage to reduce the treatment burden for children and their families while optimizing treatment outcomes through improved compliance. We're eager to see this innovative therapy become available to children with growth hormone deficiency in China, offering them a better and safer treatment option.”
“The acceptance of BLA for eftansomatropin alfa represents a pivotal moment for TJ Bio, as we advance the first differentiated long-acting growth hormone product toward potential marketing approval in China,” said Dr. Jingwu Zang, Founder and Chairman of TJ Bio. “This milestone marks our first BLA submission of a series of advanced late-stage assets in our pipeline and reflects our dedication to transforming innovative science into clinically differentiated treatment options for patients. In this case, through years of research and development, we have delivered a differentiated growth hormone product that is shown to be safe, efficacious, and more convenient to address the treatment burden of currently used daily injections. Together with Jumpcan, we are committed to bringing this differentiated therapy to patients as quickly as possible in recognition of the pressing need for a more convenient treatment option for PGHD children and their families.”
“At Jumpcan, our leadership in pediatric therapeutic area is built on our commitment to bringing innovative, high-quality treatments to patients,” said Mr. Wei Cao, General Manager of Jumpcan. “Our collaboration with TJ Bio on eftansomatropin alfa exemplifies this commitment and represents a cornerstone of our pediatric therapeutic strategy. This BLA submission marks a significant step forward in our partnership, and we're excited and ready to bring this innovative product to the market after its approval.”
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About Eftansomatropin Alfa
Eftansomatropin alfa is a differentiated long-acting recombinant fusion protein of human growth hormone currently under review for market approval in China. It is designed to provide a safe, efficacious and more convenient weekly treatment option. The product leverages innovative hyFc® fusion protein technology, combining human growth hormone with a proprietary engineered human immunoglobulin to achieve enhanced drug stability and extended half-life, enabling an once-weekly dosing regimen.
Mechanistically, eftansomatropin alfa acts as a replacement for endogenous growth hormone, stimulating hepatic production of insulin-like growth factor 1 (IGF-1) and maintaining elevated serum IGF-1 levels. IGF-1, serving as a key mediator of growth-promoting properties, activates osteoblasts and chondrocytes throughout the body to stimulate skeletal growth and development.
In November 2021, TJ Biopharma and Jumpcan entered into a strategic partnership for the development, manufacturing, and commercialization of eftansomatropin alfa.
About Pediatric Growth Hormone Deficiency
Pediatric growth hormone deficiency (PGHD) is an endocrine disorder that impairs children’s growth and development. In China, approximately 3.4 million children are affected by growth hormone deficiency, yet fewer than 5% receive a standardized growth hormone treatment, highlighting a unmet medical need and treatment gap.
The current standard of care requires daily injections of short-acting growth hormone treatments. Studies show that about 22% of children miss four or more injections monthly, which compromises the treatment outcomes. This underscores a critical need for a more convenient weekly dosing option to enhance treatment adherence and improve treatment outcomes.
China’s growth hormone market has experienced remarkable expansion, growing from US$600 million in 2018 to US$1.7 billion in 2022, representing a compound annual growth rate of 29.3%. The market has emerged as the global leader, capturing 34% of worldwide market share and surpassing the United States. Driven by increasing treatment adoption, earlier intervention, and the transition to weekly dosing options, the market is projected to reach US$4.8 billion by 2030.
About TJ Biopharma
TJ Biopharma is a fully integrated biotech company at the forefront of pioneering cutting-edge therapies for autoimmune diseases, immuno-oncology and metabolic disorders. Through its R&D and manufacturing establishments in China, including comprehensive R&D centers in Shanghai and Beijing and a state-of-the-art GMP manufacturing facility in Hangzhou, TJ Biopharma has developed a differentiated and advanced pipeline of innovative clinical and pre-clinical assets, with its first series of advanced clinical assets now progressing toward market approval. Over the years, the Company has established a series of development and commercialization partnerships with Jumpcan, CSPC, Sanofi, and others to commercialize its pipeline products. TJ Biopharma is committed to driving excellence in drug innovation and development through a diverse and sustainable revenue model comprising global asset transactions, contract manufacturing services and future sales revenues. For more information, please visit: TJBio.com.
About Jumpcan Pharmaceutical
Jumpcan Pharmaceutical is a leading Chinese pharmaceutical company focused on developing and commercializing both traditional Chinese medicines and innovative drugs, along with healthcare and consumer products. As a national high-tech enterprise and technology innovation demonstration company, Jumpcan maintains fully integrated capabilities across R&D, manufacturing, and commercialization. Listed on the Shanghai Stock Exchange and ranked among China's top 20 pharmaceutical companies, Jumpcan is committed to addressing evolving healthcare needs through continuous innovation and market expansion. The company pursues a patient-centric approach to drug development while advancing its global presence through strategic industrialization and commercialization initiatives. For more information, please visit: www.jumpcan.com.
References:
- Frost & Sullivan. China Growth Hormone Market Research Report, 2023.
- E&Y. White Paper on Growth Hormone Usage Among Chinese Children. 2021.