Visen Pharmaceuticals, a joint venture between Denmark-based Ascendis Pharma A/S (NASDAQ: ASND) and Chinese healthcare investment firm Vivo Capital, has announced topline results from the ACcomplisH China Phase II trial. The study assessed the safety and efficacy of once-weekly TransCon CNP (navepegritide) in children with achondroplasia (ACH) aged 2 to 10 years in China. CNP, or C-type natriuretic peptide, is a naturally occurring peptide that regulates bone growth and has shown potential in treating growth and skeletal disorders such as dwarfism.
Patients dosed with TransCon CNP at the 100 μg CNP/kg/week demonstrated significantly higher annualized growth velocity (AGV) compared to placebo, with an AGV of 1.180 cm/year at Week 52 (P=0.018).
Achondroplasia’s Global Impact and Treatment Landscape
ACH, the most common form of skeletal dysplasia, affects approximately 250,000 people worldwide. Individuals with ACH may experience severe skeletal complications and comorbidities, including sleep apnea, chronic back and leg pain, and in severe cases, sudden infant death from cervical compression. ACH is included in China’s second batch of rare disease list.
TransCon CNP, globally owned and developed by Ascendis Pharma, is undergoing a global randomized, double-blind, placebo-controlled Phase III pivotal study involving 80 ACH children aged 2 to 11. Visen holds exclusive rights for the development, manufacturing, and commercialization of TransCon CNP, human growth hormone (hGH), and parathyroid hormone in Greater China.- Flcube.com
