VISEN Pharmaceuticals, a biotech company 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, announced that the China Center for Drug Evaluation (CDE) of National Medical Products Administration (NMPA) has approved the IND application to conduct a phase 2 clinical trial of TransCon TM C-Type Natriuretic Peptide (CNP) for patients with achondroplasia (ACH). The trial is ready to be launched in China as "the ACcomplisH China trial", in coordination with the ACcomplisH trial, an ongoing phase 2 global clinical trial of TransCon CNP being conducted globally outside of China by Ascendis Pharma.
TransCon CNP is an investigational once-weekly long-acting prodrug of CNP in development for the treatment of ACH in pediatric patients. It is designed to provide continuous CNP exposure which inhibits the effect of constitutive fibroblastgrowth factor receptor 3 (FGFR3) over-activation 24 hours a day, aiming to promote bone growth and ameliorate and prevent complications and comorbidities of ACH. TransCon CNP received Orphan Designation and Orphan Drug Designation (ODD) by the European Commission and the United States Food and Drug Administration (U.S. FDA), respectively.
ACH Poses Heavy Burden on Patients, their Families, and Society
ACH is a genetic disease and the most common form of dwarfism, caused by an autosomal dominant activating mutation in fibroblast growth factor receptor 3 (FGFR3) that leads to an imbalance between the FGFR3 and CNP signaling pathways. Around 80 percent of individuals with ACH have parents with normal stature and have achondroplasia as the result of a de novo pathogenic variant[1] and may experience severe skeletal complications and comorbidities. According to available data, the global incidence of the disease is approximately 1 in 25,000[2] in the newborn, affecting about 250,000 people worldwide[3] . Affected individuals have rhizomelic shortening of the limbs, macrocephaly, and characteristic facial features with frontal bossing and midface retrusion. Hypo dystonia in infancy is typical, and patients typically reach an average height of only about 1.31m for males, 1.24m for females by adulthood[4]. ACH can also lead to a variety of serious complications, including foramen magnum stenosis, obstructive sleep apnea and chronic ear infections, resulting in poor quality of life and an increased risk of death.
Achondroplasia can be diagnosed by characteristic clinical and radiographic findings in most affected individuals[1]. In individuals in whom there is diagnostic uncertainty or who have atypical findings, identification of a heterozygous pathogenic variant in FGFR3 can establish the diagnosis[1]. There are currently no effective drugs approved for ACH[2]. Due to the lack of fundamental treatment options, the prevention and control for a variety of comorbidities often leads to high medical expenses, bringing a heavy burden to patients, their families, and the social insurance system. Additional difficulties in self-care, education, and employment, low income and other factors provide obstacles to patients' full integration into society, which further increases the burden of living, and in turn results in the unemployment, care burden on families and increase in social insurance expenditures. The situation underlines the urgent need to accelerate the research and development of new drugs targeting the pathogeny to alleviate the burden on patients, families, and society.
Phase 2 Clinical Trial of TransCon CNP Sets to Accelerate Research and Development
Preclinical and clinical data have shown that increased CNP can offset the effects of the FGFR3 mutation, thereby promoting bone growth[5]. However, the half-life of natural CNP in the human body is short, only 2-3 minutes[6], requiring continuous intravenous infusion, posing significant difficulties for clinical application.
Currently, TransCon CNP is the world's only long-acting CNP designed with the patented Tra...
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