FDA Grants Priority Review to TransCon® CNP NDA

Ascendis Pharma A/S has announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for TransCon CNP (navepegritide) for priority review. This investigational drug aims to treat children with achondroplasia, a genetic disorder that impacts bone growth and can cause a variety of complications. The FDA's acceptance is an important step toward potentially improving treatment options for those affected by this condition.

The FDA's priority review is a designation reserved for treatments that could significantly enhance the safety or effectiveness of managing serious medical conditions. For TransCon CNP, the Prescription Drug User Fee Act (PDUFA) goal date is set for November 30, 2025. This timeline indicates when the FDA plans to complete its review of the drug application. Notably, the FDA has stated that an advisory committee meeting is not planned for this application, suggesting a streamline in the evaluation process.

TransCon CNP is a prodrug of the C-type natriuretic peptide (CNP) and is administered weekly. The treatment is designed to maintain continuous exposure to active CNP, targeting tissues throughout the body, including growth plates and skeletal muscle. This mechanism aims to address the overactive fibroblast growth factor receptor 3 (FGFR3) pathway associated with achondroplasia. Clinical trials have demonstrated that TransCon CNP can significantly improve annualized growth velocity and offer multiple benefits beyond linear growth compared to a placebo. These benefits include enhancements in lower limb alignment, spinal canal dimensions, and muscle strength. The safety and tolerability profile of TransCon CNP is reportedly similar to that of a placebo.

Chandler Crews, founder of The Chandler Project, emphasized the urgent need for therapies that address the profound medical challenges faced by individuals with achondroplasia. The hope is that TransCon CNP can improve health outcomes where current treatments fall short.

Dr. Janet Legare, a professor of pediatrics, noted her encouragement at the FDA's priority review designation for TransCon CNP, highlighting its potential as a new treatment for children with achondroplasia. Aimee Shu, Executive Vice President of Endocrine & Rare Disease Medical Sciences at Ascendis Pharma, also expressed optimism about the drug's prospects. According to Shu, the clinical trials are the first to show improvements beyond linear growth at 52 weeks, underscoring the need for an effective treatment option to manage the complications arising from achondroplasia.

Achondroplasia affects over 250,000 people globally and is characterized by a genetic variant that disrupts the balance between the FGFR3 and CNP signaling pathways. This imbalance leads to various complications, including skeletal dysplasia, muscular and neurological issues, and cardiorespiratory challenges. The condition is not just limited to bone growth disorders; it can significantly impact quality of life through various stages of life, necessitating multiple surgeries and procedures to manage its complications.

Ascendis Pharma A/S is dedicated to developing new therapies through its TransCon technology platform, focusing on unmet medical needs. The company's commitment to science and patient care drives its innovation efforts, as demonstrated by the development of TransCon CNP. With headquarters in Copenhagen, Denmark, Ascendis Pharma continues to expand its reach with facilities across Europe and the United States. The potential approval of TransCon CNP could mark a significant advancement in the treatment of achondroplasia, offering hope for improved outcomes for affected individuals and their families.