In a significant advancement for children with achondroplasia, Spain has approved a new drug, ApproaCH of TransCon™ CNP (navepegritida), which aims to improve growth rates while simplifying administration. This follows the earlier approval of VOXZOGO, the first medication for this condition.
Recent clinical trial results indicate that the new drug not only enhances annual growth rates—1.57 cm more for children aged 2 to 5 and 1.78 cm for those aged 5 to 11—but also positively impacts mental health and overall quality of life. Verónica Díaz, Medical Director at Ascendis Pharma Iberia, explains that the drug's unique delivery technology allows for weekly injections instead of daily, making it easier for young patients.
Achondroplasia, a genetic condition affecting height, can lead to various medical complications and significantly impact mental well-being. With over 70% of affected individuals requiring surgery during their lifetime and many facing chronic pain and low self-esteem, this new treatment could be life-changing.
Ascendis Pharma is optimistic about seeking regulatory approval in the U.S. and Europe by 2025, aiming to address the needs of patients in Spain and Portugal. If approved, the drug could assist approximately 15 new patients each year in Spain, marking a hopeful future for families affected by this condition.