The U.S. Food and Drug Administration has accepted a new application seeking full approval for VOXZOGO (vosoritide), the first medicine designed to help children with achondroplasia grow taller. A decision is expected by February 28, 2027.
Achondroplasia is the most common form of disproportionate short stature, affecting about one in 25,000 live births worldwide. It is caused by a genetic mutation that slows bone growth. VOXZOGO, a C-type natriuretic peptide analog, works by promoting endochondral bone growth, counteracting the underlying genetic defect.
The application is backed by data from three ongoing studies that represent the largest and longest evidence base for any achondroplasia treatment. Beyond height, the data show improvements in body proportionality, arm span, and other skeletal health measures. If approved, VOXZOGO would become the first therapy for achondroplasia to convert from accelerated approval to traditional approval based on adult height outcomes and extended follow-up.
The drug received accelerated approval from the FDA in 2021, allowing earlier patient access based on measures that reasonably predict clinical benefit. This supplemental application aims to confirm that benefit with long-term results. VOXZOGO is currently approved in more than 50 countries and has been used by over 5,000 children. It is recommended for children whose growth plates remain open, typically those under 18 years of age.
Looking ahead, the FDA’s decision will determine whether VOXZOGO becomes the first fully approved therapy for achondroplasia in the United States, offering families a treatment supported by comprehensive long-term evidence. BioMarin continues to evaluate the drug for additional outcomes such as spinal morphology and quality of life, with the goal of improving care for children living with this condition.