BioMarin’s VOXZOGO sNDA Accepted by FDA for Full Approval in Children with Achondroplasia

BioMarin Pharmaceutical has received U.S. Food and Drug Administration (FDA) acceptance of its supplemental New Drug Application seeking traditional approval of VOXZOGO® (vosoritide) for children with achondroplasia. The FDA has assigned a Prescription Drug User Fee Act target action date of February 28, 2027.

The submission follows VOXZOGO’s accelerated approval in 2021 and is intended to satisfy the FDA’s postmarketing requirement to confirm long-term clinical benefit. If approved, VOXZOGO would become the first therapy for achondroplasia to transition from accelerated to traditional FDA approval based on comprehensive long-term clinical evidence.

Long-term data support sustained clinical benefit

The sNDA is supported by long-term safety and efficacy findings from three ongoing studies 111-205 (NCT02724228), 111-208 (NCT03989947), and 111-302 (NCT03424018). Together, these studies represent the largest and longest clinical evidence package generated for a therapy in achondroplasia.

Beyond improvements in linear growth, the submission includes additional clinically meaningful outcomes that assess broader skeletal development. These include adult height, body proportionality, arm span, and other measures of skeletal growth evaluated over extended follow-up.

According to BioMarin, the accumulated data demonstrate durable improvements in multiple growth-related parameters while continuing to support the therapy’s long-term safety profile.

VOXZOGO targets the underlying cause of achondroplasia

Achondroplasia is the most common form of skeletal dysplasia causing disproportionate short stature. The disorder results from mutations in the FGFR3 gene, which suppresses endochondral bone growth during skeletal development.

VOXZOGO is a C-type natriuretic peptide (CNP) analog that counteracts excessive FGFR3 signaling. By restoring downstream signaling involved in bone formation, the therapy promotes endochondral bone growth in children whose growth plates remain open.

More than 80% of affected children are born to parents of average stature as a result of spontaneous genetic mutations. The condition occurs in approximately one in every 25,000 live births worldwide.

Clinical development extends beyond annualized growth velocity

VOXZOGO originally received accelerated approval in the United States based on improvements in annualized growth velocity. The current application expands the evidence base by demonstrating sustained benefits beyond linear growth alone.

BioMarin continues to evaluate additional clinically relevant outcomes through ongoing extension studies, including spinal morphology, spinal stenosis, tibial bowing, body proportionality, arm span, and patient quality of life.

The therapy has now been used in more than 5,000 infants and children across over 50 countries, providing substantial real-world clinical experience.

Clinical Implication

Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin, said the submission is supported by the most extensive evidence generated for any medicine in achondroplasia. He noted that the long-term data demonstrate clinically meaningful improvements across multiple skeletal growth measures beyond annualized growth velocity and could establish VOXZOGO as the first achondroplasia therapy to convert from accelerated to traditional FDA approval.

Regulatory Path Forward

The FDA is expected to complete its review by February 28, 2027. A positive decision would convert VOXZOGO’s current accelerated approval into full approval based on confirmed long-term clinical benefit.

VOXZOGO is currently approved in the United States, Japan, and Australia for increasing linear growth in children with achondroplasia and open growth plates. In the European Union, it is approved for children aged four months and older whose diagnosis has been confirmed by appropriate genetic testing.

What This Means for Patients

Children receiving VOXZOGO may soon benefit from full FDA approval supported by long-term evidence showing sustained improvements not only in height but also in overall skeletal development, including body proportions and arm span. Traditional approval would strengthen confidence in the therapy’s long-term clinical benefits for families and healthcare providers.

Reference

U.S. Food and Drug Administration Accepts BioMarin’s Supplemental New Drug Application for Full Approval of VOXZOGO® (vosoritide) for Children with Achondroplasia | BioMarin Corporate