SAN RAFAEL, Calif., July 13, 2026
BioMarin Pharmaceutical Inc. has reached a significant regulatory milestone after the U.S. Food and Drug Administration (FDA) accepted the company’s Supplemental New Drug Application (sNDA) seeking traditional full approval for VOXZOGO® (vosoritide) in children with achondroplasia, the most common form of skeletal dysplasia that causes disproportionate short stature. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2027, moving the therapy one step closer to converting from its current accelerated approval status to full approval. The application is supported by the largest and longest body of clinical evidence ever generated for a medicine in achondroplasia, including long-term safety and efficacy findings demonstrating improvements not only in linear growth but also in adult height, body proportionality, arm span, and overall skeletal development. If approved, VOXZOGO would become the first therapy for achondroplasia to transition from accelerated approval to traditional approval based on comprehensive long-term clinical outcomes, representing a landmark achievement in rare disease medicine.
Long-Term Clinical Data Strengthens FDA Submission
The accepted sNDA is supported by extensive data collected from three ongoing clinical studies, providing the FDA with the most comprehensive evidence package ever assembled for an achondroplasia therapy. These studies evaluated long-term safety, sustained efficacy, adult height outcomes, body proportionality, arm span, and additional skeletal growth measures, confirming clinically meaningful benefits beyond annualized growth velocity. VOXZOGO, a C-type natriuretic peptide (CNP) analog, works by regulating the signaling pathway downstream of the FGFR3 gene, helping restore normal bone growth in children whose skeletal development is impaired by the genetic mutation responsible for achondroplasia. Since receiving FDA accelerated approval in 2021, VOXZOGO has become the only approved medicine designed to promote bone growth in children with achondroplasia from birth and has now been used by more than 5,000 infants and children across over 50 countries, demonstrating its expanding global clinical impact. The long-term evidence submitted aims to satisfy the FDA’s post-marketing requirements by confirming durable clinical benefits across multiple meaningful outcomes.
VOXZOGO Continues to Transform Rare Disease Treatment
Achondroplasia affects approximately one in every 25,000 live births worldwide and results from a mutation in the FGFR3 gene, disrupting normal endochondral bone growth and causing disproportionate skeletal development. More than 80% of affected children are born to parents of average stature, making early diagnosis and intervention essential for improving long-term outcomes. International treatment guidelines recommend initiating VOXZOGO as early as possible while growth plates remain open, enabling children to maximize growth potential during critical developmental years. Beyond increasing height, BioMarin continues to evaluate VOXZOGO’s impact on spinal morphology, tibial bowing, body proportionality, quality of life, and other clinically important skeletal outcomes, reflecting a broader commitment to improving the overall health and mobility of patients living with achondroplasia. Continued clinical development is expected to further strengthen understanding of the therapy’s long-term benefits while expanding treatment opportunities for children with rare skeletal disorders.
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Regulatory Milestone Reinforces BioMarin’s Rare Disease Leadership
The FDA’s acceptance of the VOXZOGO sNDA highlights BioMarin’s continued leadership in rare disease innovation and advances the company’s mission of delivering transformative therapies for genetically defined disorders. Conversion from accelerated approval to traditional approval would represent an important validation of VOXZOGO’s long-term clinical value while strengthening physician confidence and expanding treatment opportunities for eligible patients worldwide. With a robust portfolio of rare disease therapies and an active pipeline targeting serious inherited disorders, BioMarin continues to demonstrate how sustained investment in genetic medicine and long-term clinical research can produce meaningful advances for patients with significant unmet medical needs. As the FDA reviews the application ahead of the February 2027 PDUFA decision, the rare disease community will closely follow what could become another major regulatory success for one of the biotechnology industry’s leading innovators in precision medicine.
Source: BioMarin press release
