Biotech Advances VOXZOGO Toward Landmark US FDA Approval

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1. Ownership and Share Structure Information

The information listed above was updated on the date this article was published and was compiled from information from the company and various other data providers.

BioMarin Pharmaceuticals Inc. (BMRN:NASDAQ) announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) seeking traditional approval for VOXZOGO® (vosoritide) to treat children with the genetic bone-growth disorder achondroplasia, according to a July 13 release.

The FDA assigned the application a Prescription Drug User Fee Act (PDUFA) target action date of Feb. 28, 2027.

"This submission for VOXZOGO is supported by the largest body of evidence for any medicine in achondroplasia, reflecting BioMarin's long-standing commitment to advancing the science of skeletal growth," said BioMarin Executive Vice President and Chief Research and Development Officer Dr. Greg Friberg. "The clinical data demonstrate meaningful improvements across multiple skeletal growth-related measures beyond annualized growth velocity in children with this condition. If approved, VOXZOGO would be the first therapy for achondroplasia to convert from accelerated approval to traditional approval based on a comprehensive clinical data package, including adult height outcomes and other clinical measures evaluated over extended follow-up."

The application is backed by extensive long-term safety and efficacy results from three ongoing studies, identified as 111-205, 111-208, and 111-302. BioMarin said the research demonstrated clinically meaningful improvements in growth as well as important skeletal growth measures, including body proportionality and arm span. The submission also contains the longest-running safety and efficacy dataset assembled for any therapy studied in achondroplasia.

VOXZOGO received accelerated FDA approval in 2021 through a pathway intended to speed patient access to treatments based on measures that are reasonably likely to predict clinical benefit. BioMarin said the current filing is designed to satisfy the postmarketing requirement by confirming the benefit and securing full approval through long-term data from the three ongoing studies, which continue to show meaningful improvements in growth and skeletal health outcomes for children with achondroplasia.

More than 80% of Affected Children Born to Average-Height Parents

Achondroplasia is the most common form of skeletal dysplasia that causes disproportionate short stature. The condition develops when endochondral ossification slows, leading to abnormal development of the long bones, spine, face, and base of the skull. It is caused by a mutation in the FGFR3 gene, which normally acts as a regulator of bone growth.

BioMarin said more than 80% of children with achondroplasia are born to parents of average height, with the condition resulting from a spontaneous genetic mutation. The disorder occurs in approximately one out of every 25,000 live births worldwide. The company is studying VOXZOGO in children whose growth plates remain "open," generally those younger than 18 years old, a population representing about 25% of people with achondroplasia. BioMarin added that further information about its clinical studies involving achondroplasia, hypochondroplasia, and other skeletal disorders is available through its clinical trials website.

According to BioMarin, VOXZOGO is a C-type natriuretic peptide (CNP) analog that targets the biological pathway downstream of FGFR3 to stimulate endochondral bone growth, which is impaired in children with achondroplasia because of a gain-of-function mutation in the FGFR3 gene. The company said the therapy is the only approved medicine that supports growth in children with achondroplasia beginning at birth, and international treatment guidelines recommend starting therapy as early as possible.

Since its initial approval in 2021, VOXZOGO has been used to treat more than 5,000 infants and children in over 50 countries. BioMarin said it continues to study the therapy's effects on clinically important measures, including arm span, tibial bowing, body proportionality, spinal morphology, including spinal stenosis, and quality of life.

The company said VOXZOGO is approved in the United States, Japan, and Australia to increase linear growth in children of all ages with achondroplasia whose growth plates remain open. In the European Union, the treatment is approved for children four months of age and older whose growth plates have not closed, provided genetic testing confirms the diagnosis. In the United States, the indication remains under accelerated approval based on improved annualized growth velocity, with continued approval dependent on confirmatory evidence of clinical benefit. BioMarin said it plans to meet that postmarketing requirement by comparing data from its ongoing open-label extension studies with available natural history data.

BioMarin described itself as a global biotechnology company focused on developing treatments for rare diseases caused by genetic conditions. The San Rafael, California-based company said it has concentrated on advancing medicines that address genetically defined disorders since its founding in 1997.

The company said its portfolio includes nine commercial therapies supported by a broad pipeline of clinical and preclinical drug candidates. BioMarin added that its research and development strategy emphasizes innovative approaches to drug discovery with the goal of creating category-defining therapies that deliver meaningful benefits for patients living with rare genetic diseases.

Therapy Converting From Accelerated to Traditional Approval

According to an updated research note on July 14 by H.C. Wainwright & Co. Analyst Mitchell S. Kapoor, the acceptance by the FDA was "anticipated."

"If approved, VOXZOGO would be the first therapy for ACH to convert from accelerated approval to traditional approval based on a comprehensive clinical data package, including adult height outcomes and other clinical measures evaluated over extended follow-up," Kapoor wrote.

The analyst said the prospect of VOXZOGO receiving full approval aligns with existing expectations and is not expected to materially change the competitive outlook as rival programs continue to advance. Attention is now focused on BioMarin's expected 2027 guidance for hypochondroplasia (HCH), quarterly trends in VOXZOGO patient starts, treatment persistence, and potential pressure on new patient starts and treatment switching in the United States following the introduction of Ascendis Pharma's YUVIWEL.

The analyst maintained a Neutral rating on BioMarin shares along with a 12-month price target of US$50.

Using a discounted cash flow model with a 10% discount rate and a 1% terminal growth rate, Kapoor estimated BioMarin's enterprise value at about US$12.3 billion. The report said the company is expected to have roughly US$4.3 billion in debt after accounting for convertible debt following the closing of the Amicus transaction. Based on an estimated 192 million fully diluted shares outstanding and projected year-end 2026 cash and equivalents of about US$2.1 billion, the analyst arrived at the US$50 12-month price target.

The report said key risks include slower-than-expected commercial adoption of BioMarin's products, particularly VOXZOGO, delays in regulatory approvals for late-stage pipeline candidates, and the possibility of long-term shareholder dilution.

Co. Holds Advantage Over Competitors, Analyst Says

On May 21, Wedbush Analyst Yun Zhong wrote an updated note on the company, rating it Outperform with a US$94 per share price target.

The analyst said positive Phase 3 results in hypochondroplasia should allow BioMarin to expand the potential patient population for VOXZOGO and strengthen the therapy's leadership position in skeletal disorders. The report highlighted a placebo-adjusted increase of 2.33 centimeters per year in annualized growth velocity after 52 weeks, exceeding the improvement previously reported in the Phase 3 achondroplasia study.

The report suggested the successful trial could support development in additional skeletal disorders, which together could represent a commercial opportunity more than 10 times larger than the combined markets for achondroplasia and hypochondroplasia.

Zhong noted that hypochondroplasia may be more common than previously recognized because the condition is often underdiagnosed due to its relatively subtle clinical features. While earlier thinking suggested disproportionate short stature was the primary concern, unlike achondroplasia, which is associated with complications such as foramen magnum stenosis, sleep apnea, and spinal stenosis, the analyst cited a retrospective real-world matched cohort study conducted by BioMarin showing significantly higher rates of respiratory, cardiovascular, orthopedic, and mental health-related comorbidities among patients with hypochondroplasia. The report also noted that these patients may face an increased risk of temporal lobe epilepsy and learning disabilities.

The analyst said BioMarin currently holds an advantage over competitors in the hypochondroplasia market, although rival programs continue to advance.

The Catalyst: Diagnostic Methods More Accurate

The global skeletal dysplasia market size was estimated at US$3.2 billion in 2025 and is projected to reach US$6.9 billion by 2035, growing at a CAGR of 8.1% from 2026 to 2035, according to a report by Vantage Market Research.

The report said the market continues to gain momentum as healthcare providers improve recognition of rare genetic bone disorders, diagnostic methods become more accurate, and targeted therapies become increasingly integrated into specialized treatment programs.

According to the report, the sector combines elements of rare disease therapeutics and precision medicine, with advances in treatment, supportive regulatory policies, and long-term patient care strategies serving as the primary factors shaping its long-term growth and strategic significance.

"The skeletal dysplasia market occupies a highly specialized niche within the broader rare disease therapeutics ecosystem, characterized by low patient volumes but high clinical complexity and lifetime care requirements," the report said. "The market's role is defined by its integration across diagnosis, therapeutic intervention, and long-term disease management, where multidisciplinary care models are essential. Unlike high-prevalence therapeutic areas, this market operates under a precision-driven paradigm, where each intervention is closely aligned with genetic subtypes and disease severity."

This market reflects an evolving maturity profile, transitioning from symptomatic management toward disease-modifying approaches. The shift is driven by advances in genetic research and regulatory frameworks that incentivize orphan drug development. For CXOs, the market represents a convergence of scientific innovation and reimbursement complexity, where success depends on aligning clinical efficacy with payer acceptance. The strategic relevance lies in navigating fragmented patient populations while building scalable therapeutic and diagnostic solutions that can sustain long-term engagement. 

The report said the market is evolving beyond treatments that primarily manage symptoms and is increasingly focused on therapies designed to modify the underlying disease. It attributed this transition to advances in genetic research and regulatory policies that encourage the development of orphan drugs.

According to the report, the market presents both opportunities and challenges for healthcare executives by combining rapid scientific progress with increasingly complex reimbursement requirements. The report said long-term success will depend on demonstrating strong clinical outcomes that satisfy payers while developing scalable therapeutic and diagnostic platforms capable of serving fragmented patient populations over time.

Meanwhile, the global genetic disorders market size was US$15.95 billion in 2023 and is expected to surpass around US$50.61 billion by 2033, growing at a CAGR of 12.24% from 2024 to 2033, according to BioSpace. The genetic disease market is expected to grow with the identification of 6,000-8,000 rare diseases (RDs), of which approximately 80% are genetic and 50-75% initiate from early childhood.

Ownership and Share Information1

BioMarin Pharmaceutical Inc. has a market cap of CA$11.52 billion, with 193.28 million shares outstanding. The company's 52-week range is US$49.26-US$66.28.

Institutions own 95% of shares, while management and insiders own nearly 2% of shares, and retail owns the rest.

Common Investor Questions

What did the FDA do? It accepted BioMarin's supplemental New Drug Application seeking full (traditional) approval of VOXZOGO for children with achondroplasia.

When will the FDA decide? The PDUFA target action date is Feb. 28, 2027.

What is achondroplasia? The most common form of skeletal dysplasia, causing disproportionate short stature, is caused by an FGFR3 gene mutation. It occurs in about 1 in 25,000 births, and over 80% of cases arise spontaneously in children of average-height parents.

What's the market opportunity? The global skeletal dysplasia market is projected to grow from US$3.2B (2025) to US$6.9B by 2035 (8.1% CAGR).