BioMarin Shares New Data Reinforcing Its Leadership in Bone Health at the American Society for Bone and Mineral Research Annual Meeting

Children who received VOXZOGO showed anatomical improvements in spinal morphology

VOXZOGO continued to significantly improve growth in children who received the medicine after puberty onset

SAN RAFAEL, Calif., Sept. 8, 2025 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced new data from 14 studies were presented at the American Society for Bone and Mineral Research 2025 (ASBMR) Annual Meeting in Seattle, including results demonstrating treatment with VOXZOGO^® (vosoritide) led to anatomical improvements in spinal morphology in young children, and continued efficacy in children who received the medicine after the onset of puberty.

VOXZOGO Demonstrated Positive Impact on Spinal Morphology

An analysis of data from the randomized, double-blind, placebo-controlled Phase 2 CANOPY clinical study (111-206) in children ages 5 and under with achondroplasia examined the impact of VOXZOGO on spinal morphology, one of the factors that can lead to spinal stenosis, a serious complication of the condition where clinical evidence is limited. Spinal stenosis often results in narrowing of the spinal canal and additional pressure on the spinal cord and nerve roots, resulting in a host of symptoms in children, including pain, numbness, difficulty walking and bladder issues.

Researchers found that children (n=40) who received VOXZOGO for 52 weeks experienced measurable improvement in interpedicular distance (IPD) and greater spinal canal width in all five vertebrae in the lower back (L1 through L5) compared to children who received placebo (n=27). Children who received VOXZOGO (57%) in this study also demonstrated a reduction in thoracolumbar kyphosis (TLK), or excessive curvature of the spine, compared to those who received placebo (33%).

"The impact of achondroplasia on the spine can lead to great discomfort and, in some cases, disability in children living with the condition, with symptoms persisting well into adulthood," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "VOXZOGO is the only approved medicine with data supporting a positive impact on spinal morphology, which could potentially lead to clinical improvements in painful characteristics associated with achondroplasia, such as spinal stenosis. Today's research adds to the immense body of evidence demonstrating how VOXZOGO's benefit can extend beyond improving growth and may reduce the significant complications associated with achondroplasia in children."

Results from the open-label, Phase 3 CANOPY long-term extension study (111-302) examined whether teenagers with achondroplasia continued to benefit from the medicine after puberty onset compared to untreated children. In young men (mean age of puberty onset = 12.1 years) who received VOXZOGO (n=33), researchers found that the participants experienced 24.62 cm of growth from the mean age of puberty onset until age 18 compared to 17.07 cm in untreated individuals, a difference of 7.55 cm. For young women (mean age of puberty onset = 10.7 years), growth from the mean age of puberty until age 16 was 21.20 cm compared to 13.13 cm in untreated individuals, a difference of 8.07 cm. VOXZOGO is approved in children of all ages with achondroplasia until their growth plates close, which typically occurs several years after the onset of puberty.

Observational Studies Reinforce Unmet Need in ENPP1 Deficiency

BioMarin presented research from four studies reflecting its leadership in deepening the scientific understanding of and highlighting the unmet medical needs for people with ENPP1 deficiency, a rare, serious and progressive genetic condition that affects blood vessels, soft tissues and bones. BioMarin is advancing BMN 401, a potential first-in-disease enzyme therapy for the condition. Initial pivotal data readout for the ENERGY 3 study in children ages 1-12 years with ENPP1 deficiency is anticipated in the first half of 2026, with potential launch in 2027.

Below are key presentations for BioMarin (all times in Pacific Time):

VOXZOGO and Skeletal Conditions

Effect of Long-Term Vosoritide Treatment in Pediatric Participants With Achondroplasia on Bone Mineral Density and Bone Content: Results From Quantitative Computed Tomography Analyses
Oral Presentation (pre-meeting)
Thursday, Sept. 4, 9:25 a.m.

Poster #Sat-572
Saturday, Sept. 6, 2 – 3:30 p.m.

Comprehensive Analysis of FGFR3 Variants Identified >10 Candidate Hypochondroplasia Variants with Potential to Increase Diagnostic Yield
Plenary Poster #Fri-506
Friday, Sept. 5, 5 – 6:30 p.m.

Poster #Sun-506
Sunday, Sept. 7, 2 – 3:30 p.m.

C-Type Natriuretic Peptide (CNP) Agonist CNP38 Improves Growth in the Hyp Mouse Model of XLH
Plenary Poster #Fri-266
Friday, Sept. 5, 5 – 6:30 p.m.

Poster #Sat-266
Saturday, Sept. 6, 2 – 3:30 p.m.

Continued Growth After Puberty in Participants With Achondroplasia Treated With Vosoritide in a Phase 3 Long-Term Extension Trial
Poster #Sat-555
Saturday, Sept. 6, 2 – 3:30 p.m.

CNP Agonism: A Novel Anabolic Approach for Osteoporosis
Poster #Sat-580
Saturday, Sept. 6, 2 – 3:30 p.m.

Vosoritide, a C-Type Natriuretic Peptide (CNP) Analog, Alone and in Combination with Alendronate Improves Incidence of New Fractures and Long Bone Growth in the OIM Mouse Model for Osteogenesis Imperfecta: A Promising Therapeutic Approach
Poster #Sat-297
Saturday, Sept. 6, 2 – 3:30 p.m.

Criteria for Defining Inadequate Response to Growth Hormone in Noonan Syndrome, Turner Syndrome, and SHOX Deficiency
Poster #Sun-504
Sunday, Sept. 7, 2 – 3:30 p.m.

Fracture Incidence is Reduced and Bone Microarchitecure is Improved by CNP Analog Adjuvant Therapy in Young Mice with Moderate-to-Severe Osteogenesis Imperfecta
Poster #Sun-542
Sunday, Sept. 7, 2 – 3:30 p.m.

Effect of Vosoritide on Spine Morphology in Young Children With Achondroplasia: 1-Year Results From a Double-Blind, Randomized Phase 2 Study
Poster #Sun-555
Sunday, Sept. 7, 2 – 3:30 p.m.

Molecular Insights into C-Type Natriuretic Peptides Role in Enhancing Bone Growth and Mineralization
Poster #Sun-133
Sunday, Sept. 7, 2 – 3:30 p.m.

ENPP1 Deficiency

Accelerating Research and Development for Rare Bone Conditions in Europe: A Multistakeholder Call to Action
Poster #Sat-583 (late-breaking)
Saturday, Sept. 6, 2 – 3:30 p.m.

Patient and Caregiver Insights on the Real-World Burden of the Rare Genetic Disorders, ENPP1 Deficiency and ABCC6 Deficiency
Poster #Sat-352
Saturday, Sept. 6, 2 – 3:30 p.m.

Plasma Pyrophosphate (PPi) Levels Correlate with Severity of Clinical Manifestations in a Mouse Model of ENPP1 Deficiency
Poster #Sun-298
Sunday, Sept. 7, 2 – 3:30 p.m.

Qualitative Evaluation of Hypophosphatemic Rickets Due to ENPP1 Deficiency
Poster #Sun-352
Sunday, Sept. 7, 2 – 3:30 p.m.

About Achondroplasia

Achondroplasia, the most common form of skeletal dysplasia, is characterized by impaired bone growth caused by a change in the FGFR3 gene. Bone growth is regulated by multiple biological processes including signaling pathways through fibroblast growth factor (FGF, which slows bone growth) and C-type natriuretic peptide (CNP, which increases bone growth). In achondroplasia, these signals are out of balance, resulting in a slowing of endochondral ossification, and causing disproportionate short stature and disordered architecture in the long bones, spine, face, and base of the skull.

More than 80% of children with achondroplasia have parents of average stature and have the condition as a result of a spontaneous gene mutation. The worldwide incidence rate of achondroplasia is about one in 25,000 live births. VOXZOGO is being evaluated in children whose growth plates are still "open," typically those under 18 years of age. Approximately 25% of people with achondroplasia fall into this category.

About ENPP1 Deficiency

ENPP1 deficiency is a rare genetic condition caused by changes in the ENPP1 gene, leading to progressive damage to blood vessels, soft tissues and bones. Infants with this condition are often diagnosed with generalized arterial calcification of infancy (GACI) Type 1, and about 50% of them do not survive beyond six months. Children with ENPP1 deficiency typically develop a type of rickets called autosomal-recessive hypophosphatemic rickets type 2 (ARHR2), while adolescents and adults may develop osteomalacia (softened bones), both of which may cause pain and difficulty with movement. Individuals may also experience hearing loss, calcification in arteries and joints, and heart problems.

BMN 401 (formerly INZ-701) is a potential first-in-disease treatment for ENPP1 deficiency, and initial pivotal data from the ENERGY 3 study in children ages 1-12 years is anticipated in the first half of 2026, with potential launch in 2027.

About VOXZOGO

In children with achondroplasia, endochondral bone growth, an essential process by which bone tissue is created, is negatively regulated due to a gain of function mutation in FGFR3. VOXZOGO, a C-type natriuretic peptide (CNP) analog, acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.

VOXZOGO is approved in the U.S., Japan and Australia to increase linear growth in children of all ages with achondroplasia with open epiphyses, and VOXZOGO is indicated in the EU for the treatment of achondroplasia in children 4 months of age and older whose epiphyses are not closed, as confirmed by appropriate genetic testing. In the U.S., this indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trial(s). To fulfill this post-marketing requirement, BioMarin intends to use the ongoing open-label extension studies compared to available natural history.

Patient Support Accessing VOXZOGO

To reach a BioMarin RareConnections^® Case Manager, please call, toll-free, 1-833-VOXZOGO (1-833-869-9646) or e-mail [email protected]. For more information about VOXZOGO, please visit www.voxzogo.com. For additional information regarding this product, please contact BioMarin Medical Information at [email protected].

VOXZOGO U.S. Important Safety Information

What is VOXZOGO used for?

• VOXZOGO is a prescription medicine used to increase linear growth in children with achondroplasia and open growth plates (epiphyses).
• VOXZOGO is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials.

What is the most important safety information about VOXZOGO?

• VOXZOGO may cause serious side effects including a temporary decrease in blood pressure in some patients. To reduce the risk of a decrease in blood pressure and associated symptoms (dizziness, feeling tired, or nausea), patients should eat a meal and drink 8 to 10 ounces of fluid within 1 hour before receiving VOXZOGO.

What are the most common side effects of VOXZOGO?

• The most common side effects of VOXZOGO include injection site reactions (including redness, itching, swelling, bruising, rash, hives, and injection site pain), high levels of blood alkaline phosphatase shown in blood tests, vomiting, joint pain, decreased blood pressure, and stomachache. These are not all the possible side effects of VOXZOGO. Ask your healthcare provider for medical advice about side effects, and about any side effects that bother the patient or that do not go away.

How is VOXZOGO taken?

• VOXZOGO is taken daily as an injection given under the skin, administered by a caregiver after a healthcare provider determines the caregiver is able to administer VOXZOGO. Do not try to inject VOXZOGO until you have been shown the right way by your healthcare provider. VOXZOGO is supplied with Instructions for Use that describe the steps for preparing, injecting, and disposing VOXZOGO. Caregivers should review the Instructions for Use for guidance and any time they receive a refill of VOXZOGO in case any changes have been made.
• Inject VOXZOGO 1 time every day, at about the same time each day. If a dose of VOXZOGO is missed, it can be given within 12 hours from the missed dose. After 12 hours, skip the missed dose and administer the next daily dose as usual.
• The dose of VOXZOGO is based on body weight. Your healthcare provider will adjust the dose based on changes in weight following regular check-ups.
• Your healthcare provider will monitor the patient's growth and tell you when to stop taking VOXZOGO if they determine the patient is no longer able to grow. Stop administering VOXZOGO if instructed by your healthcare provider.

What should you tell the doctor before or during taking VOXZOGO?

• Tell your doctor about all of the patient's medical conditions including
  • If the patient has heart disease (cardiac or vascular disease), or if the patient is on blood pressure medicine (anti-hypertensive medicine).
  • If the patient has kidney problems or renal impairment.
  • If the patient is pregnant or plans to become pregnant. It is not known if VOXZOGO will harm the unborn baby.
  • If the patient is breastfeeding or plans to breastfeed. It is not known if VOXZOGO passes into breast milk.
• Tell your doctor about all of the medicines the patient takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

You may report side effects to BioMarin at 1-866-906-6100. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Please see additional safety information in the full Prescribing Information and Patient Information.

About BioMarin

BioMarin is a global biotechnology company dedicated to translating the promise of genetic discovery into medicines that make a profound impact on the life of each patient. The San Rafael, California-based company, founded in 1997, has a proven track record of innovation with eight commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that offer new possibilities for people living with genetically defined conditions around the world. To learn more, please visit www.biomarin.com.

Forward-Looking Statements

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: data presented at the American Society for Bone and Mineral Research (ASBMR) 2025 Annual Meeting, including the oral and poster presentations; VOXZOGO's efficacy, safety and impact on children with achondroplasia, including the potential positive impact on spinal morphology, ability to significantly improve growth in children who receive the medicine after puberty onset, and the potential to reduce the significant complications associated with achondroplasia in children; VOXZOGO's potential benefits, safety and impact on children with hypochondroplasia, Noonan syndrome, Turner syndrome and SHOX deficiency; and BioMarin's plans and expectations for the development of BMN 401, including the expected initial pivotal data readout for the ENERGY 3 study in children ages 1-12 years with ENPP1 deficiency in the first half of 2026, with potential launch in 2027. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results and timing of current and planned pre-clinical studies and clinical trials of VOXZOGO and BMN 401; any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the U.S. Food and Drug Administration, the European Medicines Agency, the European Commission and other regulatory authorities; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, as such factors may be updated by any subsequent reports. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin^®, BioMarin RareConnections^® and VOXZOGO^® are registered trademarks of BioMarin Pharmaceutical Inc.

SOURCE BioMarin Pharmaceutical Inc.