BioMarin Presents New Data on the Effect of Long-Term Treatment with VOXZOGO® (vosoritide) on Arm Span, Bone Health and Growth in Children With Achondroplasia at the Pediatric Endocrine Society's 2026 Annual Meeting

Rhea-AI Summary

BioMarin (Nasdaq: BMRN) presented data at PES 2026 showing long-term VOXZOGO (vosoritide) treatment improved arm span Z-scores, maintained arm span-to-height proportionality, and preserved bone mineral density while increasing bone mineral content in children with achondroplasia.

Children starting treatment after age 5 had mean standing height gains of 10.60 cm at six years and 13.59 cm at eight years versus untreated cohorts; topline Phase 3 hypochondroplasia results are expected in 1H 2026.

Positive

• Arm span Z-scores improved from baseline across all age groups
• Arm span-to-height ratio remained stable, indicating proportional skeletal growth
• Standing height mean differences versus natural history: +10.60 cm at six years and +13.59 cm at eight years for treatment initiated after age 5
• Bone mineral content (BMC) increased over up to six years of treatment while BMD Z-scores remained consistent

Negative

• Topline Phase 3 results for hypochondroplasia are pending in 1H 2026, creating near-term clinical readout uncertainty
• Key long-term findings compare treated cohorts to untreated natural history cohorts rather than randomized concurrent controls

Market Context

This announcement highlights extensive VOXZOGO experience, with more than 10 years of research and l...

Analysis

This announcement highlights extensive VOXZOGO experience, with more than 10 years of research and long-term data showing proportional growth and maintained bone health in achondroplasia. New hypochondroplasia findings, including 12‑month improvements in BMD and BMC with strong statistical significance (p<0.0001), broaden the program’s potential. In context of recent approvals and the Amicus acquisition, investors may watch upcoming Phase 3 topline results and how VOXZOGO’s label and real-world uptake evolve.

Key Figures

Height gain after 6 years: 10.60 cm Height gain after 8 years: 13.59 cm Study size: 119 children +5 more

8 metrics

Height gain after 6 years 10.60 cm Mean difference in standing height vs untreated cohorts after six years on VOXZOGO

Height gain after 8 years 13.59 cm Mean difference in standing height vs untreated cohorts after eight years on VOXZOGO

Study size 119 children Children receiving VOXZOGO with annual DXA bone assessments up to six years

BMD improvement 0.03 g/cm2 Increase in total body minus head BMD after 12 months in hypochondroplasia Phase 2 study

BMC increase 54.84 g Increase in total body minus head BMC after 12 months in hypochondroplasia Phase 2 study

P-value p<0.0001 Statistical significance for VOXZOGO height gains and hypochondroplasia bone measures

Clinical research duration more than 10 years Years of VOXZOGO clinical research referenced in achondroplasia program

Treatment duration 12 months Prospective hypochondroplasia study measuring bone content and density

Historical Context

5 past events · Latest: Apr 27 (Positive)

Pattern 5 events

Date	Event	Sentiment	24h Move	Catalyst
Apr 27	Acquisition completion	Positive	+1.2%	Closed all-cash Amicus deal at $14.50 per share (~$4.8B value).
Apr 27	Earnings call notice	Neutral	+1.2%	Announced timing of Q1 2026 results call and business update.
Apr 21	Board transition	Neutral	-1.2%	Named Ian T. Clark as incoming Chair following June 2 meeting.
Mar 12	VOXZOGO data update	Positive	-2.1%	Early VOXZOGO treatment improved proportionality and arm span with multi‑year gains.
Feb 27	FDA approval	Positive	-3.2%	PALYNZIQ approved for adolescents 12+ with PKU based on Phase 3 data.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Pattern Detected

Recent history shows that even clearly positive clinical and regulatory updates (VOXZOGO data, FDA approval) sometimes coincided with negative price moves, while the large Amicus acquisition drew a modestly positive reaction.

Recent Company History

Over the past few months, BioMarin reported several major developments. On Feb 27, 2026, PALYNZIQ gained FDA approval, followed by new VOXZOGO growth data on Mar 12. Governance changes came with a Board leadership transition on Apr 21. In late April, the company completed a $4.8 billion all-cash acquisition of Amicus Therapeutics and scheduled its Q1 2026 earnings call for May 4, 2026. Today’s VOXZOGO long-term efficacy data extend this stream of product-focused updates.

Key Terms

achondroplasia, hypochondroplasia, bone mineral content, bone mineral density, +4 more

8 terms

achondroplasia medical

"VOXZOGO is the only approved treatment for children with achondroplasia starting at birth"

A genetic condition that causes the most common form of short stature, where a specific change in a growth-control gene makes the long bones grow more slowly, producing a distinct body proportion and sometimes breathing, spinal or joint issues. It matters to investors because it defines a clear patient population, predictable medical needs and regulatory pathways for drugs or devices—similar to a niche market with steady, long-term demand for effective treatments or supportive care.

hypochondroplasia medical

"additional data from studies of VOXZOGO for hypochondroplasia, ahead of pivotal topline Phase 3 data"

A genetic growth disorder that causes shorter-than-average limbs and reduced adult height by affecting bone development; think of it as a change in the body’s blueprint that slows or limits normal bone lengthening. For investors, hypochondroplasia matters because it defines a clear patient group and medical need that can drive demand for diagnostics, therapies, or supportive services, shaping market size, clinical development paths, and regulatory attention in related biotech and healthcare companies.

bone mineral content medical

"impact of long-term treatment on bone mineral content (BMC) and bone mineral density (BMD)"

Bone mineral content is the amount of mineral, mainly calcium and phosphorus, contained in a bone and is usually measured by a medical scan to show how dense or strong the bone is. Investors care because changes in this measure indicate whether drugs, medical devices, or supplements intended to prevent or treat bone loss are working, which influences product approval, market demand, and a company’s revenue prospects — think of it as the ‘weight’ that keeps a building’s support pillars solid.

bone mineral density medical

"impact of long-term treatment on bone mineral content (BMC) and bone mineral density (BMD)"

A measure of how much mineral — mainly calcium — is packed into a given area of bone, usually obtained from a painless scan; higher values generally mean stronger bones and lower fracture risk. Investors care because changes in this measure are often used as proof that drugs, medical devices, or diagnostics work, so it can drive clinical approval, insurance coverage and sales prospects much like a car’s crash-test rating influences buyer confidence and market demand.

dual X-ray absorptiometry medical

"BMC and bone mineral density (BMD) assessed by dual X-ray absorptiometry (DXA) every year"

A dual x-ray absorptiometry (DXA) scan is a low-dose imaging test that measures bone density by passing two X-ray beams through the body to estimate how much mineral is in the bones. Like weighing the structural parts of a building to judge strength, it helps diagnose osteoporosis and fracture risk; for investors, DXA use and sales signal demand for medical devices, screening services, related drugs, and follow-on healthcare revenue streams.

Phase 2 medical

"Design of a Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate the Safety"

Phase 2 is the mid-stage clinical trial where a new drug or treatment is tested in a larger group of patients to see if it works and to keep checking safety after initial human testing. Think of it as a field test that proves whether a product actually delivers its promised benefit. Investors watch Phase 2 closely because its results strongly influence a medicine’s chances of reaching the market, the size of its potential sales, and the company’s valuation.

Phase 3 medical

"registration-enabling Phase 3 pivotal clinical trial of VOXZOGO in children with hypochondroplasia"

Phase 3 is the late-stage clinical testing step for a new drug or medical treatment, where the product is given to large groups of patients to confirm effectiveness, monitor side effects, and compare it to standard care. Successful Phase 3 results are often the final scientific hurdle before regulators decide on approval and market launch—like passing a final exam before graduation—and can sharply change a company's valuation and future revenue prospects.

pivotal clinical trial medical

"registration-enabling Phase 3 pivotal clinical trial of VOXZOGO in children with hypochondroplasia"

A pivotal clinical trial is a late-stage medical study designed to provide the main evidence regulators use to decide whether a drug or medical device can be approved and sold; think of it as the product’s final exam. For investors, the trial’s outcome is critical because a clear pass can unlock regulatory approval, market access and future revenue, while failure can halt commercialization and materially affect a company’s valuation.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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VOXZOGO is the only approved treatment for children with achondroplasia starting at birth, with over 10 years of clinical research demonstrating the long-term benefit on complications associated with achondroplasia

Researchers will present additional data from studies of VOXZOGO for hypochondroplasia, ahead of pivotal topline Phase 3 data expected in the first half of 2026

SAN RAFAEL, Calif., May 2, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced new research from studies of VOXZOGO^® (vosoritide) in children with achondroplasia demonstrating positive impact on important health indicators, including arm span and bone density. The data will be presented, along with new data from studies of VOXZOGO in hypochondroplasia, at the Pediatric Endocrine Society's 2026 Annual Meeting (PES) in San Francisco.

Long-Term Treatment Leads to Meaningful Improvement in Multiple Health Measures

Data from three ongoing long-term extension clinical trials demonstrated the impact of long-term treatment with VOXZOGO on measures beyond height, including arm span and bone health. Researchers showed that arm span Z-scores improved from baseline in all age groups, and the arm span-to-height ratio also remained stable over time, showing treatment resulted in proportional skeletal growth. Children who initiated treatment with VOXZOGO after age 5 also achieved a mean difference in standing height of 10.60 cm after six years of treatment (p<0.0001) and 13.59 cm after eight years of treatment (p<0.0001), compared with untreated natural history cohorts.

"With VOXZOGO, we now have a depth and duration of evidence that is unmatched in the treatment of achondroplasia — providing meaningful insight not only into growth, but into the broader, sustained impact on a child's health," said Bradley Miller, M.D., Ph.D., pediatric endocrinologist at the University of Minnesota Medical School. "When you can see consistent benefits over time, it gives you greater confidence to intervene early and treat with purpose."

Another study with 119 children who received VOXZOGO measured the impact of long-term treatment on bone mineral content (BMC) and bone mineral density (BMD) assessed by dual X-ray absorptiometry (DXA) every year for up to six years. Results showed that BMC increased over time, while BMD Z‑scores remained consistent year over year, demonstrating that bone health was maintained in children who received long-term VOXZOGO treatment.

"We are committed to understanding and sharing the clear impact of long-term treatment with VOXZOGO on health measures beyond height, including arm span and bone density, that are meaningful to the thousands of children around the world receiving this medicine," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "With more than ten years of clinical research now, we have seen again and again a breadth of data that continue to demonstrate that long-term and early treatment are critical to ensure the greatest benefit in children with achondroplasia."

New Research in Hypochondroplasia; Topline Phase 3 Results Expected in 1H 2026

Two studies focused on VOXZOGO in hypochondroplasia will also be presented, including one from a single-arm Phase 2 study conducted by Children's National Hospital that showed a statistically significant improvement in total body minus head BMD of 0.03 g/cm² (p<0.0001) and BMC of 54.84 g (p<0.0001) after 12 months in children who received the medicine.

The company plans to share topline results from its registration-enabling Phase 3 pivotal clinical trial of VOXZOGO in children with hypochondroplasia (CANOPY-HCH-3) in the first half of 2026. If the clinical results are positive, the company plans to submit data to health authorities in the second half of 2026 to seek approval for this new indication.

Below are key poster presentations for achondroplasia and hypochondroplasia at PES, with all times listed in Pacific Daylight Time:

Design of a Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate the Safety and Efficacy of Vosoritide in Infants and Children With Hypochondroplasia Aged <3 Years
Poster #12, Poster Session 2
Friday, May 1, 7:15 – 8:15 a.m.

Effect of Long-Term Vosoritide Treatment in Pediatric Participants With Achondroplasia on Bone Mineral Density and Bone Mineral Content Measured with Dual X-Ray Absorptiometry
Poster #16, Poster Session 3
Friday, May 1, 12:30 – 2 p.m.

Real-World Long-Term Effectiveness and Safety Outcomes of Vosoritide in Adolescents With Achondroplasia in Japan
Poster #58, Poster Session 3
Friday, May 1, 12:30 – 2 p.m.

Vosoritide Treatment Improves Linear Growth and Absolute Bone Content and Density in Children With Hypochondroplasia: A 12-Month Prospective Study
Poster #40, Poster Session 3
Friday, May 1, 12:30 – 2 p.m.

Effect of Long-Term Vosoritide Treatment on Growth in Children With Achondroplasia in Open-Label, Multicenter Clinical Trials
Poster #32, Poster Session 5
Saturday, May 2, 12:30 – 2 p.m.

Improving Guideline-Directed Management of Achondroplasia: Results of a Pre-Implementation Study
Poster #12, Poster Session 5
Saturday, May 2, 12:30 – 2 p.m.

About Achondroplasia

Achondroplasia, the most common form of skeletal dysplasia leading to disproportionate short stature in humans, is characterized by slowing of endochondral ossification, which results in disproportionate short stature and disordered architecture in the long bones, spine, face and base of the skull. This condition is caused by a change in the FGFR3 gene, a negative regulator of bone growth.

More than 80% of children with achondroplasia have parents of average stature and have the condition as the result of a spontaneous gene mutation. The worldwide incidence rate of achondroplasia is about one in 25,000 live births. VOXZOGO is being tested 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.

For more information about our clinical trials in achondroplasia, hypochondroplasia and other skeletal conditions, please visit clinicaltrials.biomarin.com.

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 the only approved medicine to support the growth of children with achondroplasia starting from birth, with international consensus guidelines recommending initiation of VOXZOGO as early as possible. First approved in 2021, VOXZOGO has helped more than 5,000 infants and children in more than 50 countries. Through our ongoing studies, BioMarin continues to evaluate VOXZOGO on key clinical endpoints relevant for achondroplasia patients, such as arm span, tibial bowing (leg bowing), body proportionality, spinal morphology (including spinal stenosis) and quality of life measures.

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

BioMarin's robust support services have ensured a seamless treatment experience, spearheaded by Clinical Coordinators, who have conducted hundreds of trainings for families with achondroplasia since approval. BioMarin provides resources to support families navigating achondroplasia, including a caregiver mentorship program that connects parents with other caregivers, and a U.S. doctor directory that helps families and healthcare professionals identify clinicians experienced in achondroplasia care.

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

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 leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with a portfolio of 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 have a profound impact on patients. 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: the data to be presented at the Pediatric Endocrine Society's 2026 Annual Meeting, including the key poster presentations; the development of BioMarin's VOXZOGO program, including plans to share topline results from the Phase 3 pivotal clinical trial of VOXZOGO in children with hypochondroplasia (CANOPY-HCH-3) in the first half of 2026 and to submit data to health authorities in the second half of 2026 if such results are positive; the safety profile and potential benefits of VOXZOGO for children with achondroplasia and hypochondroplasia; and the continued clinical development of VOXZOGO in multiple indications. 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, 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 (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Annual Report on Form 10-K for the year ended December 31, 2025, as such factors may be updated by any subsequent filings with the SEC. 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.

Contacts:
Investors	Media
Traci McCarty	Andrew Villani
BioMarin Pharmaceutical Inc.	BioMarin Pharmaceutical Inc.
(415) 455-7558	(628) 269-7393

 

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SOURCE BioMarin Pharmaceutical Inc.

FAQ

What long-term growth benefits did BioMarin (BMRN) report for VOXZOGO in achondroplasia?

VOXZOGO produced meaningful height gains: +10.60 cm at six years and +13.59 cm at eight years for children who began after age 5. According to BioMarin, those figures compare treated participants to untreated natural history cohorts, measured in long-term extension trials.

How did VOXZOGO affect arm span and proportional growth in children with achondroplasia (BMRN)?

Arm span Z-scores improved from baseline across all age groups and arm span-to-height ratio remained stable. According to BioMarin, this indicates treatment resulted in proportional skeletal growth beyond gains in standing height.

What were the bone health findings for long-term VOXZOGO treatment reported by BioMarin (BMRN)?

Long-term data showed bone mineral content increased over time while bone mineral density Z-scores remained consistent. According to BioMarin, DXA assessments annually up to six years demonstrated maintained bone health with continued treatment.

When will BioMarin (BMRN) report pivotal Phase 3 topline results for VOXZOGO in hypochondroplasia?

BioMarin expects to share topline Phase 3 results for hypochondroplasia in the first half of 2026. According to BioMarin, a positive outcome would lead to submission of data to health authorities in the second half of 2026.

What hypochondroplasia clinical signals did BioMarin present ahead of the Phase 3 readout (BMRN)?

A single-arm Phase 2 study reported statistically significant increases in total body minus head BMD (+0.03 g/cm2) and BMC (+54.84 g) after 12 months. According to BioMarin, these findings support ongoing pivotal evaluation in CANOPY-HCH-3.