BioMarin Announces Positive Phase 3 Pivotal Study Results for VOXZOGO® (vosoritide) in Children with Hypochondroplasia

Rhea-AI Summary

BioMarin (Nasdaq:BMRN) reported positive Phase 3 CANOPY-HCH-3 results for VOXZOGO (vosoritide) in children with hypochondroplasia. The study met its primary endpoint, showing an LS mean difference in annualized growth velocity of +2.33 cm/year versus placebo at week 52 (p<0.0001).

VOXZOGO also led to statistically significant gains in standing height, height Z-score (both p<0.0001) and arm span (p=0.004). Safety was consistent with the established achondroplasia profile, with no new safety signals. A sNDA to the FDA is planned for Q3 2026, followed by EMA and other global filings.

Positive

• Primary endpoint met with LS mean AGV difference of +2.33 cm/year vs placebo
• Statistically significant improvements in standing height and height Z-score at week 52
• Arm span, a key functional endpoint, improved significantly (p=0.004)
• Safety profile aligned with prior achondroplasia experience; no new safety signals
• sNDA submission to FDA planned for third quarter 2026

Negative

• None.

News Market Reaction – BMRN

+7.77%

26 alerts

+7.77% Session close to close

+3.7% Peak in 20 hr 58 min

$10.52B Market Cap

1.1x Rel. Volume

In the May 21 session, BMRN gained 7.77%, reflecting a notable positive market reaction. Argus tracked a peak move of +3.7% during that session. Our momentum scanner triggered 26 alerts that day, indicating elevated trading interest and price volatility.

Data tracked by StockTitan Argus on the day of publication.

Market Context

The stock moved +7.8% in the session following this news. A strong positive reaction aligns with the...

Analysis

The stock moved +7.8% in the session following this news. A strong positive reaction aligns with the magnitude of the VOXZOGO Phase 3 data, which showed an AGV benefit of 2.33 cm/yr and multiple endpoints at p<0.0001. Historically, BioMarin’s clinical wins often produced modest or even negative moves, so a larger gain would mark a break from the average -1.73% response to similar news and could still be sensitive to broader pipeline or balance sheet concerns.

Key Figures

AGV improvement: 2.33 cm/yr AGV p-value: p<0.0001 Standing height p-value: p<0.0001 +5 more

8 metrics

AGV improvement 2.33 cm/yr LS mean difference vs placebo at week 52

AGV p-value p<0.0001 Primary endpoint annualized growth velocity at week 52

Standing height p-value p<0.0001 Change in standing height vs placebo after one year

Height Z-score p-value p<0.0001 Change in height Z-score vs placebo after one year

Arm span p-value p=0.004 Improvement in arm span vs placebo at week 52

Study duration 52 weeks CANOPY-HCH-3 Phase 3 assessment period

Planned sNDA timing Q3 2026 Target for U.S. FDA supplemental NDA submission

Publication date May 20, 2026 Press release announcement of Phase 3 VOXZOGO results

Previous Clinical trial Reports

5 past events · Latest: May 18 (Negative)

Same Type Pattern 5 events

Date	Event	Sentiment	24h Move	Catalyst
May 18	Phase 3 update	Negative	-4.1%	BMN 401 met biomarker endpoint but missed key clinical measures.
Oct 29	Regulatory filing	Positive	-3.0%	FDA accepted PALYNZIQ sBLA for priority review in adolescents.
Sep 06	Pivotal trial data	Positive	-2.4%	PALYNZIQ Phase 3 showed large Phe reductions and strong response rates.
Jun 24	Long-term data	Positive	+1.1%	Five-year ROCTAVIAN data confirmed durable efficacy and safety.
Jun 07	Long-term data	Positive	-0.2%	Four-year ROCTAVIAN Phase 3 results reinforced bleed control durability.

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

Pattern Detected

Clinical news has often been strong fundamentally but met with muted or negative price reactions; most prior clinical updates saw small declines despite positive data.

Recent Company History

Over the past year, BioMarin has repeatedly reported late-stage clinical data across multiple programs, including PALYNZIQ and ROCTAVIAN, and most updates showed statistically significant efficacy with acceptable safety. Yet, same‑tag clinical headlines often coincided with modest share price declines, with an average move of about -1.73%. The latest VOXZOGO hypochondroplasia Phase 3 success continues the pattern of strong rare‑disease data, now focused on growth outcomes in children.

Key Terms

phase 3, pivotal, annualized growth velocity, height z-score, +4 more

8 terms

phase 3 medical

"BioMarin today announced that the Phase 3 CANOPY-HCH-3 study evaluating..."

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 medical

"BioMarin Announces Positive Phase 3 Pivotal Study Results for VOXZOGO..."

Pivotal describes a test, event, or decision that is decisive for whether a product, idea, or plan moves forward—most often used for a late-stage clinical trial or a regulatory review whose result determines approval or rejection. Investors care because a positive outcome typically unlocks major value (like market access or sales), while a negative one can sharply reduce expected future revenue; think of it as the final exam that decides whether a project passes or fails.

annualized growth velocity medical

"improvement in annualized growth velocity (AGV, change from baseline)..."

Annualized growth velocity is a way of expressing how fast a financial metric (such as revenue, earnings, or user base) would grow over a full year based on growth observed during a shorter period. Investors use it to compare performance across companies or timeframes and to project future size; think of timing how far a car travels in one minute and converting that to a miles‑per‑hour estimate to understand overall speed.

height z-score medical

"statistically significant increases in standing height and height Z-score..."

A height z-score is a standardized number that shows how an individual’s height compares to a reference population, measured in units of standard deviation. Think of it like saying someone is two “steps” above or below the average height for their age and sex. Investors care because it’s a common, objective measure used in clinical trials, product labeling and patient eligibility for growth-related therapies, so changes in z-scores can affect regulatory decisions, market size estimates and treatment value.

statistically significant technical

"demonstrating a statistically significant increase in the change from baseline..."

"Statistically significant" means that a result or difference observed in data is unlikely to have occurred by chance alone, suggesting there is a real underlying effect. For investors, it indicates that the findings or patterns they see are likely meaningful and not just random noise, helping them make more informed decisions based on reliable information. Think of it as a pattern that is strong enough to stand out clearly from background randomness.

supplemental new drug application regulatory

"Supplemental New Drug Application (sNDA) submission to U.S. Food and Drug..."

A supplemental new drug application is a request submitted to regulatory authorities to make changes to an existing approved medication, such as adding new uses, strengths, or formulations. For investors, it signals that a pharmaceutical company is seeking approval for new product developments or expanded applications, which can impact the company's future sales, market potential, and stock value.

u.s. food and drug administration regulatory

"sNDA submission to U.S. Food and Drug Administration (FDA) planned..."

The U.S. Food and Drug Administration is the federal agency that evaluates and enforces safety, effectiveness and labeling standards for medicines, medical devices, vaccines, food and related products before they reach consumers. For investors it matters because FDA approvals, warnings or recalls determine whether a product can be sold, how quickly it reaches the market and how costly compliance will be—changes that directly affect a company’s revenue, costs and stock value.

european medicines agency regulatory

"followed by submissions to the European Medicines Agency (EMA)..."

The European Medicines Agency is the central drug regulator that evaluates and authorizes medicines for use across the European Union and related countries, similar to a referee or safety inspector who checks that a medicine is safe and effective before it can be sold. Its decisions matter to investors because approvals, rejections, or safety warnings directly affect a drug maker’s ability to sell products, generate revenue, and face legal or reputational risks, which in turn influence stock value.

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

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Study met primary endpoint, exceeding expectations with a highly statistically significant improvement in annualized growth velocity (AGV, change from baseline) of 2.33 cm/yr compared to placebo at week 52, as well as statistically significant increases in standing height and height Z-score

Study demonstrated statistically significant improvement in arm span at week 52 compared to placebo, a key measure linked to functional independence

Safety findings consistent with the established profile in achondroplasia with no new safety signals identified

Supplemental New Drug Application (sNDA) submission to U.S. Food and Drug Administration (FDA) planned for third quarter, followed by submissions to the European Medicines Agency (EMA) and other regional health authorities

 Full data to be presented at an upcoming medical meeting 

SAN RAFAEL, Calif., May 20, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the Phase 3 CANOPY-HCH-3 study evaluating treatment with VOXZOGO^® (vosoritide) in children with hypochondroplasia met its primary endpoint, demonstrating a statistically significant increase in the change from baseline at week 52 in annualized growth velocity (AGV) compared to placebo (LS mean difference +2.33 cm/yr, p<0.0001). Children who received VOXZOGO also showed a statistically significant increase in standing height (p<0.0001) and height Z-score (p<0.0001) versus placebo after one year of treatment.

Additionally, significant improvements in arm span were demonstrated (p=0.004), a key prespecified secondary endpoint in the study. These positive findings could have important implications for children with hypochondroplasia, offering improvements in reach, daily activities and independence, which have been highlighted as meaningful outcomes by the community.

"As someone who treats children with hypochondroplasia, I find these positive results tremendously encouraging. Seeing these improvements in growth is a milestone we have hoped for after so many years without treatment options," said Dr. Andrew Dauber, who is the lead study investigator and Chief of Endocrinology at Children's National Hospital in Washington, D.C. "These data suggest we may be approaching a new era in how we care for children with hypochondroplasia."

"For the first time, a pivotal Phase 3 study in hypochondroplasia has demonstrated impressive gains in growth, including improvements in arm span, highlighting VOXZOGO's potential to fundamentally change the treatment landscape for this condition," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "We are incredibly pleased with these results, which exceeded our expectations for this study, and we are deeply grateful to the families and investigators who made this outcome possible. We look forward to sharing these data with regulatory authorities and the broader community as we work to bring this medicine to children living with hypochondroplasia around the world."

The safety findings in the study for VOXZOGO were consistent with the established profile in achondroplasia and no new safety signals were observed. Full results from the CANOPY-HCH-3 study will be presented at an upcoming medical meeting.

The supplemental New Drug Application (sNDA) submission to the U.S. Food and Drug Administration (FDA) is planned for the third quarter of 2026, followed by submissions to the European Medicines Agency (EMA) and other regional health authorities.

About CANOPY-HCH-3

CANOPY-HCH-3 (Study 111-303) is a global Phase 3, 1:1 randomized, double-blind, placebo-controlled, multicenter study designed to evaluate the efficacy and safety of VOXZOGO in 80 children ages 3 to 17 with hypochondroplasia. The primary endpoint of the study was change from baseline in annualized growth velocity (AGV) at 52 weeks compared to placebo. Key secondary pre-specified endpoints included changes from baseline in standing height, height Z-score, arm span, upper to lower body segment ratio, and health-related quality of life, along with assessments of safety and tolerability. Study participants will continue to be followed in a long-term extension study.

About Hypochondroplasia

Hypochondroplasia is a rare, genetic skeletal dysplasia characterized by impaired bone growth, leading to disproportionate short stature and skeletal differences that can affect the long bones, spine, and other parts of the skeleton and may impact physical functioning and overall quality of life. The condition presents with a broad and variable clinical spectrum and may include otolaryngologic (related to the ears, nose and throat) and neurological complications and is often diagnosed in toddlerhood or early school age based on clinical and radiological findings.

There are currently no medicines approved by the U.S. Food and Drug Administration or the European Medicines Agency for the treatment of hypochondroplasia.

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

About VOXZOGO (vosoritide)

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 submitted its U.S. supplemental New Drug Application (sNDA) for full approval of VOXZOGO for achondroplasia in April 2026, and the company expects to be notified of sNDA acceptance by the third quarter of 2026.

The use of VOXZOGO to treat hypochondroplasia has not yet been approved by any regulatory agency.

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 development of BioMarin's VOXZOGO program generally, and the results of the pivotal Phase 3 CANOPY-HCH-3 study evaluating treatment with VOXZOGO in children with hypochondroplasia particularly; the safety profile and potential benefits of VOXZOGO for children with hypochondroplasia, including VOXZOGO's potential to fundamentally change the treatment landscape for hypochondroplasia; BioMarin's plans to submit for regulatory approval to global health authorities in the second half of 2026; and BioMarin's expectations regarding the demand for VOXZOGO. 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, the final analysis of the results from CANOPY-HCH-3, 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 Quarterly Report on Form 10-Q for the quarter ended March 31, 2026, 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^® 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 Phase 3 results did BioMarin (BMRN) announce for VOXZOGO in hypochondroplasia on May 20, 2026?

BioMarin announced that VOXZOGO met the primary endpoint in the Phase 3 CANOPY-HCH-3 study for hypochondroplasia. According to BioMarin, annualized growth velocity improved by an LS mean difference of +2.33 cm/year versus placebo at week 52 (p<0.0001).

How did VOXZOGO affect growth measures in children with hypochondroplasia in BioMarin's Phase 3 study?

VOXZOGO increased annualized growth velocity, standing height and height Z-score compared with placebo after one year of treatment. According to BioMarin, all three measures showed statistically significant improvements, with p-values less than 0.0001 for standing height and height Z-score.

Why is arm span improvement important in BioMarin's VOXZOGO Phase 3 hypochondroplasia trial (BMRN)?

Arm span improvement is a prespecified secondary endpoint linked to reach and functional independence. According to BioMarin, VOXZOGO achieved a statistically significant arm span benefit versus placebo at week 52 (p=0.004), which may support better daily activities for affected children.

What did BioMarin report about VOXZOGO safety in the CANOPY-HCH-3 hypochondroplasia trial?

BioMarin reported that VOXZOGO safety in hypochondroplasia was consistent with its established profile in achondroplasia. According to BioMarin, no new safety signals were identified in the CANOPY-HCH-3 Phase 3 study after 52 weeks of treatment.

When will BioMarin (BMRN) file for FDA approval of VOXZOGO in hypochondroplasia?

BioMarin plans to submit a supplemental New Drug Application (sNDA) to the FDA in the third quarter of 2026. According to BioMarin, this will be followed by filings to the European Medicines Agency and additional regional health authorities.

What could VOXZOGO's Phase 3 hypochondroplasia data mean for the treatment landscape?

The data indicate clinically meaningful gains in growth measures, including arm span, versus placebo. According to BioMarin and study investigators, these results suggest VOXZOGO may offer a new pharmacologic option for children with hypochondroplasia, pending regulatory review.