BioMarin Announces New Three-Year VOXZOGO® (vosoritide) Data in Hypochondroplasia and BMN 333 Early Results at ENDO 2026

Rhea-AI Summary

BioMarin (Nasdaq:BMRN) reported new VOXZOGO (vosoritide) and BMN 333 data at ENDO 2026. A three-year Phase 2 extension in 13 children with hypochondroplasia showed sustained gains in annualized growth velocity and height SDS, with a favorable safety profile.

Phase 1 results for investigational BMN 333 in healthy adults supported weekly dosing for achondroplasia, with over 13-fold higher free CNP exposure than another long-acting CNP agent and no dose-limiting toxicities. BioMarin plans a VOXZOGO sNDA in Q3 2026 and has begun a registration-enabling Phase 2/3 BMN 333 trial.

Positive

• VOXZOGO Phase 2 extension: mean height SDS improved 0.72 over three years
• VOXZOGO annualized growth velocity rose from 4.27 to 7.24 cm/year at year one (p<0.001)
• Hypochondroplasia study reported sustained growth gains through years two and three
• VOXZOGO three-year treatment showed a favorable safety profile in 13 children
• BMN 333 Phase 1 supported weekly dosing with prolonged target engagement in adults
• Maximum BMN 333 dose increased free CNP exposure >13x versus another long-acting CNP
• BMN 333 Phase 1 reported no dose-limiting toxicities or treatment-related serious events
• Registration-enabling Phase 2/3 BMN 333 study has begun enrollment
• VOXZOGO sNDA for hypochondroplasia planned for FDA submission in Q3 2026

Negative

• VOXZOGO hypochondroplasia extension data based on 13 treated children
• BMN 333 human data currently limited to a Phase 1 study in healthy adults
• BMN 333 Phase 2/3 dose-finding data update not expected until 2027

News Market Reaction – BMRN

-0.95%

-0.95% Session close to close

In the Jun 16 session, BMRN declined 0.95%, reflecting a mild negative market reaction.

Data tracked by StockTitan Argus on the day of publication.

Market Context

This announcement highlights strengthening evidence for VOXZOGO in hypochondroplasia, with three‑yea...

Analysis

This announcement highlights strengthening evidence for VOXZOGO in hypochondroplasia, with three‑year data showing improved growth metrics, and encouraging early BMN 333 results that support weekly dosing in achondroplasia. Together, they expand BioMarin’s skeletal dysplasia franchise alongside prior Phase 3 VOXZOGO data and ongoing late‑stage plans. Investors may watch upcoming sNDA timing, BMN 333 Phase 2/3 enrollment progress, and future updates expected in 2027 to gauge how this platform evolves.

Key Figures

Patients in VOXZOGO study: 13 children Height SDS change: 0.72 SD improvement Baseline AGV: 4.27 cm/year +5 more

8 metrics

Patients in VOXZOGO study 13 children Phase 2 three-year extension in hypochondroplasia

Height SDS change 0.72 SD improvement Mean height SDS over three years of VOXZOGO treatment

Baseline AGV 4.27 cm/year Annualized growth velocity at baseline in VOXZOGO study

Year 1 AGV 7.24 cm/year Annualized growth velocity after one year of VOXZOGO

P-value for AGV change p<0.001 Statistical significance of AGV increase vs baseline

Max BMN 333 dose 500 μg/kg Maximum examined dose in Phase 1 healthy adult study

Free CNP exposure increase More than 13 times BMN 333 vs another long-acting CNP agent at max dose

Phase 2/3 data timing 2027 Expected dose-finding data update for BMN 333 Phase 2/3

Historical Context

4 past events · Latest: May 20 (Positive)

Pattern 4 events

Date	Event	Sentiment	24h Move	Catalyst
May 20	Phase 3 VOXZOGO data	Positive	+7.8%	Positive Phase 3 hypochondroplasia results with strong growth velocity benefit.
May 18	BMN 401 Phase 3 update	Negative	-4.1%	One co-primary endpoint met but no clear clinical benefit on key measures.
May 04	Q1 2026 earnings	Neutral	-4.0%	Revenue growth and higher guidance but lower GAAP and non‑GAAP earnings.
Apr 27	Amicus acquisition close	Positive	+1.2%	Closed all‑cash Amicus deal adding approved rare disease therapies and pipeline.

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

Pattern Detected

Recent major clinical and transaction headlines for BioMarin have generally seen price moves that align with the news tone, while the latest earnings release showed a negative reaction despite mixed fundamentals.

Recent Company History

Over the past few months, BioMarin has reported several significant updates. On Apr 27, it completed the $4.8 billion Amicus acquisition at $14.50 per share, followed by Q1 2026 results on May 4 with $766 million revenue and raised full‑year guidance, yet shares fell. Subsequent clinical news has been key: mixed Phase 3 results for BMN 401 on May 18 and clearly positive Phase 3 VOXZOGO data in hypochondroplasia on May 20, which drove a strong gain. Today’s longer‑term VOXZOGO and early BMN 333 data extend this clinical narrative.

Key Terms

annualized growth velocity, height standard deviation score, p<0.001, Phase 1, +4 more

8 terms

annualized growth velocity medical

"demonstrated sustained improvements in annualized growth velocity (AGV) and height standard..."

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 standard deviation score medical

"demonstrated sustained improvements in annualized growth velocity (AGV) and height standard deviation score (SDS)..."

A height standard deviation score quantifies how far an individual’s height is from the average height for people of the same age and sex, expressed in units of the population’s typical spread. For investors, it matters because regulators and clinical studies use this objective, comparable number to show whether a treatment or condition meaningfully changes growth—think of it like a student’s test score that shows how many grade-level steps they are above or below the class average.

p<0.001 technical

"mean AGV increased from 4.27 cm/year at baseline to 7.24 cm/year at year one (p<0.001)..."

p<0.001 indicates that the chance of seeing the reported result (or something more extreme) if there were actually no real effect is less than 0.1%. For investors, that low probability means the finding is very unlikely to be a fluke—like getting nearly all heads in many coin tosses—so it raises confidence in the result, but it does not by itself prove a meaningful financial impact or establish cause-and-effect.

Phase 1 medical

"In a Phase 1 single-ascending dose study in healthy adults, BMN 333 demonstrated..."

Phase 1 is the first stage of testing a new drug or medical treatment in people, focused primarily on safety, how the body handles the product, and finding a tolerated dose. Think of it as a short, tightly controlled experiment with a small group to check for dangerous side effects before wider testing; for investors it is an early milestone that reduces some uncertainty but still carries high risk and potential for both big value changes and setbacks.

Phase 2/3 medical

"BioMarin began enrolling patients in the registration-enabling Phase 2/3 study of BMN 333."

A phase 2/3 trial is a combined clinical study that first evaluates how well a treatment works and the best dose, then expands into a larger test to confirm those results and safety. For investors, it matters because moving into a phase 2/3 signals that an experimental therapy has shown initial promise and will be tested at scale, which can materially change the odds and timeline for regulatory approval and commercial potential.

C-type natriuretic peptide medical

"investigational BMN 333, BioMarin's long-acting C-type natriuretic peptide (CNP) for achondroplasia."

C-type natriuretic peptide is a small, naturally occurring protein the body uses to relax blood vessels and regulate cell growth, especially in blood vessel walls and bone. Think of it as a local traffic cop that calms traffic and tells cells when to slow down dividing. For investors, it matters because drugs or tests that mimic or measure this peptide can become treatments or diagnostics for heart, vascular and growth disorders, affecting market value and regulatory risk.

pharmacodynamic medical

"demonstrated sustained systemic exposure associated with prolonged pharmacodynamic target engagement..."

Pharmacodynamic describes how a drug acts on the body — the biological effects it produces, how strong those effects are, and how long they last. For investors, pharmacodynamic data show whether a treatment actually works and at what dose, shaping expectations about a drug’s safety, effectiveness, regulatory success and market potential; think of it like testing how well a key turns a lock and whether it reliably opens the door.

dose-limiting toxicities medical

"BMN 333 was well tolerated across all dose levels evaluated, with no dose-limiting toxicities..."

Dose-limiting toxicities are the harmful side effects seen in early clinical trials that are severe enough to stop researchers from raising a drug’s dose. Like a car’s speed limiter marking the safe top speed, DLTs define the maximum tolerable dose, and they matter to investors because they determine whether a medicine can reach effective levels, influence development timelines, costs, and regulatory chances, and thus affect a drug’s commercial prospects.

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

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Data from an investigator-led study showed treatment with VOXZOGO resulted in sustained improvements in growth over three years in children with hypochondroplasia

New Phase 1 results also presented for investigational BMN 333 support potential weekly dosing in children with achondroplasia

SAN RAFAEL, Calif., June 16, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced new data from studies of VOXZOGO^® (vosoritide) in children with hypochondroplasia and the investigational medicine BMN 333 in achondroplasia were presented at ENDO 2026, the Endocrine Society Annual Meeting, in Chicago.

New data from a Phase 2, investigator-sponsored three-year extension study of VOXZOGO in 13 children with hypochondroplasia, led by Andrew Dauber, M.D. and investigators at Children's National Hospital, demonstrated sustained improvements in annualized growth velocity (AGV) and height standard deviation score (SDS), with a favorable safety profile. Mean height SDS improved by 0.72 SD over three years of treatment, while mean AGV increased from 4.27 cm/year at baseline to 7.24 cm/year at year one (p<0.001) and remained above baseline through years two and three.

BioMarin recently announced positive topline results from CANOPY-HCH-3, its registration-enabling Phase 3 pivotal study evaluating VOXZOGO in children with hypochondroplasia. These results will be included in the supplemental New Drug Application submission to the U.S. Food and Drug Administration planned for the third quarter of 2026.

"Building on the excellent Phase 3 results we recently announced, these new longer-term data further reinforce the potential of VOXZOGO to meaningfully improve growth outcomes for children with hypochondroplasia," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "Importantly, we continue to observe sustained growth improvements over time and a favorable safety profile, adding to the growing body of evidence supporting VOXZOGO for this potential new indication."

New BMN 333 Phase 1 Data Support Advancement Into Late-Stage Development

Additional data being presented at ENDO 2026 highlight the potential of investigational BMN 333, BioMarin's long-acting C-type natriuretic peptide (CNP) for achondroplasia. In a Phase 1 single-ascending dose study in healthy adults, BMN 333 demonstrated sustained systemic exposure associated with prolonged pharmacodynamic target engagement, supporting a weekly dosing schedule. The maximum examined dose of BMN 333 (500 μg/kg) increased exposure to free CNP by more than 13 times compared to another long-acting CNP agent, reflecting the potential of BMN 333 to become the new standard of care in achondroplasia. BMN 333 was well tolerated across all dose levels evaluated, with no dose-limiting toxicities or treatment-related serious adverse events. 

In April, BioMarin began enrolling patients in the registration-enabling Phase 2/3 study of BMN 333. A data update from the dose-finding segment of this study is expected in 2027.

Below are key presentations for achondroplasia and hypochondroplasia at ENDO, with all times listed in Central Daylight Time:

Vosoritide Treatment in Children With Hypochondroplasia: Three-Year Results From a Phase 2 Extension Trial
Oral Presentation #ORF47-08
Monday, June 15, 3 – 3:15 p.m.

BMN 333 Achieves High Sustained Released Vosoritide Exposure With Favorable Safety: Phase 1 Results That Support Phase 2/3 Trials in Achondroplasia
Poster Presentation #SUN-212 
Sunday, June 14, 9 a.m. – 4 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.

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. BioMarin estimates that roughly 14,000 children with hypochondroplasia within the company's global footprint may be eligible for treatment with VOXZOGO.

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

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.

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

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 nine 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 ENDO 2026, the Endocrine Society Annual Meeting, including oral and poster presentations; BioMarin's plans and expectations for the development of VOXZOGO for children with hypochondroplasia, including safety profile and potential benefits and plans to submit a supplemental New Drug Application to the U.S. Food and Drug Administration (FDA) during the third quarter of 2026; BioMarin's plans and expectations for the development of BMN 333 for children with achondroplasia, including plans to provide a data update from the dose finding segment of the registration-enabling Phase 2/3 study of BMN 333 in 2027; the safety profile and potential benefits of BMN 333, including the potential to become the new standard of care in achondroplasia; and BioMarin's estimate regarding total addressable patient population (TAPP) with respect to the conditions targeted by BioMarin's product candidates and commercial products, including hypochondroplasia. 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 FDA, 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^®, 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 new VOXZOGO hypochondroplasia data did BioMarin (BMRN) present at ENDO 2026?

BioMarin reported three-year Phase 2 extension data showing sustained growth improvements in children with hypochondroplasia. According to BioMarin, mean height SDS improved by 0.72 and annualized growth velocity rose from 4.27 to 7.24 cm/year at year one, remaining above baseline thereafter.

How did VOXZOGO affect annualized growth velocity in BioMarin's hypochondroplasia study?

VOXZOGO increased annualized growth velocity from 4.27 cm/year at baseline to 7.24 cm/year after one year. According to BioMarin, this change was statistically significant (p<0.001) and growth velocity stayed above baseline through years two and three of treatment.

What did BioMarin announce about its VOXZOGO sNDA for hypochondroplasia (BMRN)?

BioMarin plans to include recent Phase 3 topline results and new three-year hypochondroplasia data in a VOXZOGO supplemental NDA. According to BioMarin, this submission to the US Food and Drug Administration is planned for the third quarter of 2026.

What are the key Phase 1 results for BioMarin's BMN 333 in achondroplasia?

Phase 1 data in healthy adults showed BMN 333 achieved sustained systemic exposure and prolonged pharmacodynamic target engagement. According to BioMarin, the highest dose (500 μg/kg) increased free CNP exposure more than 13-fold versus another long-acting CNP, with no dose-limiting toxicities observed.

How often could BMN 333 be dosed for achondroplasia based on ENDO 2026 data?

Phase 1 results supported a potential weekly dosing schedule for BMN 333 in achondroplasia. According to BioMarin, sustained systemic exposure and prolonged target engagement at examined doses underpinned the decision to advance this long-acting CNP agent into a registration-enabling Phase 2/3 study.

When will investors see next data from BioMarin's BMN 333 Phase 2/3 trial?

A data update from the dose-finding portion of the BMN 333 registration-enabling Phase 2/3 study is expected in 2027. According to BioMarin, enrollment for this trial in children with achondroplasia began in April, following supportive Phase 1 safety and exposure findings.

What safety outcomes were reported for VOXZOGO and BMN 333 at ENDO 2026?

VOXZOGO showed a favorable safety profile over three years in 13 children with hypochondroplasia. According to BioMarin, BMN 333 was well tolerated in healthy adults across all evaluated doses, with no dose-limiting toxicities or treatment-related serious adverse events reported.