Last updated
Revenue
R&D Expenses
Operating CF
Total Assets
Total Liabilities
Equity
Shares Out.
D/E Ratio
Dilution Watch
Financials
52W LOW $49.2652W HIGH $70.98
Volume
2.62 M
Value Traded
341.09 M
Short % Float
6.31%
Days to Cover
4.9
+2.75%Week
-4.18%1 Month
-4.22%3 Month
+6.34%6 Month
-31.51%5 Year
+260.6%All Time
Cash Data
- Monthly Burn
- Burn Trend
- Dilution Risk
- SEC Filing
Key Stats
Market Cap
11.13 B
EPS (TTM)
0.38
P/E Ratio
152.49
Ent. Value
15.10 B
Total Shares
193.61 M
Float Shares
191.08 M
Insiders
1.29%
Institutions
102.34%
Upcoming Catalysts
FDA decisions, readouts and PDUFA dates · scored by probability of approval
BMRN Catalyst Timeline
Dated clinical, regulatory and corporate events for BioMarin Pharmaceutical Inc.
Catalyst Timeline
Dated clinical, regulatory & corporate events for BioMarin Pharmaceutical Inc.
1583Total events
22Upcoming
433Tier-1 (high impact)
2010 to 2034Coverage
Upcoming catalysts 2
Event history 50
●Oral PresentationPresentation
Oral Presentation #1138: C-Type Natriuretic Peptide (CNP) Agonist CNP38 Promotes Longitudinal Bone Growth by Growth Plate Expansion in the Hyp Mouse Model of XLH
X-linked hypophosphatemia (XLH)source ↗
●Poster PresentationPresentation
Poster Presentation #115: Development of BMN 333, a Long-Acting Version of Vosoritide for FGFR3-Related Dwarfism and Other Skeletal Dysplasias
FGFR3-related dwarfism and other skeletal dysplasiassource ↗
●Poster PresentationVosoritideApprovedPresentation
Poster Presentation #474: Comparison of Quantitative Computed Tomography Bone Mineral Density Measurements of Vosoritide-Treated Children With Achondroplasia to an Untreated Achondroplasia Reference Population
achondroplasiasource ↗
●Poster PresentationPresentation
Poster Presentation #521: Prospective, Observational Study of a Cohort of Hypophosphatasia Patients' Phenotypes Over Time and the Utility of Digital Outcomes Measures in This Population
hypophosphatasiasource ↗
●Poster PresentationPresentation
Poster Presentation #554: Efficacy and Safety of BMN 401 in Children With ENPP1 Deficiency: 52-Week Results From the Pivotal ENERGY 3 Trial
ENPP1 deficiencysource ↗
●Poster PresentationVosoritideApprovedPresentation
Poster Presentation #486: Examining the Effect of Vosoritide Treatment on Bone Strength in Children With Achondroplasia
achondroplasiasource ↗
●Poster PresentationPresentation
Poster Presentation #473: Economic Burden of Hypophosphatasia: A Systematic Literature Review
hypophosphatasiasource ↗
●Poster PresentationPresentation
Poster Presentation #092: BMN 333 promotes endochondral bone growth in juvenile mice and nonhuman primates
skeletal dysplasiassource ↗
●Management ChangeCorporate
Amy Wireman retiring as Chief People Officer; to remain in advisory capacity
▲Long Term DataPOMBILITIClinical Data
Five-year Phase 3 PROPEL OLE data for POMBILITI + OPFOLDA presented at WMS
late-onset Pompe diseasesource ↗
▲Long Term DataOPFOLDAClinical Data
Four-year Phase 3 PROPEL OLE data for POMBILITI + OPFOLDA presented at WMS
late-onset Pompe diseasesource ↗
▲Poster PresentationPOMBILITIPresentation
Four-year efficacy and safety of cipaglucosidase alfa plus miglustat in Danish subpopulation presented at WMS
late-onset Pompe diseasesource ↗
●Poster PresentationPresentation
Baseline disease burden and real-world monitoring practices from followME registry presented at WMS
late-onset Pompe diseasesource ↗
▲Long Term DataPOMBILITIClinical Data
Five-year Phase 3 PROPEL OLE data for POMBILITI + OPFOLDA presented at WMS
late-onset Pompe diseasesource ↗
●RWE DataClinical Data
followME Pompe Journey Registry real-world insights presented at WMS
late-onset Pompe diseasesource ↗
▲Poster PresentationPOMBILITIPresentation
Five-year efficacy and safety of cipaglucosidase alfa plus miglustat from PROPEL baseline presented at WMS
late-onset Pompe diseasesource ↗
▲Late-BreakerPOMBILITIPresentation
Late-breaking WMS presentation of five-year PROPEL OLE data
late-onset Pompe diseasesource ↗
▲Poster PresentationnivudirsenPresentation
Phase 1/2 results of BMN 351 in exon 51 skip-amenable DMD presented at WMS
Duchenne muscular dystrophysource ↗
▲AcquiredCorporate
BioMarin completes acquisition of Alesta Therapeutics, adding ALE1 to pipeline
▲IP Litigation RulingIP / Competition
Global settlement with Ascendis Pharma resolving patent disputes
▲Partnership / LicenseYuviwelCorporate
License granted to Ascendis for BioMarin patents related to Yuviwel
achondroplasia and hypochondroplasiasource ↗
◆AcquisitionCorporate
BioMarin entered into definitive agreement to acquire Alesta Therapeutics
◆Oral PresentationPresentation
BioMarin management to present at Canaccord Genuity 46th Annual Growth Conference
●Timing GuidanceVosoritideApprovedPresentation
Expects to be notified of sNDA acceptance for achondroplasia by Q3 2026
achondroplasiasource ↗
▲Topline ReadoutVosoritideApprovedClinical Data
Positive topline results from Phase 3 CANOPY-HCH-3 study of VOXZOGO in hypochondroplasia
hypochondroplasiasource ↗
▲Oral PresentationVosoritideApprovedPresentation
Three-year results from Phase 2 extension trial of VOXZOGO in hypochondroplasia presented at ENDO 2026
hypochondroplasiasource ↗
▲Poster PresentationPresentation
Phase 1 results of BMN 333 supporting weekly dosing presented at ENDO 2026
achondroplasiasource ↗
◆Management ChangeCorporate
Ian T. Clark elected Chair of the Board, subject to election at Annual Meeting
▲Primary Endpoint MetVosoritideApprovedClinical Data
Phase 3 CANOPY-HCH-3 study met primary endpoint with statistically significant improvement in annualized growth velocity
hypochondroplasiasource ↗
▲Secondary Endpoint DataVosoritideApprovedClinical Data
Statistically significant improvement in arm span at week 52
hypochondroplasiasource ↗
▼Topline ReadoutClinical Data
ENERGY 3 topline results: met PPi co-primary endpoint, missed RGI-C co-primary endpoint
ENPP1 deficiencysource ↗
●Enrollment MilestoneTrial
12 mg/kg dose cohort continues to enroll
Duchenne muscular dystrophysource ↗
●Timing GuidanceVosoritideApprovedPresentation
Enrollment progressing in Phase 2 study of VOXZOGO in children under 3 with hypochondroplasia
hypochondroplasiasource ↗
▲Poster PresentationVosoritideApprovedPresentation
Poster presentation on effect of long-term vosoritide treatment on growth in achondroplasia (open-label, multicenter trials)
achondroplasiasource ↗
▲Poster PresentationVosoritideApprovedPresentation
Poster presentation on vosoritide treatment improving linear growth and bone content/density in hypochondroplasia (12-month prospective study)
hypochondroplasiasource ↗
▲Oral PresentationVosoritideApprovedPresentation
Reported new long-term data at PES annual meeting
achondroplasiasource ↗
◆Timing GuidancePresentation
Update on longer-term outlook of Amicus integration expected next quarter
▲Enrollment MilestoneTrial
Enrollment began in Phase 2/3 study of BMN 333 in achondroplasia
achondroplasiasource ↗
▲First Patient DosedTrial
First patient enrolled in registration-enabling Phase 2/3 study of BMN 333
achondroplasiasource ↗
●sNDA SubmissionVosoritideApprovedRegulatory Filing
Submitted sNDA for full approval of VOXZOGO for achondroplasia
achondroplasiasource ↗
◆Topline ReadoutClinical Data
Expects to share BMN 401 Phase 3 topline data in Q2 2026
ENPP1 deficiencysource ↗
◆Topline ReadoutVosoritideApprovedClinical Data
Expects to share Phase 3 topline data for VOXZOGO for hypochondroplasia in Q2 2026
hypochondroplasiasource ↗
▲Poster PresentationVosoritideApprovedPresentation
Poster presentation: Effectiveness of Vosoritide in Children With Achondroplasia Starting Treatment Aged <2 Years: Interim Results From a Japanese Real-World Study
achondroplasiasource ↗
▲Poster PresentationVosoritideApprovedPresentation
Poster presentation: Real-World Healthcare Utilization, Effectiveness, Health-Related Quality of Life, and Adherence to Vosoritide in Children with Achondroplasia in the United States
achondroplasiasource ↗
Past FDA Catalysts and PDUFA Decisions
How BMRN actually traded into and out of each decision
| Date | Drug | Catalyst | Stage | Reaction | Event Move % | Best Trade % |
|---|---|---|---|---|---|---|
| 2026-10-11 | Vosoritide (VOXZOGO) | Phase 3 data presentation | Phase 3 |
Reaction: 30 trading days around the event · Event Move: close to close on decision day · Best Trade: peak run-up from entry 10 days before the decision
Drug Pipeline Intelligence
Pipeline Score
Pipeline Value
Commercial Value
Valuation Signal
7
Drugs Scored
rNPV / MCap
Top Drug
Top 10%
Mid Cap(rank 67 of 657)
Percentile Rank
Commercial products (revenue basis: trailing 4 quarters of total revenue (10-K and 10-Q), to Jun 2026; product lines: last four quarters to Jun 2026 (10-K and 10-Q))
| Product | Revenue | Prior period | Change |
|---|---|---|---|
| VOXZOGO | |||
| VIMIZIM | |||
| NAGLAZYME | |||
| PALYNZIQ | |||
| BRINEURA | |||
| ALDURAZYME | |||
| GALAFOLD | |||
| KUVAN | |||
| ROCTAVIAN |
| Drug | Indication | Phase | PTRS | rNPV | Status | Enroll | Velocity | Design | Est. Completion | ML Signal | Last Change |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Vosoritide Small moleculeNCT06455059 | Hypochondroplasia | Phase 3 | ACTIVE NOT_RECRUITING | 280 | Aug 1, 2026 | ||||||
| Vosoritide Small moleculeMarketedNCT03989947 | Achondroplasia | Phase 3 | ACTIVE NOT_RECRUITING | 547 | May 1, 2038 | ||||||
| Vosoritide Small moleculeNCT06668805 | Noonan Syndrome | Phase 2 | ACTIVE NOT_RECRUITING | 30 | Sep 1, 2041 | ||||||
| Vosoritide Small moleculeNCT06382155 | Idiopathic Short Stature | Phase 2 | RECRUITING | 100 | Dec 1, 2036 | ||||||
| INZ-701 Small moleculeNCT06046820 | Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency | Phase 3 | ACTIVE NOT_RECRUITING | 64 | Feb 1, 2026 | ||||||
| INZ-701 Small moleculeNCT06462547 | Gene Mutations | Phase 2 | ACTIVE NOT_RECRUITING | 22 | Oct 1, 2026 | ||||||
| BMN 351 Small moleculeNCT06280209 | Duchenne Muscular Dystrophy | Phase 2 | ACTIVE NOT_RECRUITING | 36 | Apr 14, 2027 | ||||||
| BMN 349 Small moleculeNCT06738017 | Alpha 1-Antitrypsin Deficiency | Phase 1 | ACTIVE NOT_RECRUITING | 6 | Aug 19, 2026 | ||||||
| BMN 307 Small moleculeNCT04480567 | Phenylketonuria (PKU) | Phase 1 | ACTIVE NOT_RECRUITING | 100 | Dec 1, 2027 | ||||||
| BMN 333 Small moleculeNCT07441876 | Achondroplasia | Phase 2 | RECRUITING | 160 | Sep 1, 2029 | ||||||
| Pegvaliase Small moleculeMarketedNCT05270837 | Phenylketonuria (PKU) | Phase 3 | ACTIVE NOT_RECRUITING | 92 | Oct 1, 2027 | ||||||
| BMN 331 Gene therapyNCT05121376 | Hereditary Angioedema | Phase 1 | ACTIVE NOT_RECRUITING | 44 | Nov 1, 2028 | ||||||
| N-acetylgalactosamine 4-sulfatase Small moleculeCompletedNCT00048620 | Mucopolysaccharidosis VI | Phase 3 | COMPLETED | 78 | Nov 1, 2005 | ||||||
| INZ-701 Small moleculeCompletedNCT05030831 | ATP-Binding Cassette Subfamily C Member 6 Deficiency | Phase 1 | COMPLETED | 10 | Jul 9, 2024 | ||||||
| INZ-701 Small moleculeCompletedNCT06283589 | Calciphylaxis | Phase 1 | COMPLETED | 11 | Dec 13, 2024 | ||||||
| BMN 165 Monoclonal antibodyMarketedNCT01560286 | Phenylketonuria | Phase 3 | COMPLETED | 500 | Jul 1, 2015 | ||||||
| Kuvan Small moleculeMarketedNCT01376908 | Phenylketonuria | Phase 3 | COMPLETED | 56 | Feb 17, 2017 | ||||||
| sapropterin Small moleculeMarketedNCT00789568 | Phenylketonuria | Phase 3 | COMPLETED | 1,402 | Oct 1, 2009 | ||||||
| BMN 190 recombinant human tripeptidyl peptidase-1 Monoclonal antibodyMarketedNCT02678689 | Jansky-Bielschowsky Disease | Phase 2 | COMPLETED | 14 | Apr 20, 2022 | ||||||
| sapropterin Small moleculeMarketedNCT00445978 | Sickle Cell Disease | Phase 2 | COMPLETED | 32 | Jun 1, 2009 | ||||||
| 6R-BH4 Small moleculeMarketedNCT00625820 | Kidney Disease | Phase 2 | COMPLETED | 17 | Sep 1, 2008 | ||||||
| sapropterin Small moleculeMarketedNCT00532844 | Endothelial Dysfunction | Phase 2 | COMPLETED | 52 | Mar 1, 2009 | ||||||
| sapropterin Small moleculeMarketedNCT00403494 | Intermittent Claudication | Phase 2 | COMPLETED | 190 | Jan 1, 2009 | ||||||
| 6R-BH4 Small moleculeMarketedNCT00325962 | Hypertension | Phase 2 | COMPLETED | 84 | Dec 1, 2008 | ||||||
| BMN 190 Monoclonal antibodyMarketedNCT01907087 | Jansky-Bielschowsky Disease | Phase 1 | COMPLETED | 47 | Nov 1, 2015 |
Clinical Trial Results
Readouts, endpoints and source filings for every BMRN program
| Drug Name | Indication | Phase | Date | Trial Results Summary | Title | Source |
|---|---|---|---|---|---|---|
| POMBILITI + OPFOLDA | late-onset Pompe disease | Phase 3 | 2026-10-02 | 0.7%; 10.5%; -2.8%; -4.4%; 51.2%; n=42; 5Read More | BioMarin Announces Five-Year Phase 3 Data for POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) at World Muscle Society Annual CongressRead More |
Inside Trades
| Insider | Side | Shares | Price | Value | Date |
|---|---|---|---|---|---|
| Davis George EricOfficer (EVP, Chief Legal Officer) | Sell | -17,557 61,003 held | $66.28 | 09/02/2026 |
Hedge Fund 13F Activity
Hedge Funds invested in BMRN
| Fund | % of Portfolio | Current MV | Shares Owned | Activity |
|---|---|---|---|---|
| Deep Track Capital, LP | 2.8 % (154.5 %) | 189.25 M | 3.31 M | 187.61% ( 2.16 M) |
BMRN Institutional Ownership Trends
BMRN Short Interest
Shares sold short, counted twice a month.
6.31%
Short % of Float
of tradable shares sold short
12.1M
Shares Sold Short
+4.6% vs prior count
4.9 Moderate
Days to Cover
trading days of normal volume to buy every short share back
2.5M
Avg Daily Volume
the average used for days to cover
Options Data
Option Volume
Open Interest
Call OI
Put OI
Put-Call Ratio
OI 30-day avg
Today vs avg
Option Volume
Call Vol
Put Vol
Put-Call Ratio
Vol 30-day avg
Today vs avg
Option Chain Statistics
| Expiry | Call Vol | Put Vol | Vol P/C | Call OI | Put OI | OI P/C | IV Call | IV Call OI-Wtd | IV Call Vol-Wtd | IV Put | IV Put OI-Wtd | IV Put Vol-Wtd |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 2026-11-20 | ||||||||||||
Real-Time Option Chain
| Calls | Strike | Puts | ||||
|---|---|---|---|---|---|---|
| Last | Vol | OI | Last | Vol | OI | |
| No data available | ||||||
Calls vs Puts by Expiry
CallsPutsLog scale
Competitive positioning
How BMRN ranks across every disease it competes in
Competitive Position is a premium feature
See how BMRN ranks against every competitor across each disease it develops in: phase, best drug, trials and likelihood of approval.
Start free trialBMRN News
BMRN▲ +3.6%today
BioMarin Enters Into Exclusive Worldwide License Agreement With GC Biopharma and Hanmi Pharm for Investigational Next-Generation Enzyme Replacement Therapy, Adding a Second Potential Treatment for Fabry Disease
BioMarin has entered an exclusive worldwide license agreement with GC Biopharma and Hanmi Pharm for HM15421/GC1134A, a long-acting enzyme replacement therapy for Fabry disease. The agreement includes a $70 million upfront payment and potential additional payments of up to $405 million based on milestones. This therapy aims to provide a new treatment option for patients with Fabry disease.
Read more →BMRN▲ +3.6%today
BioMarin to Host Third Quarter 2026 Financial Results Conference Call and Webcast on Monday, November 2, 2026, at 4:30pm ET
BioMarin Pharmaceutical Inc. will host a conference call on November 2, 2026, at 4:30 p.m. ET. CEO Alexander Hardy will discuss the company's third quarter 2026 financial results and provide a business update. Interested parties can access the call via the investor section of BioMarin's website.
Read more →BMRN1cBio Received Shareholder Proceeds Upon Closing of BioMarin Pharmaceutical Inc.'s Acquisition of Alesta Therapeutics
1cBio has highlighted the successful acquisition of Alesta Therapeutics by BioMarin Pharmaceutical Inc., which closed on September 1, 2026. This acquisition includes Alesta's lead program, ALE1, aimed at treating hypophosphatasia, a rare genetic bone disease. 1cBio retains rights to future milestone payments and royalties from ALE1's development.
Read more →BMRNBioMarin Presents New Data Advancing Understanding of Achondroplasia and Hypochondroplasia Treatment at the American Society for Bone and Mineral Research Annual Meeting
BioMarin Pharmaceutical Inc. presented new data at the ASBMR 2026 Annual Meeting, highlighting the effects of VOXZOGO on growth in children with achondroplasia and hypochondroplasia. The findings suggest that early treatment may enhance growth-related outcomes, particularly in critical areas of the skull. The company has submitted a supplemental New Drug Application for VOXZOGO's use in hypochondroplasia, aiming for a 2027 launch if approved.
Read more →BMRNBioMarin Appoints Cara Bauer as Chief People Officer
BioMarin Pharmaceutical Inc. has appointed Cara Bauer, Ph.D., as the new Chief People Officer, effective October 5, 2026. With over 25 years of experience in human resources across various industries, Ms. Bauer succeeds Amy Wireman, who is retiring. Her extensive background includes leadership roles at Kyverna Therapeutics and Kite, a Gilead company, and she aims to foster a patient-centered culture at BioMarin.
Read more →BMRNBioMarin Announces Five-Year Phase 3 Data for POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) at World Muscle Society Annual Congress
BioMarin has presented five-year data on the efficacy and safety of POMBILITI (cipaglucosidase alfa-atga) combined with OPFOLDA (miglustat) for late-onset Pompe disease at the World Muscle Society Annual Congress. The results indicate sustained motor function and stabilization of pulmonary function over the long term. The study included 82 individuals, showing significant benefits for both ERT-experienced and ERT-naïve patients. No new safety concerns were reported, although some adverse events were noted.
Read more →BMRNBioMarin Announces Appointment of Robert Plenge, M.D., Ph.D., Chief Research Officer of Bristol Myers Squibb, to Board of Directors
BioMarin Pharmaceutical Inc. has appointed Dr. Robert Plenge to its Board of Directors, effective September 10, 2026. Dr. Plenge, currently Chief Research Officer at Bristol Myers Squibb, brings extensive experience in human genetics and drug discovery. His appointment is expected to enhance BioMarin's research capabilities and pipeline development for rare genetic conditions.
Read more →BMRNBioMarin Announces Positive Results of Phase 3 VOXZOGO® (vosoritide) Study in Hypochondroplasia Published in NEJM Evidence
BioMarin announced positive results from the Phase 3 CANOPY-HCH-3 study of VOXZOGO in children with hypochondroplasia, showing significant improvements in growth metrics. The findings were published in NEJM Evidence and presented at the ESPE 2026 Annual Meeting. BioMarin has submitted a supplemental New Drug Application to the FDA to expand VOXZOGO's indication to this condition, aiming for a 2027 launch.
Read more →BMRNBioMarin to Participate in Upcoming Investor Conferences
BioMarin Pharmaceutical Inc. will present at several investor conferences in September 2026, including the Wells Fargo 21st Annual Healthcare Conference and the Morgan Stanley 24th Annual Global Healthcare Conference. The company is recognized for its innovative approach to drug development and has a strong pipeline of therapies for genetically defined conditions. Live audio webcasts of the presentations will be available.
Read more →BMRNBioMarin Announces Close of the Alesta Therapeutics Acquisition
BioMarin Pharmaceutical Inc. has completed the acquisition of Alesta Therapeutics, enhancing its portfolio with ALE1, a potential first oral therapy for hypophosphatasia. ALE1 is currently undergoing a Phase 1/2a clinical trial to evaluate its safety and efficacy. This acquisition positions BioMarin to address significant unmet needs in rare genetic bone diseases.
Read more →BMRNBioMarin Announces Global Settlement with Ascendis Pharma A/S; Ascendis Will Pay Royalties to BioMarin on Yuviwel Sales in U.S., EU, Brazil and South Korea
BioMarin Pharmaceutical has reached a global settlement with Ascendis Pharma, resolving patent disputes related to Ascendis's Yuviwel. As part of the agreement, Ascendis will pay BioMarin royalties on Yuviwel sales in several regions, including the U.S. and EU. The settlement allows BioMarin to continue its focus on developing treatments for genetic conditions.
Read more →BMRNBioMarin to Acquire Alesta Therapeutics to Gain ALE1, a Potential First Oral Therapy for Hypophosphatasia, Adding an Important Clinical Program to BioMarin's Pipeline
BioMarin Pharmaceutical Inc. has announced its acquisition of Alesta Therapeutics for $275 million, aiming to gain ALE1, a potential first oral therapy for hypophosphatasia. The acquisition is set to enhance BioMarin's pipeline and address significant unmet needs in rare diseases. Alesta will spin out its non-ALE1 assets prior to the transaction's completion, which is expected soon, subject to customary conditions.
Read more →BMRN▲ +7.9%on this news
BioMarin to Participate at the Canaccord Genuity 46th Annual Growth Conference on Wednesday, August 12, 2026, at 8:00AM ET in Boston
BioMarin Pharmaceutical Inc. will present at the Canaccord Genuity 46th Annual Growth Conference on August 12, 2026, at 8:00 AM ET in Boston. An audio webcast of the presentation will be available live, with an archived version accessible later. The company is recognized for its commitment to developing therapies for genetically defined conditions.
Read more →BMRNBioMarin Reports Second Quarter 2026 Financial and Operating Results
BioMarin Pharmaceutical Inc. reported a 20% increase in total revenues for Q2 2026, reaching $990 million. The company raised its full-year revenue guidance to at least $3.875 billion, driven by strong demand for VOXZOGO and new product additions. However, GAAP net income fell sharply to $45 million due to acquisition-related costs and lower revenue from some existing products. The company is optimistic about future growth, especially with the expected launch of VOXZOGO for hypochondroplasia.
Read more →BMRNBioMarin to Host Second Quarter 2026 Financial Results Conference Call and Webcast on Thursday, August 6, 2026, at 4:30pm ET
BioMarin Pharmaceutical Inc. has announced a conference call scheduled for August 6, 2026, to discuss its second quarter financial results and provide a business update. The call will be hosted by CEO Alexander Hardy and will be accessible via a live audio webcast. A replay will be available for one week post-call.
Read more →BMRNBioMarin and n-Lorem Foundation Enter Early Research Collaboration to Develop Potential First-in-Disease Medicine for Newly Identified ReNU Syndrome
BioMarin Pharmaceutical and n-Lorem Foundation have announced a collaboration to develop an antisense oligonucleotide (ASO) medicine targeting ReNU syndrome, a rare neurodevelopmental condition. This partnership aims to address the underlying genetic cause of the disease, which currently has no approved treatments. The investigational ASO candidate will focus on the RNU4-2 variant, responsible for a significant portion of ReNU syndrome cases.
Read more →BMRNU.S. Food and Drug Administration Accepts BioMarin's Supplemental New Drug Application for Full Approval of VOXZOGO® (vosoritide) for Children With Achondroplasia
BioMarin announced that the FDA has accepted its supplemental New Drug Application for VOXZOGO (vosoritide) for full approval in children with achondroplasia. The application is based on extensive long-term safety and efficacy data from three ongoing studies. The FDA has set a target action date of February 28, 2027, for this application.
Read more →BMRNBioMarin Announces New Three-Year VOXZOGO® (vosoritide) Data in Hypochondroplasia and BMN 333 Early Results at ENDO 2026
BioMarin presented three-year VOXZOGO data for hypochondroplasia showing mean height SDS improved by 0.72 SD. Annualized growth velocity rose from 4.27 cm/year at baseline to 7.24 cm/year in year one. The company also shared early BMN 333 results supporting weekly dosing for achondroplasia. These findings support the upcoming supplemental New Drug Application submission planned for the third quarter of 2026.
Read more →BMRN▲ +7.8%on this news
BioMarin Announces Positive Phase 3 Pivotal Study Results for VOXZOGO® (vosoritide) in Children with Hypochondroplasia
BioMarin announced positive results from its Phase 3 CANOPY-HCH-3 study for VOXZOGO (vosoritide) in children with hypochondroplasia. The study demonstrated a statistically significant increase in annualized growth velocity, standing height, and arm span compared to placebo. Safety findings were consistent with the established profile, and a supplemental New Drug Application is planned for submission to the FDA in Q3 2026.
Read more →BMRNBioMarin Provides Update on Phase 3 Trial for BMN 401 in Children Aged 1-12 With ENPP1 Deficiency
BioMarin announced that its Phase 3 ENERGY 3 trial for BMN 401 in children with ENPP1 deficiency met only one of two co-primary endpoints. Treatment significantly increased plasma inorganic pyrophosphate levels compared to the control group. However, the study failed to show improvement in radiographic scores measuring rickets severity. The company is now evaluating the data to determine its next steps.
Read more →About BioMarin Pharmaceutical Inc.
Headquarters105 DIGITAL DRIVE, NOVATO, CA, 94949
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