{"success":true,"data":{"pressRelease":{"id":"65165","rtpr_id":"nPn7HxTxfa","ticker":"BMRN","exchange":"NASDAQ","all_tickers":["BMRN"],"title":"BioMarin Announces New Three-Year VOXZOGO® (vosoritide) Data in Hypochondroplasia and BMN 333 Early Results at ENDO 2026","author":"PR Newswire","published_at":"2026-06-16T13:05:03.085Z","article_body":"BioMarin Announces New Three-Year VOXZOGO® (vosoritide) Data in Hypochondroplasia and BMN 333 Early Results at ENDO 2026\n\nPR Newswire\n\nSAN RAFAEL, Calif., June 16, 2026\n\nData from an investigator-led study showed treatment with VOXZOGO resulted in\nsustained improvements in growth over three years in children with\nhypochondroplasia\n\nNew Phase 1 results also presented for investigational BMN 333 support\npotential weekly dosing in children with achondroplasia\n\nSAN RAFAEL, Calif., June 16, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc.\n(Nasdaq: BMRN) announced new data from studies of VOXZOGO(®) (vosoritide) in\nchildren with hypochondroplasia and the investigational medicine BMN 333 in\nachondroplasia were presented at ENDO 2026, the Endocrine Society Annual\nMeeting, in Chicago.\n\nNew data from a Phase 2, investigator-sponsored three-year extension study of\nVOXZOGO in 13 children with hypochondroplasia, led by Andrew Dauber, M.D. and\ninvestigators at Children's National Hospital, demonstrated sustained\nimprovements in annualized growth velocity (AGV) and height standard deviation\nscore (SDS), with a favorable safety profile. Mean height SDS improved by 0.72\nSD over three years of treatment, while mean AGV increased from 4.27 cm/year\nat baseline to 7.24 cm/year at year one (p<0.001) and remained above\nbaseline through years two and three.\n\nBioMarin recently announced positive topline results from CANOPY-HCH-3, its\nregistration-enabling Phase 3 pivotal study evaluating VOXZOGO in children\nwith hypochondroplasia. These results will be included in the supplemental New\nDrug Application submission to the U.S. Food and Drug Administration planned\nfor the third quarter of 2026.\n\n\"Building on the excellent Phase 3 results we recently announced, these new\nlonger-term data further reinforce the potential of VOXZOGO to meaningfully\nimprove growth outcomes for children with hypochondroplasia,\" said Greg\nFriberg, M.D., Executive Vice President and Chief Research & Development\nOfficer at BioMarin. \"Importantly, we continue to observe sustained growth\nimprovements over time and a favorable safety profile, adding to the growing\nbody of evidence supporting VOXZOGO for this potential new indication.\"\n\nNew BMN 333 Phase 1 Data Support Advancement Into Late-Stage Development\n\nAdditional data being presented at ENDO 2026 highlight the potential of\ninvestigational BMN 333, BioMarin's long-acting C-type natriuretic peptide\n(CNP) for achondroplasia. In a Phase 1 single-ascending dose study in healthy\nadults, BMN 333 demonstrated sustained systemic exposure associated with\nprolonged pharmacodynamic target engagement, supporting a weekly dosing\nschedule. The maximum examined dose of BMN 333 (500 μg/kg) increased exposure\nto free CNP by more than 13 times compared to another long-acting CNP agent,\nreflecting the potential of BMN 333 to become the new standard of care in\nachondroplasia. BMN 333 was well tolerated across all dose levels evaluated,\nwith no dose-limiting toxicities or treatment-related serious adverse\nevents.\n\nIn April, BioMarin began enrolling patients in the registration-enabling Phase\n2/3 study of BMN 333. A data update from the dose-finding segment of this\nstudy is expected in 2027.\n\nBelow are key presentations for achondroplasia and hypochondroplasia at ENDO,\nwith all times listed in Central Daylight Time:\n\nVosoritide Treatment in Children With Hypochondroplasia: Three-Year Results\nFrom a Phase 2 Extension Trial\nOral Presentation #ORF47-08\nMonday, June 15, 3 – 3:15 p.m.\n\nBMN 333 Achieves High Sustained Released Vosoritide Exposure With Favorable\nSafety: Phase 1 Results That Support Phase 2/3 Trials in Achondroplasia\nPoster Presentation #SUN-212\nSunday, June 14, 9 a.m. – 4 p.m.\n\nAbout Achondroplasia\n\nAchondroplasia, the most common form of skeletal dysplasia leading to\ndisproportionate short stature in humans, is characterized by slowing of\nendochondral ossification, which results in disproportionate short stature and\ndisordered architecture in the long bones, spine, face and base of the skull.\nThis condition is caused by a change in the FGFR3 gene, a negative regulator\nof bone growth.\n\nMore than 80% of children with achondroplasia have parents of average stature\nand have the condition as the result of a spontaneous gene mutation. The\nworldwide incidence rate of achondroplasia is about one in 25,000 live births.\nVOXZOGO is being tested in children whose growth plates are still \"open,\"\ntypically those under 18 years of age. Approximately 25% of people with\nachondroplasia fall into this category.\n\nAbout Hypochondroplasia\n\nHypochondroplasia is a rare, genetic skeletal dysplasia characterized by\nimpaired bone growth, leading to disproportionate short stature and skeletal\ndifferences that can affect the long bones, spine and other parts of the\nskeleton and may impact physical functioning and overall quality of life. The\ncondition presents with a broad and variable clinical spectrum and may include\notolaryngologic (related to the ears, nose and throat) and neurological\ncomplications and is often diagnosed in toddlerhood or early school age based\non clinical and radiological findings. BioMarin estimates that roughly 14,000\nchildren with hypochondroplasia within the company's global footprint may be\neligible for treatment with VOXZOGO.\n\nThere are currently no medicines approved by the U.S. Food and Drug\nAdministration or the European Medicines Agency for the treatment of\nhypochondroplasia.\n\nFor more information about our clinical trials in hypochondroplasia,\nachondroplasia and other skeletal conditions, please visit\nclinicaltrials.biomarin.com\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4710891-1&h=1441447510&u=https%3A%2F%2Fclinicaltrials.biomarin.com%2F&a=clinicaltrials.biomarin.com)\n.\n\nAbout VOXZOGO\n\nIn children with achondroplasia, endochondral bone growth, an essential\nprocess by which bone tissue is created, is negatively regulated due to a gain\nof function mutation in FGFR3. VOXZOGO, a C-type natriuretic peptide (CNP)\nanalog, acts as a positive regulator of the signaling pathway downstream of\nFGFR3 to promote endochondral bone growth.\n\nVOXZOGO is the only approved medicine to support the growth of children with\nachondroplasia starting from birth, with international consensus guidelines\nrecommending initiation of VOXZOGO as early as possible. First approved in\n2021, VOXZOGO has helped more than 5,000 infants and children in more than 50\ncountries. Through our ongoing studies, BioMarin continues to evaluate VOXZOGO\non key clinical endpoints relevant for achondroplasia patients, such as arm\nspan, tibial bowing (leg bowing), body proportionality, spinal morphology\n(including spinal stenosis) and quality of life measures.\n\nVOXZOGO is approved in the U.S., Japan and Australia to increase linear growth\nin children of all ages with achondroplasia with open epiphyses, and VOXZOGO\nis indicated in the EU for the treatment of achondroplasia in children 4\nmonths of age and older whose epiphyses are not closed, as confirmed by\nappropriate genetic testing. In the U.S., this indication is approved under\naccelerated approval based on an improvement in annualized growth velocity.\nContinued approval may be contingent upon verification and description of\nclinical benefit in confirmatory trial(s). To fulfill this post-marketing\nrequirement, BioMarin intends to use the ongoing open-label extension studies\ncompared to available natural history.\n\nThe use of VOXZOGO to treat hypochondroplasia has not yet been approved by any\nregulatory agency.\n\nPatient Support Accessing VOXZOGO\n\nBioMarin's robust support services have ensured a seamless treatment\nexperience, spearheaded by Clinical Coordinators, who have conducted hundreds\nof trainings for families with achondroplasia since approval. BioMarin\nprovides resources to support families navigating achondroplasia, including a\ncaregiver mentorship program that connects parents with other caregivers, and\na U.S. doctor directory that helps families and healthcare professionals\nidentify clinicians experienced in achondroplasia care.\n\nTo reach a BioMarin RareConnections(®) Case Manager, please call, toll-free,\n1-833-VOXZOGO (1-833-869-9646) or e-mail\nVOXZOGOSupport@biomarin-rareconnections.com\n(mailto:VOXZOGOSupport@biomarin-rareconnections.com) . For more information\nabout VOXZOGO, please visit www.voxzogo.com\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4710891-1&h=2227173943&u=http%3A%2F%2Fwww.voxzogo.com%2F&a=www.voxzogo.com)\n. For additional information regarding this product, please contact BioMarin\nMedical Information at medinfo@bmrn.com (mailto:medinfo@bmrn.com) .\n\nVOXZOGO U.S. Important Safety Information\n\nWhat is VOXZOGO used for?\n\n * VOXZOGO is a prescription medicine used to increase linear growth in children\nwith achondroplasia and open growth plates (epiphyses).\n * VOXZOGO is approved under accelerated approval based on an improvement in\nannualized growth velocity. Continued approval may be contingent upon\nverification and description of clinical benefit in confirmatory trials.\nWhat is the most important safety information about VOXZOGO?\n\n * VOXZOGO may cause serious side effects including a temporary decrease in blood\npressure in some patients. To reduce the risk of a decrease in blood pressure\nand associated symptoms (dizziness, feeling tired, or nausea), patients should\neat a meal and drink 8 to 10 ounces of fluid within 1 hour before receiving\nVOXZOGO.\nWhat are the most common side effects of VOXZOGO?\n\n * The most common side effects of VOXZOGO include injection site reactions\n(including redness, itching, swelling, bruising, rash, hives, and injection\nsite pain), high levels of blood alkaline phosphatase shown in blood tests,\nvomiting, joint pain, decreased blood pressure, and stomachache. These are not\nall the possible side effects of VOXZOGO. Ask your healthcare provider for\nmedical advice about side effects, and about any side effects that bother the\npatient or that do not go away.\nHow is VOXZOGO taken?\n\n * VOXZOGO is taken daily as an injection given under the skin, administered by a\ncaregiver after a healthcare provider determines the caregiver is able to\nadminister VOXZOGO. Do not try to inject VOXZOGO until you have been shown the\nright way by your healthcare provider. VOXZOGO is supplied with Instructions\nfor Use that describe the steps for preparing, injecting, and disposing\nVOXZOGO. Caregivers should review the Instructions for Use for guidance and\nany time they receive a refill of VOXZOGO in case any changes have been made.\n * Inject VOXZOGO 1 time every day, at about the same time each day. If a dose of\nVOXZOGO is missed, it can be given within 12 hours from the missed dose. After\n12 hours, skip the missed dose and administer the next daily dose as usual.\n * The dose of VOXZOGO is based on body weight. Your healthcare provider will\nadjust the dose based on changes in weight following regular check-ups.\n * Your healthcare provider will monitor the patient's growth and tell you when\nto stop taking VOXZOGO if they determine the patient is no longer able to\ngrow. Stop administering VOXZOGO if instructed by your healthcare provider.\nWhat should you tell the doctor before or during taking VOXZOGO?\n\n * Tell your doctor about all of the patient's medical conditions including\n* If the patient has heart disease (cardiac or vascular disease), or if the\npatient is on blood pressure medicine (anti-hypertensive medicine).\n * If the patient has kidney problems or renal impairment.\n * If the patient is pregnant or plans to become pregnant. It is not known if\nVOXZOGO will harm the unborn baby.\n * If the patient is breastfeeding or plans to breastfeed. It is not known if\nVOXZOGO passes into breast milk.\n * Tell your doctor about all of the medicines the patient takes, including\nprescription and over-the-counter medicines, vitamins, and herbal supplements.\nYou may report side effects to BioMarin at 1-866-906-6100. You are encouraged\nto report negative side effects of prescription drugs to the FDA. Visit\nwww.fda.gov/medwatch\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4710891-1&h=550579757&u=http%3A%2F%2Fwww.fda.gov%2Fmedwatch&a=www.fda.gov%2Fmedwatch)\n, or call 1-800-FDA-1088.\n\nPlease see additional safety information in the full Prescribing Information\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4710891-1&h=526800756&u=https%3A%2F%2Fd34r3hkxgxjdtw.cloudfront.net%2F6f836309-d95f-42af-b717-2efa058ad82d%2F11d8e586-f965-4525-bc33-93c4b147f11b%2F11d8e586-f965-4525-bc33-93c4b147f11b_source__v.pdf&a=Prescribing+Information)\nand Patient Information\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4710891-1&h=1641176897&u=https%3A%2F%2Fd34r3hkxgxjdtw.cloudfront.net%2F6f836309-d95f-42af-b717-2efa058ad82d%2F0a782181-f499-4c71-94b2-67d56ff1110e%2F0a782181-f499-4c71-94b2-67d56ff1110e_source__v.pdf&a=Patient+Information)\n.\n\nAbout BioMarin\n\nBioMarin is a leading, global rare disease biotechnology company focused on\ndelivering medicines for people living with genetically defined conditions.\nFounded in 1997, the San Rafael, California-based company has a proven track\nrecord of innovation, with nine commercial therapies and a strong clinical and\npreclinical pipeline. Using a distinctive approach to drug discovery and\ndevelopment, BioMarin seeks to unleash the full potential of genetic science\nby pursuing category-defining medicines that have a profound impact on\npatients. To learn more, please visit www.biomarin.com\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4710891-1&h=3669995565&u=https%3A%2F%2Fwww.biomarin.com%2F&a=www.biomarin.com)\n.\n\nForward-Looking Statements\n\nThis press release contains forward-looking statements about the business\nprospects of BioMarin Pharmaceutical Inc. (BioMarin), including without\nlimitation, statements about: the data to be presented at ENDO 2026, the\nEndocrine Society Annual Meeting, including oral and poster presentations;\nBioMarin's plans and expectations for the development of VOXZOGO for children\nwith hypochondroplasia, including safety profile and potential benefits and\nplans to submit a supplemental New Drug Application to the U.S. Food and Drug\nAdministration (FDA) during the third quarter of 2026; BioMarin's plans and\nexpectations for the development of BMN 333 for children with achondroplasia,\nincluding plans to provide a data update from the dose finding segment of the\nregistration-enabling Phase 2/3 study of BMN 333 in 2027; the safety profile\nand potential benefits of BMN 333, including the potential to become the new\nstandard of care in achondroplasia; and BioMarin's estimate regarding total\naddressable patient population (TAPP) with respect to the conditions targeted\nby BioMarin's product candidates and commercial products, including\nhypochondroplasia. These forward-looking statements are predictions and\ninvolve risks and uncertainties such that actual results may differ materially\nfrom these statements. These risks and uncertainties include, among others,\nany potential adverse events observed in the continuing monitoring of the\npatients in the clinical trials; the content and timing of decisions by the\nFDA, the European Medicines Agency, the European Commission and other\nregulatory authorities; and those factors detailed in BioMarin's filings with\nthe Securities and Exchange Commission (SEC), including, without limitation,\nthe factors contained under the caption \"Risk Factors\" in BioMarin's Quarterly\nReport on Form 10-Q for the quarter ended March 31, 2026, as such factors may\nbe updated by any subsequent filings with the SEC. Investors are urged not to\nplace undue reliance on forward-looking statements, which speak only as of the\ndate hereof. BioMarin is under no obligation, and expressly disclaims any\nobligation to update or alter any forward-looking statement, whether as a\nresult of new information, future events or otherwise.\n\nBioMarin(®), BioMarin RareConnections(® )and VOXZOGO(®) are registered\ntrademarks of BioMarin Pharmaceutical Inc.\n Contacts:\n\n Investors                                                                      Media\n\n Traci McCarty                                                                  Andrew Villani\n\n BioMarin Pharmaceutical Inc.                                                   BioMarin Pharmaceutical Inc.\n (415) 455-7558                                                                 (628) 269-7393\n\nView original content to download\nmultimedia:https://www.prnewswire.com/news-releases/biomarin-announces-new-three-year-voxzogo-vosoritide-data-in-hypochondroplasia-and-bmn-333-early-results-at-endo-2026-302800847.html\n(https://www.prnewswire.com/news-releases/biomarin-announces-new-three-year-voxzogo-vosoritide-data-in-hypochondroplasia-and-bmn-333-early-results-at-endo-2026-302800847.html)\n\nSOURCE BioMarin Pharmaceutical Inc.\n\n\n\nPhoto: \nhttps://mmx.prnewswire.com/media/MS776208/BioMarin_Pharmaceutical_Inc_Logo.jpg?id=OA2704701\n\nCopyright (c) 2026 PR Newswire Association,LLC. All Rights Reserved.","article_body_html":"","raw_payload":{"data":{"id":"nPn7HxTxfa","title":"BioMarin Announces New Three-Year VOXZOGO® (vosoritide) Data in Hypochondroplasia and BMN 333 Early Results at ENDO 2026","author":"PR Newswire","ticker":"BMRN","created":"2026-06-16T13:05:03.085Z","tickers":["BMRN"],"exchange":"NASDAQ","article_body":"BioMarin Announces New Three-Year VOXZOGO® (vosoritide) Data in Hypochondroplasia and BMN 333 Early Results at ENDO 2026\n\nPR Newswire\n\nSAN RAFAEL, Calif., June 16, 2026\n\nData from an investigator-led study showed treatment with VOXZOGO resulted in\nsustained improvements in growth over three years in children with\nhypochondroplasia\n\nNew Phase 1 results also presented for investigational BMN 333 support\npotential weekly dosing in children with achondroplasia\n\nSAN RAFAEL, Calif., June 16, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc.\n(Nasdaq: BMRN) announced new data from studies of VOXZOGO(®) (vosoritide) in\nchildren with hypochondroplasia and the investigational medicine BMN 333 in\nachondroplasia were presented at ENDO 2026, the Endocrine Society Annual\nMeeting, in Chicago.\n\nNew data from a Phase 2, investigator-sponsored three-year extension study of\nVOXZOGO in 13 children with hypochondroplasia, led by Andrew Dauber, M.D. and\ninvestigators at Children's National Hospital, demonstrated sustained\nimprovements in annualized growth velocity (AGV) and height standard deviation\nscore (SDS), with a favorable safety profile. Mean height SDS improved by 0.72\nSD over three years of treatment, while mean AGV increased from 4.27 cm/year\nat baseline to 7.24 cm/year at year one (p<0.001) and remained above\nbaseline through years two and three.\n\nBioMarin recently announced positive topline results from CANOPY-HCH-3, its\nregistration-enabling Phase 3 pivotal study evaluating VOXZOGO in children\nwith hypochondroplasia. These results will be included in the supplemental New\nDrug Application submission to the U.S. Food and Drug Administration planned\nfor the third quarter of 2026.\n\n\"Building on the excellent Phase 3 results we recently announced, these new\nlonger-term data further reinforce the potential of VOXZOGO to meaningfully\nimprove growth outcomes for children with hypochondroplasia,\" said Greg\nFriberg, M.D., Executive Vice President and Chief Research & Development\nOfficer at BioMarin. \"Importantly, we continue to observe sustained growth\nimprovements over time and a favorable safety profile, adding to the growing\nbody of evidence supporting VOXZOGO for this potential new indication.\"\n\nNew BMN 333 Phase 1 Data Support Advancement Into Late-Stage Development\n\nAdditional data being presented at ENDO 2026 highlight the potential of\ninvestigational BMN 333, BioMarin's long-acting C-type natriuretic peptide\n(CNP) for achondroplasia. In a Phase 1 single-ascending dose study in healthy\nadults, BMN 333 demonstrated sustained systemic exposure associated with\nprolonged pharmacodynamic target engagement, supporting a weekly dosing\nschedule. The maximum examined dose of BMN 333 (500 μg/kg) increased exposure\nto free CNP by more than 13 times compared to another long-acting CNP agent,\nreflecting the potential of BMN 333 to become the new standard of care in\nachondroplasia. BMN 333 was well tolerated across all dose levels evaluated,\nwith no dose-limiting toxicities or treatment-related serious adverse\nevents.\n\nIn April, BioMarin began enrolling patients in the registration-enabling Phase\n2/3 study of BMN 333. A data update from the dose-finding segment of this\nstudy is expected in 2027.\n\nBelow are key presentations for achondroplasia and hypochondroplasia at ENDO,\nwith all times listed in Central Daylight Time:\n\nVosoritide Treatment in Children With Hypochondroplasia: Three-Year Results\nFrom a Phase 2 Extension Trial\nOral Presentation #ORF47-08\nMonday, June 15, 3 – 3:15 p.m.\n\nBMN 333 Achieves High Sustained Released Vosoritide Exposure With Favorable\nSafety: Phase 1 Results That Support Phase 2/3 Trials in Achondroplasia\nPoster Presentation #SUN-212\nSunday, June 14, 9 a.m. – 4 p.m.\n\nAbout Achondroplasia\n\nAchondroplasia, the most common form of skeletal dysplasia leading to\ndisproportionate short stature in humans, is characterized by slowing of\nendochondral ossification, which results in disproportionate short stature and\ndisordered architecture in the long bones, spine, face and base of the skull.\nThis condition is caused by a change in the FGFR3 gene, a negative regulator\nof bone growth.\n\nMore than 80% of children with achondroplasia have parents of average stature\nand have the condition as the result of a spontaneous gene mutation. The\nworldwide incidence rate of achondroplasia is about one in 25,000 live births.\nVOXZOGO is being tested in children whose growth plates are still \"open,\"\ntypically those under 18 years of age. Approximately 25% of people with\nachondroplasia fall into this category.\n\nAbout Hypochondroplasia\n\nHypochondroplasia is a rare, genetic skeletal dysplasia characterized by\nimpaired bone growth, leading to disproportionate short stature and skeletal\ndifferences that can affect the long bones, spine and other parts of the\nskeleton and may impact physical functioning and overall quality of life. The\ncondition presents with a broad and variable clinical spectrum and may include\notolaryngologic (related to the ears, nose and throat) and neurological\ncomplications and is often diagnosed in toddlerhood or early school age based\non clinical and radiological findings. BioMarin estimates that roughly 14,000\nchildren with hypochondroplasia within the company's global footprint may be\neligible for treatment with VOXZOGO.\n\nThere are currently no medicines approved by the U.S. Food and Drug\nAdministration or the European Medicines Agency for the treatment of\nhypochondroplasia.\n\nFor more information about our clinical trials in hypochondroplasia,\nachondroplasia and other skeletal conditions, please visit\nclinicaltrials.biomarin.com\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4710891-1&h=1441447510&u=https%3A%2F%2Fclinicaltrials.biomarin.com%2F&a=clinicaltrials.biomarin.com)\n.\n\nAbout VOXZOGO\n\nIn children with achondroplasia, endochondral bone growth, an essential\nprocess by which bone tissue is created, is negatively regulated due to a gain\nof function mutation in FGFR3. VOXZOGO, a C-type natriuretic peptide (CNP)\nanalog, acts as a positive regulator of the signaling pathway downstream of\nFGFR3 to promote endochondral bone growth.\n\nVOXZOGO is the only approved medicine to support the growth of children with\nachondroplasia starting from birth, with international consensus guidelines\nrecommending initiation of VOXZOGO as early as possible. First approved in\n2021, VOXZOGO has helped more than 5,000 infants and children in more than 50\ncountries. Through our ongoing studies, BioMarin continues to evaluate VOXZOGO\non key clinical endpoints relevant for achondroplasia patients, such as arm\nspan, tibial bowing (leg bowing), body proportionality, spinal morphology\n(including spinal stenosis) and quality of life measures.\n\nVOXZOGO is approved in the U.S., Japan and Australia to increase linear growth\nin children of all ages with achondroplasia with open epiphyses, and VOXZOGO\nis indicated in the EU for the treatment of achondroplasia in children 4\nmonths of age and older whose epiphyses are not closed, as confirmed by\nappropriate genetic testing. In the U.S., this indication is approved under\naccelerated approval based on an improvement in annualized growth velocity.\nContinued approval may be contingent upon verification and description of\nclinical benefit in confirmatory trial(s). To fulfill this post-marketing\nrequirement, BioMarin intends to use the ongoing open-label extension studies\ncompared to available natural history.\n\nThe use of VOXZOGO to treat hypochondroplasia has not yet been approved by any\nregulatory agency.\n\nPatient Support Accessing VOXZOGO\n\nBioMarin's robust support services have ensured a seamless treatment\nexperience, spearheaded by Clinical Coordinators, who have conducted hundreds\nof trainings for families with achondroplasia since approval. BioMarin\nprovides resources to support families navigating achondroplasia, including a\ncaregiver mentorship program that connects parents with other caregivers, and\na U.S. doctor directory that helps families and healthcare professionals\nidentify clinicians experienced in achondroplasia care.\n\nTo reach a BioMarin RareConnections(®) Case Manager, please call, toll-free,\n1-833-VOXZOGO (1-833-869-9646) or e-mail\nVOXZOGOSupport@biomarin-rareconnections.com\n(mailto:VOXZOGOSupport@biomarin-rareconnections.com) . For more information\nabout VOXZOGO, please visit www.voxzogo.com\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4710891-1&h=2227173943&u=http%3A%2F%2Fwww.voxzogo.com%2F&a=www.voxzogo.com)\n. For additional information regarding this product, please contact BioMarin\nMedical Information at medinfo@bmrn.com (mailto:medinfo@bmrn.com) .\n\nVOXZOGO U.S. Important Safety Information\n\nWhat is VOXZOGO used for?\n\n * VOXZOGO is a prescription medicine used to increase linear growth in children\nwith achondroplasia and open growth plates (epiphyses).\n * VOXZOGO is approved under accelerated approval based on an improvement in\nannualized growth velocity. Continued approval may be contingent upon\nverification and description of clinical benefit in confirmatory trials.\nWhat is the most important safety information about VOXZOGO?\n\n * VOXZOGO may cause serious side effects including a temporary decrease in blood\npressure in some patients. To reduce the risk of a decrease in blood pressure\nand associated symptoms (dizziness, feeling tired, or nausea), patients should\neat a meal and drink 8 to 10 ounces of fluid within 1 hour before receiving\nVOXZOGO.\nWhat are the most common side effects of VOXZOGO?\n\n * The most common side effects of VOXZOGO include injection site reactions\n(including redness, itching, swelling, bruising, rash, hives, and injection\nsite pain), high levels of blood alkaline phosphatase shown in blood tests,\nvomiting, joint pain, decreased blood pressure, and stomachache. These are not\nall the possible side effects of VOXZOGO. Ask your healthcare provider for\nmedical advice about side effects, and about any side effects that bother the\npatient or that do not go away.\nHow is VOXZOGO taken?\n\n * VOXZOGO is taken daily as an injection given under the skin, administered by a\ncaregiver after a healthcare provider determines the caregiver is able to\nadminister VOXZOGO. Do not try to inject VOXZOGO until you have been shown the\nright way by your healthcare provider. VOXZOGO is supplied with Instructions\nfor Use that describe the steps for preparing, injecting, and disposing\nVOXZOGO. Caregivers should review the Instructions for Use for guidance and\nany time they receive a refill of VOXZOGO in case any changes have been made.\n * Inject VOXZOGO 1 time every day, at about the same time each day. If a dose of\nVOXZOGO is missed, it can be given within 12 hours from the missed dose. After\n12 hours, skip the missed dose and administer the next daily dose as usual.\n * The dose of VOXZOGO is based on body weight. Your healthcare provider will\nadjust the dose based on changes in weight following regular check-ups.\n * Your healthcare provider will monitor the patient's growth and tell you when\nto stop taking VOXZOGO if they determine the patient is no longer able to\ngrow. Stop administering VOXZOGO if instructed by your healthcare provider.\nWhat should you tell the doctor before or during taking VOXZOGO?\n\n * Tell your doctor about all of the patient's medical conditions including\n* If the patient has heart disease (cardiac or vascular disease), or if the\npatient is on blood pressure medicine (anti-hypertensive medicine).\n * If the patient has kidney problems or renal impairment.\n * If the patient is pregnant or plans to become pregnant. It is not known if\nVOXZOGO will harm the unborn baby.\n * If the patient is breastfeeding or plans to breastfeed. It is not known if\nVOXZOGO passes into breast milk.\n * Tell your doctor about all of the medicines the patient takes, including\nprescription and over-the-counter medicines, vitamins, and herbal supplements.\nYou may report side effects to BioMarin at 1-866-906-6100. You are encouraged\nto report negative side effects of prescription drugs to the FDA. Visit\nwww.fda.gov/medwatch\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4710891-1&h=550579757&u=http%3A%2F%2Fwww.fda.gov%2Fmedwatch&a=www.fda.gov%2Fmedwatch)\n, or call 1-800-FDA-1088.\n\nPlease see additional safety information in the full Prescribing Information\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4710891-1&h=526800756&u=https%3A%2F%2Fd34r3hkxgxjdtw.cloudfront.net%2F6f836309-d95f-42af-b717-2efa058ad82d%2F11d8e586-f965-4525-bc33-93c4b147f11b%2F11d8e586-f965-4525-bc33-93c4b147f11b_source__v.pdf&a=Prescribing+Information)\nand Patient Information\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4710891-1&h=1641176897&u=https%3A%2F%2Fd34r3hkxgxjdtw.cloudfront.net%2F6f836309-d95f-42af-b717-2efa058ad82d%2F0a782181-f499-4c71-94b2-67d56ff1110e%2F0a782181-f499-4c71-94b2-67d56ff1110e_source__v.pdf&a=Patient+Information)\n.\n\nAbout BioMarin\n\nBioMarin is a leading, global rare disease biotechnology company focused on\ndelivering medicines for people living with genetically defined conditions.\nFounded in 1997, the San Rafael, California-based company has a proven track\nrecord of innovation, with nine commercial therapies and a strong clinical and\npreclinical pipeline. Using a distinctive approach to drug discovery and\ndevelopment, BioMarin seeks to unleash the full potential of genetic science\nby pursuing category-defining medicines that have a profound impact on\npatients. To learn more, please visit www.biomarin.com\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4710891-1&h=3669995565&u=https%3A%2F%2Fwww.biomarin.com%2F&a=www.biomarin.com)\n.\n\nForward-Looking Statements\n\nThis press release contains forward-looking statements about the business\nprospects of BioMarin Pharmaceutical Inc. (BioMarin), including without\nlimitation, statements about: the data to be presented at ENDO 2026, the\nEndocrine Society Annual Meeting, including oral and poster presentations;\nBioMarin's plans and expectations for the development of VOXZOGO for children\nwith hypochondroplasia, including safety profile and potential benefits and\nplans to submit a supplemental New Drug Application to the U.S. Food and Drug\nAdministration (FDA) during the third quarter of 2026; BioMarin's plans and\nexpectations for the development of BMN 333 for children with achondroplasia,\nincluding plans to provide a data update from the dose finding segment of the\nregistration-enabling Phase 2/3 study of BMN 333 in 2027; the safety profile\nand potential benefits of BMN 333, including the potential to become the new\nstandard of care in achondroplasia; and BioMarin's estimate regarding total\naddressable patient population (TAPP) with respect to the conditions targeted\nby BioMarin's product candidates and commercial products, including\nhypochondroplasia. These forward-looking statements are predictions and\ninvolve risks and uncertainties such that actual results may differ materially\nfrom these statements. These risks and uncertainties include, among others,\nany potential adverse events observed in the continuing monitoring of the\npatients in the clinical trials; the content and timing of decisions by the\nFDA, the European Medicines Agency, the European Commission and other\nregulatory authorities; and those factors detailed in BioMarin's filings with\nthe Securities and Exchange Commission (SEC), including, without limitation,\nthe factors contained under the caption \"Risk Factors\" in BioMarin's Quarterly\nReport on Form 10-Q for the quarter ended March 31, 2026, as such factors may\nbe updated by any subsequent filings with the SEC. Investors are urged not to\nplace undue reliance on forward-looking statements, which speak only as of the\ndate hereof. BioMarin is under no obligation, and expressly disclaims any\nobligation to update or alter any forward-looking statement, whether as a\nresult of new information, future events or otherwise.\n\nBioMarin(®), BioMarin RareConnections(® )and VOXZOGO(®) are registered\ntrademarks of BioMarin Pharmaceutical Inc.\n Contacts:\n\n Investors                                                                      Media\n\n Traci McCarty                                                                  Andrew Villani\n\n BioMarin Pharmaceutical Inc.                                                   BioMarin Pharmaceutical Inc.\n (415) 455-7558                                                                 (628) 269-7393\n\nView original content to download\nmultimedia:https://www.prnewswire.com/news-releases/biomarin-announces-new-three-year-voxzogo-vosoritide-data-in-hypochondroplasia-and-bmn-333-early-results-at-endo-2026-302800847.html\n(https://www.prnewswire.com/news-releases/biomarin-announces-new-three-year-voxzogo-vosoritide-data-in-hypochondroplasia-and-bmn-333-early-results-at-endo-2026-302800847.html)\n\nSOURCE BioMarin Pharmaceutical Inc.\n\n\n\nPhoto: \nhttps://mmx.prnewswire.com/media/MS776208/BioMarin_Pharmaceutical_Inc_Logo.jpg?id=OA2704701\n\nCopyright (c) 2026 PR Newswire Association,LLC. All Rights Reserved."},"type":"article","timestamp":"2026-06-16T13:05:03.129407948Z","server_sent_at_ms":1781615103129},"received_at":"2026-06-16T13:05:03.295Z","source_url":"https://www.prnewswire.com/news-releases/biomarin-announces-new-three-year-voxzogo-vosoritide-data-in-hypochondroplasia-and-bmn-333-early-results-at-endo-2026-302800847.html"},"analysis":{"id":"54583","press_release_id":"65165","analysis_json":{"industry":{"label":"Biotechnology","sector":"Health Care"},"redFlags":[],"eventType":"clinical_trial","narrative":"BioMarin presented three-year data showing sustained growth improvements and a favorable safety profile for VOXZOGO in children with hypochondroplasia, supporting a planned supplemental New Drug Application in the third quarter of 2026.\n\nIn the Phase 2 extension study, mean height standard deviation score improved by 0.72 SD and annualized growth velocity increased significantly from baseline to year one.\n\nEarly Phase 1 data for investigational BMN 333 demonstrated sustained exposure supporting a weekly dosing schedule for achondroplasia, with exposure levels increasing more than 13 times compared to other long-acting agents.","sentiment":"bullish","agentHooks":{"shouldPost":true,"suggestedAngle":"VOXZOGO label expansion into hypochondroplasia gains momentum with robust 3-year data; BMN 333 shows promise for weekly dosing."},"keyFigures":{"guidance":"sNDA submission for VOXZOGO in hypochondroplasia planned for Q3 2026; BMN 333 Phase 2/3 data update expected in 2027","customDimensions":{"bmn_333_max_dose":"500 μg/kg","voxzogo_agv_year1":"7.24 cm/year","voxzogo_agv_baseline":"4.27 cm/year","cnp_exposure_increase":">13 times","voxzogo_sds_improvement":"0.72 SD","hypochondroplasia_patient_pool":"14,000"}},"quotedText":"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","namedEntities":{"people":[{"name":"Andrew Dauber, M.D.","role":"Investigator, Children's National Hospital"},{"name":"Greg Friberg, M.D.","role":"Executive Vice President and Chief Research & Development Officer"},{"name":"Traci McCarty","role":"Investor Contact"},{"name":"Andrew Villani","role":"Media Contact"}],"products":["VOXZOGO","vosoritide","BMN 333"],"companies":[{"name":"BioMarin Pharmaceutical Inc.","ticker":"BMRN"},{"name":"Children's National Hospital","relationship":"research partner"}],"dollarAmounts":[]},"materialImpact":{"score":4,"reasoning":"Positive three-year extension data for VOXZOGO in hypochondroplasia supports the planned Q3 2026 sNDA submission for a major label expansion of a commercial product. Early BMN 333 data de-risks the pipeline with a convenient weekly dosing profile."},"tickerRelevance":{"others":[],"primary":"BMRN"},"globalImportance":45,"audienceRelevance":45,"eventTypeSecondary":[],"importanceComponents":{"tickerTier":"mid-cap biotech","eventGravity":"label expansion data readout","commercialAsset":"VOXZOGO"}},"event_type":"clinical_trial","event_type_secondary":null,"sentiment":"bullish","material_impact_score":4,"narrative":"BioMarin presented three-year data showing sustained growth improvements and a favorable safety profile for VOXZOGO in children with hypochondroplasia, supporting a planned supplemental New Drug Application in the third quarter of 2026.\n\nIn the Phase 2 extension study, mean height standard deviation score improved by 0.72 SD and annualized growth velocity increased significantly from baseline to year one.\n\nEarly Phase 1 data for investigational BMN 333 demonstrated sustained exposure supporting a weekly dosing schedule for achondroplasia, with exposure levels increasing more than 13 times compared to other long-acting agents.","key_figures":{"guidance":"sNDA submission for VOXZOGO in hypochondroplasia planned for Q3 2026; BMN 333 Phase 2/3 data update expected in 2027","customDimensions":{"bmn_333_max_dose":"500 μg/kg","voxzogo_agv_year1":"7.24 cm/year","voxzogo_agv_baseline":"4.27 cm/year","cnp_exposure_increase":">13 times","voxzogo_sds_improvement":"0.72 SD","hypochondroplasia_patient_pool":"14,000"}},"named_entities":{"people":[{"name":"Andrew Dauber, M.D.","role":"Investigator, Children's National Hospital"},{"name":"Greg Friberg, M.D.","role":"Executive Vice President and Chief Research & Development Officer"},{"name":"Traci McCarty","role":"Investor Contact"},{"name":"Andrew Villani","role":"Media Contact"}],"products":["VOXZOGO","vosoritide","BMN 333"],"companies":[{"name":"BioMarin Pharmaceutical Inc.","ticker":"BMRN"},{"name":"Children's National Hospital","relationship":"research partner"}],"dollarAmounts":[]},"model_name":"qwen3_6_27b_awq","prompt_hash":"sha256:727b4b9429a443af","schema_hash":"sha256:05005c02d9cffac9","created_at":"2026-06-16T14:21:43.441Z","global_importance":45,"audience_relevance":45,"importance_components":{"tickerTier":"mid-cap biotech","eventGravity":"label expansion data readout","commercialAsset":"VOXZOGO"}},"durationMs":79774,"modelName":"george-droid-qwen-72b"}}