BioMarin Presents New Data Demonstrating Favorable Safety and Strong Adherence in Real-World Clinical Practice with VOXZOGO® (vosoritide) in Children Under 3 with Achondroplasia at 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting

PR Newswire

03-20

New data also to be presented for PALYNZIQ® (pegvaliaise-pqpz), reinforcing its value in sustaining blood Phe level reduction and improving health-related quality of life for adults with ...

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}\n.meta {color:#5e5c6d;font-size:13px;margin:0 0 .5em; }\na{text-decoration:none; color:#2a4b87;}\n.meta .head { display: inline-block; overflow: hidden}\n.head .h-thumb { width: 30px; height: 30px; margin: 0; padding: 0; border-radius: 50%; float: left;}\n.head .h-content { margin: 0; padding: 0 0 0 9px; float: left;}\n.head .h-name {font-size: 13px; color: #eee; margin: 0;}\n.head .h-time {font-size: 11px; color: #7E829C; margin: 0;line-height: 11px;}\n.small {font-size: 12.5px; display: inline-block; transform: scale(0.9); -webkit-transform: scale(0.9); transform-origin: left; -webkit-transform-origin: left;}\n.smaller {font-size: 12.5px; display: inline-block; transform: scale(0.8); -webkit-transform: scale(0.8); transform-origin: left; -webkit-transform-origin: left;}\n.bt-text {font-size: 12px;margin: 1.5em 0 0 0}\n.bt-text p {margin: 0}\n</style>\n</head>\n<body>\n<div class=\"wrapper\">\n<header>\n<h2 class=\"title\">\nBioMarin Presents New Data Demonstrating Favorable Safety and Strong Adherence in Real-World Clinical Practice with VOXZOGO® (vosoritide) in Children Under 3 with Achondroplasia at 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting\n</h2>\n\n<h4 class=\"meta\">\n\n\n2025-03-20 21:00 GMT+8&nbsp;&nbsp;&nbsp;<a href=https://finance.yahoo.com/news/biomarin-presents-data-demonstrating-favorable-130000558.html><strong>PR Newswire</strong></a>\n\n\n</h4>\n\n</header>\n<article>\n<div>\n<p>New data also to be presented for PALYNZIQ® (pegvaliaise-pqpz), reinforcing its value in sustaining blood Phe level reduction and improving health-related quality of life for adults with ...</p>\n\n<a href=\"https://finance.yahoo.com/news/biomarin-presents-data-demonstrating-favorable-130000558.html\">网页链接</a>\n\n</div>\n\n\n</article>\n</div>\n</body>\n</html>\n","isBrief":false,"type":0,"news_type":1,"symbol":null,"symbol_name":null,"start_time":0,"source_url":"https://finance.yahoo.com/news/biomarin-presents-data-demonstrating-favorable-130000558.html","article_id":"2520956457","we_media_id":null,"thumbnails":[],"rights":{"source":"yahoofinance","url":"https://finance.yahoo.com/news/biomarin-presents-data-demonstrating-favorable-130000558.html","rn_cache_url":null,"customStyle":"img, svg { display: none !important; }","selectors":"article","filters":"","directOrigin":true},"url":"https://stock-news.laohu8.com/highlight/detail?id=2520956457","pubTimestamp":1742475600,"columns":[],"sourceInfo":{"source_id":"yahoofinance","name":"雅虎金融"},"weMediaInfo":null,"summary":"New data also to be presented for PALYNZIQ , reinforcing its value in sustaining blood Phe level reduction and improving health-related quality of life for adults with phenylketonuria. SAN RAFAEL, Calif., March 20, 2025 /PRNewswire/ -- BioMarin Pharmaceutical Inc.  today announced positive new data from studies of VOXZOGO in children with achondroplasia and in ongoing clinical trials investigating other skeletal conditions, as well as PALYNZIQ in adults with phenylketonuria . The latest findings further validate the long-term benefit and established safety profiles of both medicines, which continue to play a pivotal role for people living with these genetically defined conditions.PALYNZIQ Data Show Importance of Sustained Blood Phe Level Reduction and Improvement in Quality of Life in Adults with PKU","collect":0,"end_time":0,"defaultTopTitle":"yahoo.com","property":["rate"],"viewcount":null,"language":"en","relate_stocks":{},"translate_title":"BioMarin在2025年美国医学遗传学和基因组学学院(ACMG)年度临床遗传学会议上展示了新数据，证明VOXZOGO®(vosoritide)在3岁以下软骨发育不全儿童的真实临床实践中具有良好的安全性和强依从性","themeId":null,"isJumpTheme":false,"ttsUrl":null,"symbols_score_info":{"AFG":1,"BMRN":0.9},"content_text":"New data also to be presented for PALYNZIQ® (pegvaliaise-pqpz), reinforcing its value in sustaining blood Phe level reduction and improving health-related quality of life for adults with phenylketonuria\nSAN RAFAEL, Calif., March 20, 2025 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced positive new data from studies of VOXZOGO® (vosoritide) in children with achondroplasia and in ongoing clinical trials investigating other skeletal conditions, as well as PALYNZIQ® (pegvaliase-pqpz) in adults with phenylketonuria (PKU). The latest findings further validate the long-term benefit and established safety profiles of both medicines, which continue to play a pivotal role for people living with these genetically defined conditions.\n\n  \n\nThe data will be presented at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting in Los Angeles.\nData Highlight VOXZOGO Treatment Adherence and Favorable Safety Profile in Infants\nNew data from a study in Japan showed strong treatment adherence with VOXZOGO in children with achondroplasia under the age of 3, with no reported treatment-related adverse events nor any dose interruptions among 63 children followed for up to 23.7 months. These real-world findings validate VOXZOGO's established safety profile and reinforce the therapeutic benefit seen in clinical studies. The study's safety results, including in infants as young as 1 month old, add to the growing body of evidence supporting early treatment initiation.\n\"VOXZOGO is the first and only approved treatment for children with achondroplasia, and it is encouraging to see favorable safety and strong adherence in very young children receiving the medicine in real-world clinical practice. As the effects of restricted growth are apparent from birth in children with achondroplasia, we expect that earlier treatment should translate to greater benefits,\" said Greg Friberg, M.D., executive vice president and chief research & development officer at BioMarin. \"In addition to data in achondroplasia, scientific research at ACMG furthers our genetic understanding of skeletal conditions like hypochondroplasia, in which we hope to complete enrollment for our pivotal study with VOXZOGO in the first half of 2025.\"\nPALYNZIQ Data Show Importance of Sustained Blood Phe Level Reduction and Improvement in Quality of Life in Adults with PKU\nNew data from the OPAL study, a post-marketing observational trial to assess the real-world safety and efficacy of PALYNZIQ, showed lowered blood Phe levels following treatment and positive health-related quality of life (HRQoL) outcomes. Mean blood Phe level was 1029 μmol/L at baseline (n=51) and lowered to 293 μmol/L at week 96 (n=16), representing a 67.8% reduction. Improvements in HRQoL were also observed, as measured by the PKU quality of life questionnaire (PKU-QOL) and the PKU Symptom Severity and Impacts Scale (PKU-SSIS), which use a scale of 1-100, with lower scores indicating better HRQoL. Mean change from baseline of -12.4 and -11.8 respectively were observed over the same time period.\nAdditionally, secondary data analyses from the Phase 3 PRISM clinical trial program demonstrated that sustaining lowered blood Phe levels with PALYNZIQ led to improvements in attention and mood. Most notably, scores for sustained Phe levels ≤120 were significantly better than those at sustained levels of ≤600 or ≤360, suggesting blood Phe in the normal range may provide additional benefit for adults living with PKU.\nReal-world data from the Assessment of the Treatment and management LAndScape of PKU (ATLAS) study also highlighted a shift in the treatment landscape in part attributable to PALYNZIQ, with an increased proportion of people with PKU treated across 19 U.S. clinics achieving blood Phe levels ≤360 μmol/L and a reduction in the proportion of individuals with blood Phe levels >1200 μmol/L.\nThese findings demonstrate PALYNZIQ's efficacy in treating PKU, as the company works to advance this option for younger people living with the genetically defined condition, with results from a Phase 3 study in adolescents expected later this year.\nBelow are BioMarin's key presentations at ACMG, all listed in Pacific Time:\nInterdisciplinary Variant Re-Classification: FGFR3 as an Example of Genotypic Investigation in Suspected Skeletal Dysplasia PopulationPoster #P213Thursday, March 20, 10:30 – 11:30 a.m.\nHow Helpful are Sleep Studies in Determining Surgical Need in Infants with Achondroplasia?Poster #P313Thursday, March 20, 10:30 – 11:30 a.m.\nBeyond Boundaries: The Continuous Spectrum in FGFR3-Related ConditionsPoster #P373Thursday, March 20, 10:30 – 11:30 a.m.\nIndex of Sustained Phe Response and Improvements in PKU Clinical Outcome Assessments in Patients Receiving PegvaliasePoster #P009Thursday, March 20, 10:30 – 11:30 a.m.\nInitial Psychometric Evaluation of the Adult Symptom Severity and Impacts Scale (PKU-SSIS) Using Interim Data from the OPAL StudyPoster #P029Thursday, March 20, 10:30 – 11:30 a.m. \nThe Assessment of the Treatment and Management Landscape of Phenylketonuria Survey Study: Findings from 19 Clinics in the United StatesPoster #P045Thursday, March 20, 10:30 – 11:30 a.m.\nSafety Profile and Adherence of Vosoritide in Young Children with Achondroplasia in JapanPoster #P208Friday, March 21, 10:30 – 11:30 a.m.\nVosoritide as a Targeted Therapy for FGFR3-Related Thanatophoric DysplasiaPoster #P354Friday, March 21, 10:30 – 11:30 a.m.\nImprovements in Blood Phenylalanine and Health-Related Quality of Life Outcomes Among Adults with PKU Receiving Pegvaliase in the OPAL Study Poster #P002Friday, March 21, 10:30 – 11:30 a.m.\nOccurrence of Anaphylaxis in Adult Incident Pegvaliase-Treated PKU Patients in a Post-Marketing Safety Analysis in the United StatesPoster #P046Friday, March 21, 10:30 – 11:30 a.m.\nAbout Achondroplasia\nAchondroplasia, 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.\nMore 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.\nAbout Phenylketonuria\nPKU, or phenylalanine hydroxylase (PAH) deficiency, is a genetic condition affecting approximately 70,000 people in the regions of the world where BioMarin operates. This enzyme is required for the metabolism of Phe, an essential amino acid found in most protein-containing foods. If functional enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe intellectual disability, seizures, tremors, behavioral problems and psychiatric symptoms.\nAs a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all individuals with PKU born after this period in countries with newborn screening programs are diagnosed at birth and treatment is implemented soon after.\nPKU can be managed with a severe Phe-restricted diet, which is supplemented by low-protein modified foods and Phe-free medical foods; however, it is difficult for most individuals to adhere to the lifelong strict diet to the extent needed to achieve adequate control of blood Phe levels. Dietary control of Phe in childhood can prevent major developmental neurological toxicities, but poor control of Phe in adolescence and adulthood is associated with a range of neurocognitive disabilities with significant functional impact.\nAbout VOXZOGO\nIn 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.\nVOXZOGO 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.\nPatient Support Accessing VOXZOGO\nTo 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.\nAbout PALYNZIQ\nPALYNZIQ substitutes the deficient phenylalanine hydroxylase (PAH) enzyme in PKU with a PEGylated version of the enzyme phenylalanine ammonia lyase to break down Phe. PALYNZIQ is administered using a dosing regimen designed to facilitate tolerability; PALYNZIQ's safety profile consists primarily of immune-mediated responses, which can include anaphylaxis, for which robust risk management measures effective in clinical trials are in place.\nPALYNZIQ is approved to reduce blood Phe concentrations for adults in the U.S., for people 16 and older in the EU, Canada and Brazil, and for people 15 and older in Japan to reduce blood Phe concentrations in individuals with PKU who have uncontrolled blood Phe concentrations greater than 600 micromol/L on existing management.\nPatient Support Accessing PALYNZIQ\nTo reach a BioMarin RareConnections® Case Manager, please call, toll-free, 1-866-906-6100 or e-mail support@biomarin-rareconnections.com. For more information about PALYNZIQ, please visit www.palynziq.com. For additional information regarding this product, please contact BioMarin Medical Information at medinfo@bmrn.com.\nVOXZOGO U.S. Important Safety Information\nWhat is VOXZOGO used for?\n\nVOXZOGO is a prescription medicine used to increase linear growth in children with achondroplasia and open growth plates (epiphyses).\nVOXZOGO 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.\n\nWhat is the most important safety information about VOXZOGO?\n\nVOXZOGO 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.\n\nWhat are the most common side effects of VOXZOGO?\n\nThe 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.\n\nHow is VOXZOGO taken?\n\nVOXZOGO 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.\nInject 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.\nThe dose of VOXZOGO is based on body weight. Your healthcare provider will adjust the dose based on changes in weight following regular check-ups.\nYour 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.\n\nWhat should you tell the doctor before or during taking VOXZOGO?\n\nTell your doctor about all of the patient's medical conditions including\n   \nIf the patient has heart disease (cardiac or vascular disease), or if the patient is on blood pressure medicine (anti-hypertensive medicine).\nIf the patient has kidney problems or renal impairment.\nIf the patient is pregnant or plans to become pregnant. It is not known if VOXZOGO will harm the unborn baby.\nIf the patient is breastfeeding or plans to breastfeed. It is not known if VOXZOGO passes into breast milk.\n\nTell your doctor about all of the medicines the patient takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements.\n\nYou 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.\nPlease see additional safety information in the full Prescribing Information and Patient Information.\nPALYNZIQ U.S. Indication and Important Safety Information\nPALYNZIQ® (pegvaliase-pqpz) is a phenylalanine (Phe)-metabolizing enzyme indicated to reduce blood Phe levels in adult patients with phenylketonuria who have uncontrolled blood Phe levels greater than 600 micromol/L on existing management.\nBOXED WARNING: RISK OF ANAPHYLAXIS\n\nAnaphylaxis has been reported after administration of PALYNZIQ and may occur at any time during treatment\nAdminister the initial dose of PALYNZIQ under the supervision of a healthcare provider equipped to manage anaphylaxis, and closely observe patients for at least 60 minutes following injection. Prior to self-injection, confirm patient competency with self-administration, and patient's and observer's (if applicable) ability to recognize signs and symptoms of anaphylaxis and to administer auto-injectable epinephrine, if needed\nConsider having an adult observer for patients who may need assistance in recognizing and managing anaphylaxis during PALYNZIQ treatment. If an adult observer is needed, the observer should be present during and for at least 60 minutes after PALYNZIQ administration, should be able to administer auto-injectable epinephrine, and call for emergency medical support upon its use\nPrescribe auto-injectable epinephrine. Prior to the first dose, instruct the patient and observer (if applicable) on its appropriate use. Instruct the patient to seek immediate medical care upon its use. Instruct patients to carry auto-injectable epinephrine with them at all times during PALYNZIQ treatment\nPALYNZIQ is available only through a restricted program called PALYNZIQ REMS (Risk Evaluation and Mitigation Strategy). Further information, including a list of qualified pharmacies, is available at www.PALYNZIQREMS.com or by telephone at 1-855-758-REMS (1-855-758-7367)\n\nWARNINGS AND PRECAUTIONS\nAnaphylaxis\n\nSigns and symptoms of anaphylaxis reported include syncope, hypotension, hypoxia, dyspnea, wheezing, chest discomfort/chest tightness, tachycardia, angioedema (swelling of face, lips, eyes, tongue), throat tightness, skin flushing, rash, urticaria, pruritus, and gastrointestinal symptoms (vomiting, nausea, diarrhea)\nAnaphylaxis generally occurred within 1 hour after injection; however, delayed episodes occurred up to 48 hours after PALYNZIQ administration\nConsider having an adult observer for patients who may need assistance in recognizing and managing anaphylaxis during PALYNZIQ treatment. If an adult observer is needed, the observer should be present during and for at least 60 minutes after PALYNZIQ administration, should be able to administer auto-injectable epinephrine, and call for emergency medical support upon its use\nAnaphylaxis requires immediate treatment with auto-injectable epinephrine. Prescribe auto-injectable epinephrine to all patients receiving PALYNZIQ and instruct patients to carry auto-injectable epinephrine with them at all times during PALYNZIQ treatment. Prior to the first dose, instruct the patient and observer (if applicable) on how to recognize the signs and symptoms of anaphylaxis, how to properly administer auto-injectable epinephrine, and to seek immediate medical care upon its use. Consider the risks associated with auto-injectable epinephrine use when prescribing PALYNZIQ. Refer to the auto-injectable epinephrine prescribing information for complete information\nConsider the risks and benefits of readministering PALYNZIQ following an episode of anaphylaxis. If the decision is made to readminister PALYNZIQ, administer the first dose under the supervision of a healthcare provider equipped to manage anaphylaxis and closely observe the patient for at least 60 minutes following the dose. Subsequent PALYNZIQ dose titration should be based on patient tolerability and therapeutic response\nConsider premedication with an H1-receptor antagonist, H2-receptor antagonist, and/or antipyretic prior to PALYNZIQ administration based upon individual patient tolerability\n\nOther Hypersensitivity Reactions\n\nHypersensitivity reactions other than anaphylaxis have been reported in 204 of 285 (72%) patients treated with PALYNZIQ in clinical trials\nManagement of hypersensitivity reactions should be based on the severity of the reaction, recurrence of the reaction, and the clinical judgment of the healthcare provider, and may include dosage adjustment, temporary drug interruption, or treatment with antihistamines, antipyretics, and/or corticosteroids\n\nADVERSE REACTIONS\n\nThe most common adverse reactions (at least 20% of patients in either treatment phase) were injection site reactions, arthralgia, hypersensitivity reactions, headache, generalized skin reactions lasting at least 14 days, nausea, abdominal pain, vomiting, cough, oropharyngeal pain, pruritus, diarrhea, nasal congestion, fatigue, dizziness, and anxiety\nOf the 285 patients exposed to PALYNZIQ in an induction/titration/maintenance regimen in clinical trials, 44 (15%) patients discontinued treatment due to adverse reactions. The most common adverse reactions leading to treatment discontinuation were hypersensitivity reactions (6% of patients) including anaphylaxis (3% of patients), angioedema (1% of patients), arthralgia (4% of patients), generalized skin reactions lasting at least 14 days (2% of patients), and injection site reactions (1% of patients)\nThe most common adverse reactions leading to dosage reduction were arthralgia (15% of patients), hypersensitivity reactions (9% of patients), injection site reactions (4% of patients), alopecia (3% of patients), and generalized skin reactions lasting at least 14 days (2% of patients)\nThe most common adverse reactions leading to temporary drug interruption were hypersensitivity reactions (14% of patients), arthralgia (13% of patients), anaphylaxis (4% of patients), and injection site reactions (4% of patients)\nAngioedema and serum sickness: In clinical trials, 22 out of 285 (8%) patients experienced 45 episodes of angioedema (symptoms included: pharyngeal edema, swollen tongue, lip swelling, mouth swelling, eyelid edema, and face edema) occurring independent of anaphylaxis. In clinical trials, serum sickness was reported in 7 out of 285 (2%) patients\n\nBlood Phenylalanine Monitoring and Diet\n\nObtain blood Phe levels every 4 weeks until a maintenance dosage is established. Periodically monitor blood Phe levels during maintenance therapy\nCounsel patients to monitor dietary protein and Phe intake, and adjust as directed by their healthcare provider\n\nDRUG INTERACTIONS\nEffect of PALYNZIQ on Other PEGylated Products\n\nIn a single-dose study of PALYNZIQ in adult patients with PKU, two patients receiving concomitant injections of medroxyprogesterone acetate suspension (a formulation containing PEG 3350) experienced a hypersensitivity reaction. One of the two patients experienced anaphylaxis\nThe clinical effects of concomitant treatment with different PEGylated products is unknown. Monitor patients treated with PALYNZIQ and concomitantly with other PEGylated products for hypersensitivity reactions including anaphylaxis\n\nUSE IN SPECIFIC POPULATIONS\nPregnancy and Lactation\n\nPALYNZIQ may cause fetal harm when administered to a pregnant woman\nAdvise women who are exposed to PALYNZIQ during pregnancy or who become pregnant within one month following the last dose of PALYNZIQ that there is a pregnancy surveillance program that monitors pregnancy outcomes. Healthcare providers should report PALYNZIQ exposure and encourage these patients to report their pregnancy to BioMarin (1-866-906-6100)\nMonitor blood Phe levels in breastfeeding women treated with PALYNZIQ\n\nPediatric Use\n\nThe safety and effectiveness of PALYNZIQ in pediatric patients have not been established\n\nGeriatric Use\n\nClinical studies of PALYNZIQ did not include patients aged 65 years and older\n\nYou are encouraged to report suspected adverse reactions to BioMarin at 1-866-906-6100, or to FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.\nPlease see accompanying full Prescribing Information, including Boxed Warning.\nAbout BioMarin\nBioMarin is a global biotechnology company dedicated to translating the promise of genetic discovery into medicines that make a profound impact on the life of each patient. The San Rafael, California-based company, founded in 1997, has a proven track record of innovation with eight 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 offer new possibilities for people living with genetically defined conditions around the world. To learn more, please visit www.biomarin.com.\nForward-Looking Statements\nThis press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: data to be presented at the 2025 American College of Medical Genetics and Genomics Annual Clinical Genetics Meeting, including the ten poster presentations; the development of BioMarin's VOXZOGO program generally, including expectation to complete enrollment of the pivotal study with VOXZOGO in hypochondroplasia in the first half of 2025; the safety profile and potential benefits of VOXZOGO for children with achondroplasia, including the expectation that earlier treatment should lead to potentially greater benefits; the development of BioMarin's PALYNZIQ program generally; BioMarin's plans to advance PALYNZIQ for the treatment of adolescents with phenylketonuria (PKU), including expectation that results from a Phase 3 study will be available later this year; the benefits of PALYNZIQ for adults with PKU, including potential improvement in health-related quality of life; and the continued clinical development of VOXZOGO and PALYNZIQ. 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: results and timing of current and planned preclinical studies and clinical trials of VOXZOGO and PALYNZIQ; any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the U.S. Food and Drug Administration, the European Medicines Agency, the European Commission and other regulatory authorities; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption \"Risk Factors\" in BioMarin's Annual Report on Form 10-K for the year ended December 31, 2024, as such factors may be updated by any subsequent reports. Stockholders 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.\nBioMarin®, BioMarin RareConnections®, VOXZOGO® and PALYNZIQ® are registered trademarks of BioMarin Pharmaceutical Inc.\n\n\n\n\nContacts:     \n\n\n\n\n\n\n\nInvestors \nMedia \n\n\nTraci McCarty \nAndrew Villani \n\n\nBioMarin Pharmaceutical Inc. \nBioMarin Pharmaceutical Inc. \n\n\n(415) 455-7558 \n(628) 269-7393 \n\n\n\n\n View original content to download multimedia:https://www.prnewswire.com/news-releases/biomarin-presents-new-data-demonstrating-favorable-safety-and-strong-adherence-in-real-world-clinical-practice-with-voxzogo-vosoritide-in-children-under-3-with-achondroplasia-at-2025-american-college-of-medical-genetics-and-gen-302406448.html\nSOURCE BioMarin Pharmaceutical Inc.","kind":"highlight","is_publish_news":true,"is_publish_highlight":false,"is_publish_live":false,"is_publish_wemedia":null,"editions":null,"column":"","sentiment":"1","news_tag":"dataReport","news_rank":0,"symbols":[],"gpt_button":1,"code":"91000000","status":"200"}}}