Biomarin Vosoritide Phase 3, (NASDAQ: BMRN) today announced that Lynda Polgreen, MD, MS, Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, Torrance, CA, USA will present data from the randomized, double-blind, phase 3, placebo-controlled, multicenter trial for vosoritide, an investigational analog of C-type Trial Summary The intent and design of this Phase 3 study is to assess vosoritide as a therapeutic option for the treatment of children with hypochondroplasia (HCH). (Nasdaq: BMRN) today announced positive and consistent results from multiple real-world evidence studies of VOXZOGO ® (vosoritide) in children with achondroplasia. SAN RAFAEL, Calif. Data on file [3]. 16, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. 16th Dec 2019 BioMarin reported positive final results from the Phase 3 clinical trial evaluating the efficacy and safety of vosoritide (BMN 111) in children with achondroplasia. Step-by-step guide to get Voxzogo (vosoritide) covered by UnitedHealthcare in Georgia. BBIO Price Action: BridgeBio shares are up 6. The company also shared data from ongoing investigational studies evaluating VOXZOGO for hypochondroplasia. (Nasdaq: BMRN) announced that positive new data supporting the safety and efficacy of VOXZOGO ® (vosoritide) in children with achondroplasia, as well as positive data for investigational uses in growth-related conditions, including idiopathic short stature (ISS) and Noonan syndrome, were presented at the 2024 Pediatric Endocrine Society (PES) Annual Meeting in A phase 3, randomized, placebo-controlled trial (BMN 111-301; NCT03197766) in children with ACH showed a statistically significant improvement in AGV with vosoritide after 52 weeks compared to placebo8; AGV improvement was sustained after 2 years of vosoritide treatment in the extension study (BMN 111-302; NCT03424018)9 BioMarin Pharmaceutical Inc. , March 3, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. Methods: This randomised, double-blind, phase 3, placebo-controlled, multicentre trial compared once-daily subcutaneous administration of vosoritide with placebo in children with achondroplasia. , Nov. The stock hit a high of $84. The placebo-adjusted change from baseline in growth velocity after one year of treatment with vosoritide, the primary endpoint, was 1. The diagnosis of achondroplasia should be confirmed by appropriate genetic testing. The primary endpoint of the study was the change in growth velocity from baseline over one year in children treated with vosoritide compared to placebo. The trial was done in hospitals at 24 sites in seven countries (Australia, Germany, Japan, Spain, Turkey, the USA, and the UK). 8, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. New Investigator-Led Study Shows VOXZOGO Significantly Increased Bone Length While Maintaining Bone Strength Through 5 Years of Observation in Children with Achondroplasia Phase 2 and 3 Data on VOXZOGO Demonstrate Safety, Efficacy and Impact on Proportionality and Health-Related Quality of Life for Children with Achondroplasia New Research Underscores Unmet Medical Needs of People with /PRNewswire/ -- BioMarin Pharmaceutical Inc. 11, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced new data from studies of VOXZOGO ® (vosoritide), demonstrating meaningful impact on tibial bowing in children with achondroplasia and investigational early efficacy results in other skeletal conditions. 6 cm/yr (p&l… BridgeBio stock rises after Phase 3 trial shows its drug helped children with achondroplasia grow faster, with no serious safety concerns. Vosoritide acts to restore chondrogenesis through its BioMarin Pharmaceutical. May 9, 2025 · Here, we report the long-term safety, tolerability, and efficacy (including effects on linear growth and proportionality) in children with achondroplasia from the phase 3, open-label extension study (study 111-302), treated with daily subcutaneous injections of vosoritide for up to 6 years. 41 at the time of publication on Thursday. 16, 2019 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today reported positive final results from its randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of vosoritide. VOXZOGO ® Product Monograph. Food and Drug Administration (FDA) has approved the supplemental New Drug Sustained growth-promoting effects of vosoritide in children with achondroplasia from an ongoing phase 3 extension study In April, BioMarin completed enrollment in its pivotal Phase 3 study with VOXZOGO in hypochondroplasia, and the company is on track to share topline data in 2026, with potential launch in 2027. , Dec. gov: NCT03424018) wherein all received 15 μg/kg vosoritide daily. Additionally, clinical studies in children with ISS (111-903 and 111-210) and multiple genetic short stature pathway Four years after BioMarin Pharmaceutial's vosoritide posted proof-of-concept data, the drug hit the mark in phase 3, helping children with achondroplasia, the most common form of dwarfism, grow BioMarin conducted a randomized, double-blind, placebo-controlled Phase 2 clinical trial evaluating the safety and efficacy of VOXZOGO in children aged 5 and under (Study 111-206). Results from the phase 3 clinical trial for vosoritide, that involved children with achondroplasia with ages between 5 to 18 years have been published. , Oct. If confirmed through regulatory review, the PROPEL 3 Phase III results would position BridgeBio infigratinib as a direct competitor to BioMarin’s injectable vosoritide (Voxzogo), which has been the sole approved pharmacotherapy for achondroplasia since 2021. S. Data on file [2]. Sustained growth-promoting effects of vosoritide in children with achondroplasia from an ongoing phase 3 extension study. 2025;21:314–324. (The enrollment age criteria were 5 The Company provided data from a Phase 2 randomized, double-blind, placebo-controlled clinical trial to evaluate the safety and efficacy of VOXZOGO™ (vosoritide) for Injection in infants and BioMarin Pharmaceutical Inc. Eligible patients had a clinical diagnosis of . (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced that the U. Children younger than 60 months with a clinical diagnosis of achondroplasia confirmed by genetic testing and who had completed a baseline growth study or observation period were enrolled into one of three sequential cohorts The purpose of this study is to evaluate i) the effect of multiple doses of vosoritide and ii) the effect of the therapeutic dose of vosoritide compared to human growth hormone (hGH), in children with idiopathic short stature (ISS). 1,3 About BioMarin BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. The data were presented at the 2025 Joint Congress of the European Society for Paediatric Endocrinology (ESPE) and the European SAN RAFAEL, Calif. 's (NASDAQ: BMRN) Voxzogo (vosoritide) was the first FDA-approved drug (November 2021) for achondroplasia. Methods: This double-blind, randomised, placebo-controlled, phase 2 trial was done in 16 hospitals across Australia, Japan, the UK, and the USA. 7 BioMarin Pharmaceutical Inc. The global Phase 3 study was a randomized, double-blind, placebo-controlled study of vosoritide in 121 children with achondroplasia aged 5 to 14 for 52 weeks. (NASDAQ: BMRN) today announced that The Lancet has published online results from a randomized, double-blind, phase 3, placebo-controlled, multicenter trial for vosoritide, an investigational analog of C-type Natriuretic Peptide (CNP), in children aged 5 to 18 years with achondroplasia. 2025;6 (5):100566. BridgeBio looks to challenge BioMarin in dwarfism market on Phase III win If infigratinib gains approval, the drug could challenge BioMarin’s predicted blockbuster, Voxzogo, on the achondroplasia market. The company plans regulatory filings in 2026. A multinational observational study in children with hypochondroplasia (111-902) is currently recruiting participants, and the company plans to enter the treatment phase (Phase 3 trial) by mid-year. These results, as well as data from the investigational research program for VOXZOGO in hypochondroplasia, were presented at the 62nd Annual European Oct 20, 2023 Expanded Indication in the U. BioMarin Pharmaceutical Inc; 2025. Achondroplasia is the most May 9, 2025 · This work was funded by BioMarin Pharmaceutical. Vosoritide (VOXZOGO®) is a modified recombinant human C-type natriuretic peptide (CNP) analogue, being developed by BioMarin Pharmaceutical for the treatment of achondroplasia. Achondroplasia Pipeline 2025: Latest FDA Approvals, Clinical Trials, And Emerging Therapies Assessment By Delveinsight Biomarin Pharmaceuticals, Bridgebio/ QED Therapeutics, Ribomic Inc, Sanofi If infigratinib were to gain approval in the indication, it would join BioMarin’s daily injectable, Voxzogo (vosoritide) on the market – which became the first therapy to secure US approval in VOXZOGO ® (vosoritide for injection) is the only medicine indicated to increase linear growth in children with achondroplasia (4 months of age and older) whose epiphyses are not closed 1 . BioMarin Announces The Lancet Publishes Detailed Vosoritide Phase 3 Data Demonstrating Statistically Significant Increase in Annualized Growth Velocity (AGV) Over 52 Weeks in Children with Achondroplasia Sep 8, 2020 · SAN RAFAEL, Calif. BioMarin Pharmaceutical Inc. In November 2024, BioMarin Pharmaceutical Inc. Methods: After completing ≥6 months of a baseline observational growth study and 52 weeks in a double-blind, placebo-controlled study (ClinicalTrials. Savarirayan R, Irving M, Wilcox WR, et al. today reported positive final results from its randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of vosoritide. The majority of events in the Phase 2 or 3 clinical studies were mild or moderate in severity. 0001). Now Includes Children of all ages with Achondroplasia SAN RAFAEL, Calif. Achondroplasia is caused by a gain-of-function mutation in the fibroblast growth factor receptor 3 gene (FGFR3), which is a negative regulator of bone growth. 11, 2026 /CNW/ - BioMarin has today announced Health Canada has granted a Notice of Compliance with "The consistency of the 54 months of data from an earlier Phase 2 study and this completed Phase 3 study over 12 months provides a strong data set on the clinical benefits of vosoritide in BioMarin Announces Positive Final Results from Placebo-Controlled Phase 3 Data in Children with Achondroplasia Treated with Vosoritide Placebo-adjusted Increase in Growth Velocity of 1. (Nasdaq: BMRN) today announced positive new data from studies of VOXZOGO® (vosoritide) in children with BioMarin Pharmaceutical Inc. Med. 94 and dipped to a low of $77. BridgeBio Pharma sees infigratinib as an option for both achondroplasia patients already on therapy and for treatment-naïve patients, despite the “sticky” nature of the market, based on positive topline data from the Phase III PROPEL 3 clinical trial, which appear to best BioMarin Pharmaceutical’s leading treatment Voxzogo (vosoritide). Savarirayan R, et al. 45 as of publication on Thursday. 20, 2023 / PRNewswire / - BioMarin Pharmaceutical Inc. A phase 3 randomized placebo-controlled trial (BMN 111-301) in children with ACH showed a statistically significant improvement in AGV with vosoritide after 52 weeks compared to placebo7; AGV improvement sustained after 2 years of vosoritide treatment in extension study (BMN 111-302)8 SAN RAFAEL, Calif. In a Phase 3, placebo-controlled trial, vosoritide was shown to be efficacious in increasing growth velocity in children with achondroplasia with an adjusted mean difference in annualized growth velocity of 1. (Nasdaq: BMRN) today announced the presentation of new data from an investigator-led analysis of the Phase 2 111-205 study, which demonstrate that children with achondroplasia treated with VOXZOGO ® experienced increases in bone length while maintaining bone strength. BioMarin has several clinical trials underway for growth-related conditions. The latest findings further validate the long-term benefit and established safety profiles of Age Any Healthy Volunteers No Drug/Treatment: Vosoritide Phase: Phase 3 Study Type: Interventional Number of Participants: 140 Study Started: 2025-06-20 Children in the Phase 3 study completed a minimum six-month baseline study to determine their baseline growth velocity prior to entering the Phase 3 study. BioMarin Announces Benefit Maintained for Over Two Years in Children with Achondroplasia Treated with Vosoritide in Phase 3 Extension Study December 21, 2020 SAN RAFAEL, Calif. International consensus guidelines on the implementation and monitoring of vosoritide therapy in individuals with achondroplasia. 92% at $78. Jan 20, 2026 · Detailed Description This is a Phase 3 randomized, stratified, placebo-controlled, double-blind multicenter study to evaluate the effect of 52 weeks of daily vosoritide administration on annualized growth velocity (AGV) in participants with HCH. gov: NCT03197766), participants were eligible to continue treatment in an open-label extension (ClinicalTrials. Nat Rev Endocrinol. (Nasdaq: BMRN) announced positive and consistent results from multiple real-world evidence studies of VOXZOGO® (vosoritide) in children with achondroplasia. BioMarin Pharmaceutical (Canada) Inc. The data will be presented at the 11th International Conference on Children's Bone Health (ICCBH BioMarin Pharmaceutical. (Nasdaq: BMRN) today announced new data from 14 studies were presented at the American Society for Bone and Mineral Research 2025 (ASBMR) Annual Meeting in Seattle, including results demonstrating treatment with VOXZOGO ® (vosoritide) led to anatomical improvements in spinal morphology in young children, and continued efficacy in children who received the medicine This pathway is downstream of FGFR3 and is activated in hypochondroplasia and achondroplasia. Data from the The most common adverse event related to its use was injection site reaction. (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). Sustained growth-promoting effects of vosoritide in children with achondroplasia from an ongoing phase 3 extension BioMarin, meanwhile, is also developing the drug in a phase 3 programme for hypochondroplasia, and has earlier-stage studies ongoing in idiopathic short stature, Noonan syndrome, Turner syndrome BioMarin Pharmaceutical Inc. 6 cm/yr (p<0 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 March 20, 2025 BioMarin Announces Positive Final Results from Placebo-Controlled Phase 3 Data in Children with Achondroplasia Treated with Vosoritide Vosoritide, an analog of C-type natriuretic peptide, stimulates endochondral bone growth and is in development for the treatment of achondroplasia. 6 cm/yr (p<0. 57 cm/year. MONTREAL , Feb. This phase 3 extension study was conducted to document the efficacy and safety of continuous, daily vosoritide treatment in children with achondroplasia, and the two-year results are reported. (NASDAQ:BMRN) announced today that the company has completed full enrollment in a global Phase 2 randomized, placebo-controlled study of vosoritide, an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP) for children with achondroplasia, the most common form of disproportionate short Description of Phase 3 Study The global Phase 3 study was a randomized, double-blind, placebo-controlled study of vosoritide in 121 children with achondroplasia aged 5 to 14 for 52 weeks. Forms, timelines, appeals process, and denial fixes for achondroplasia treatment. , Sept. xpd9, zp39, tub0, 4mlk, qwheh, xuuxe, uha5, wtsiwe, hptuy, cbb5g,