BioMarin Pharmaceutical To Present New Data From Studies Of VOXZOGO In Children With Achondroplasia And Hypochondroplasia And From Studies Of Investigational Long-Acting BMN 333 At American Society For Bone And Mineral Research Meeting; New Analysis Showcases Impact Of VOXZOGO In Foramen Magnum And Craniocervical Junction Growth

The data include encouraging new imaging results from a post-hoc analysis of a randomized, double-blind, placebo-controlled trial evaluating the effect of VOXZOGO on the growth of the foramen magnum and craniocervical junction in children with achondroplasia, including infants.BioMarin recently submitted a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for VOXZOGO for the treatment of hypochondroplasia. The company is also on track with its submissions to the European Medicines Agency (EMA) and other regional health authorities. If approved, VOXZOGO would be the first targeted therapy for the treatment of hypochondroplasia, with a potential 2027 launch.Additional data to be presented at the meeting include research in hypophosphatasia, as well as results from the Phase 3 ENERGY 3 study of BMN 401 in children ages 1 to 12 years with ENPP1 deficiency.Below are key presentations at ASBMR, with all times listed in Eastern Daylight Time:C-Type Natriuretic Peptide (CNP) Agonist CNP38 Promotes Longitudinal Bone Growth by Growth Plate Expansion in the Hyp Mouse Model of XLHOral Presentation #1138Monday, Oct. 12, 11:30 – 11:42 a.m.BMN 333, a Long-Acting Vosoritide Investigational Product, Promotes Endochondral Bone Growth and Skeletal Microarchitecture in Juvenile Mice and Nonhuman PrimatesPoster Presentation #092Friday, Oct. 9, 5:30 – 7 p.m.Sunday, Oct. 11, 2 – 3:30 p.m.Examining the Effect of Vosoritide Treatment on Bone Strength in Children With AchondroplasiaPoster Presentation #486Saturday, Oct. 10, 2 – 3:30 p.m.Efficacy and Safety of BMN 401 in Children With ENPP1 Deficiency: 52-Week Results From the Pivotal ENERGY 3 TrialPoster Presentation #554Saturday, Oct. 10, 2 – 3:30 p.m.Economic Burden of Hypophosphatasia: A Systematic Literature ReviewPoster Presentation #473Saturday, Oct. 10, 2 – 3:30 p.m.Development of BMN 333, a Long-Acting Version of Vosoritide for FGFR3-Related Dwarfism and Other Skeletal DysplasiasPoster Presentation #115Sunday, Oct. 11, 2 – 3:30 p.m.Comparison of Quantitative Computed Tomography Bone Mineral Density Measurements of Vosoritide-Treated Children With Achondroplasia to an Untreated Achondroplasia Reference PopulationPoster Presentation #474Sunday, Oct. 11, 2 – 3:30 p.m.Vosoritide Increases Growth Velocity in Children With Hypochondroplasia: Phase 3 Trial ResultsPoster Presentation #552Sunday, Oct. 11, 2 – 3:30 p.m.The Effect of Vosoritide on Growth of the Foramen Magnum and Craniocervical Junction in Children With AchondroplasiaPoster Presentation #483Sunday, Oct. 11, 2 – 3:30 p.m.Molecular Insights into Vosoritide's Role in Enhancing Bone Growth and MineralizationPoster Presentation #362Sunday, Oct. 11, 2 – 3:30 p.m.Understanding the Impact of Hypophosphatasia (HPP) on Health-Related Quality-of-Life and Activities of Daily Living: A Patient-Based Conceptual ModelPoster Presentation #011Sunday, Oct. 11, 2 – 3:30 p.m.Prospective, Observational Study of a Cohort of Hypophosphatasia Patients' Phenotypes Over Time and the Utility of Digital Outcomes Measures in This PopulationPoster Presentation #521Sunday, Oct. 11, 2 – 3:30 p.m.ENPP1 Inhibition Improves the Bone Phenotype, Mobility, Muscle Strength and Endurance in Later-Onset HPP MiceOral Presentation #1139Monday, Oct. 12, 11:42 – 11:54 a.m.