Vosoritide

BioMarin to Present Data Showing Long-Term Benefit of VOXZOGO® (vosoritide) on Growth in Children with Achondroplasia at 2023 European Society for Paediatric Endocrinology (ESPE) Meeting

Retrieved on: 
Thursday, September 21, 2023
BioMarin Pharmaceutical, Safety, European Directive on Traditional Herbal Medicinal Products, ESPE, European Medicines Agency, Ageing, Endocrine Society, Objective, Committee, EMA, Synthetic control method, Shanda, Acorn, Epiphysis, SAN, Committee for Medicinal Products for Human Use, PDUFA, Effectiveness, Growth, Patient, European Society for Paediatric Oncology, FDA, Community Access Program, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, CHMP, Pharmaceutical industry, Toy, Achondroplasia, The Hague, Vosoritide

SAN RAFAEL, Calif., Sept. 21, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced that new data on VOXZOGO® (vosoritide) in children with achondroplasia will be presented at the 61st Annual European Society for Paediatric Endocrinology (ESPE) Meeting in The Hague, Netherlands.

Key Points: 
  • In an open-label, long-term Phase 2 extension study, VOXZOGO demonstrated consistent and durable growth benefits in young children with achondroplasia when initiated before the age of 5.
  • Both groups of treated children demonstrated substantial restoration of height when compared to untreated children with achondroplasia.
  • VOXZOGO is currently approved in Europe in children with achondroplasia who are 2 years of age and older with open growth plates.
  • VOXZOGO is also currently approved in the United States in children with achondroplasia who are 5 years of age and older with open growth plates.

BioMarin Announces Oral Presentation at ENDO2021, the Endocrine Society's Annual Meeting, with Data Demonstrating 2 Years of Treatment Benefit in Children with Achondroplasia Treated with Vosoritide

Retrieved on: 
Saturday, March 20, 2021
Health, Orphan drugs, Life sciences, Rare diseases, Drug discovery, Achondroplasia, Vosoritide

The data from the open-label extension presented at ENDO21 showed that children maintainedan increase in Annual Growth Velocity (AGV) through the second year of continuous treatment with vosoritide.

Key Points: 
  • The data from the open-label extension presented at ENDO21 showed that children maintainedan increase in Annual Growth Velocity (AGV) through the second year of continuous treatment with vosoritide.
  • Retention of subjects on treatment was high with 93% of patients originally randomized to receive vosoritide remaining on treatment two years later.
  • Overall, the data are encouraging, and allows us to imagine the potential for this first and only targeted precision treatment for achondroplasia."
  • Vosoritide has also received orphan drug designation from the FDA and EMA for the treatment of children with achondroplasia.

Achondroplasia Pipeline Review, H2 2020: 7 Companies & 7 Drug Profiles - Therapeutics Assessment, Competitive Landscape, Drug Profiles, Dormant Projects, Product Milestones - ResearchAndMarkets.com

Retrieved on: 
Friday, October 30, 2020
General Health, Pharmaceutical, Health, Health, Orphan drugs, BioMarin Pharmaceutical, Virotherapy, Achondroplasia, Vosoritide, Food and Drug Administration

Aug 20, 2020: BioMarin submits new drug application to U.S. Food and Drug Administration for Vosoritide to treat children with achondroplasia

Key Points: 

Aug 20, 2020: BioMarin submits new drug application to U.S. Food and Drug Administration for Vosoritide to treat children with achondroplasia
View source version on businesswire.com: https://www.businesswire.com/news/home/20201030005425/en/

BioMarin Announces Presentation of Vosoritide Phase 3 Data in Children with Achondroplasia at the American Society for Bone and Mineral Research 2020 Annual Meeting

Retrieved on: 
Friday, September 11, 2020
Health, Articles, Medicine, Clinical research, Orphan drugs, Design of experiments, Drug discovery, Achondroplasia, RTT, Vosoritide, Placebo-controlled study, Placebo

The data will be presented during a virtual oral presentation on Saturday, September 12 at 11:50am ET.

Key Points: 
  • The data will be presented during a virtual oral presentation on Saturday, September 12 at 11:50am ET.
  • "This study is part of a robust clinical program, and we are grateful to the participating children, families and physicians."
  • Vosoritide has also received orphan drug designation from the FDA and EMA for the treatment of children with achondroplasia.
  • 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.

BioMarin Submits New Drug Application to U.S. Food and Drug Administration for Vosoritide to Treat Children with Achondroplasia

Retrieved on: 
Thursday, August 20, 2020
Health, Medicine, Growth disorders, Rare diseases, Orphan drugs, Achondroplasia, RTT, BioMarin Pharmaceutical, Vosoritide, Epiphyseal plate, European Medicines Agency, Medication

BioMarin recently announced that the European Medicines Agency (EMA) validated the Company's Marketing Authorization Application (MAA) for vosoritide on Aug. 13, 2020.

Key Points: 
  • BioMarin recently announced that the European Medicines Agency (EMA) validated the Company's Marketing Authorization Application (MAA) for vosoritide on Aug. 13, 2020.
  • "We are grateful to the children and families who have participated in the clinical studies to evaluate the potentially first pharmacological treatment option for children with achondroplasia.
  • We also recognize a broad range of views about treatment options, and we look forward to providing children with achondroplasia and their families a treatment choice with this potential new therapy."
  • Vosoritide is being tested in children whose growth plates are still "open", typically those under 18 years of age.This is approximately 25% of people with achondroplasia.

BridgeBio Pharma’s QED Therapeutics Announces Preclinical Data Demonstrating Potential of Low-Dose Infigratinib in Achondroplasia

Retrieved on: 
Monday, May 11, 2020
Growth disorders, Rare diseases, Achondroplasia, RTT, Health, Vosoritide, Clinical medicine, Medicine

Also presented in this study was in vitro data demonstrating that, at similar concentrations, infigratinib had greater activity over a CNP analog (vosoritide) in an achondroplasia cell line.

Key Points: 
  • Also presented in this study was in vitro data demonstrating that, at similar concentrations, infigratinib had greater activity over a CNP analog (vosoritide) in an achondroplasia cell line.
  • Fully enrolling the study will help us to advance infigratinib as a potential treatment for children with achondroplasia.
  • QED Therapeutics is currently looking to continue enrolling children in PROPEL, a natural history study in children with achondroplasia.
  • Moreover, QED Therapeutics operates in a very competitive and rapidly changing environment in which new risks emerge from time to time.

BioMarin Plans Regulatory Submissions for Marketing Authorization of Vosoritide to Treat Children with Achondroplasia in 3Q 2020 in both US and Europe

Retrieved on: 
Monday, April 6, 2020
Health, Clinical research, Medicine, Articles, Growth disorders, RTT, Achondroplasia, Placebo-controlled study, Vosoritide, Placebo

If approved, vosoritide would be the first medicine for the treatment of Achondroplasia in the US and Europe.

Key Points: 
  • If approved, vosoritide would be the first medicine for the treatment of Achondroplasia in the US and Europe.
  • "Vosoritide is the first potential pharmacological treatment for the underlying cause of achondroplasia.
  • "To have such a possible treatment for achondroplasia on the horizon, where none existed before is significant progress."
  • 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.

Poised for Significant Growth and Profitability, BioMarin Shares Company Highlights During R&D Day on November 14th in New York

Retrieved on: 
Thursday, November 14, 2019
Life sciences, BioMarin Pharmaceutical, Novato, California, Virotherapy, Vosoritide, Industries, Biotechnology

2.2 cm of this additional increase occurred in the last 12 months further informing our understanding of vosoritide's ongoing treatment impact.

Key Points: 
  • 2.2 cm of this additional increase occurred in the last 12 months further informing our understanding of vosoritide's ongoing treatment impact.
  • In addition, BioMarin reaffirmed that it plans to provide top-line data on the randomized, placebo-controlled Phase 3 study by the end of the year.
  • BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases.
  • In DSS cases, it is thought that the CNP may be capable of signaling and promoting growth.

BioMarin Announces Cumulative Additional Height Gain of 9.0 cm over 54 months versus Natural History in Children with Achondroplasia Treated with Vosoritide in Phase 2 Study

Retrieved on: 
Thursday, November 14, 2019
Medicine, Health, Clinical medicine, Orphan drugs, Achondroplasia, RTT, Placebo, Vosoritide

BioMarin expects top line results from the fully enrolled Phase 3 study of vosoritide in children by year end 2019.

Key Points: 
  • BioMarin expects top line results from the fully enrolled Phase 3 study of vosoritide in children by year end 2019.
  • The global Phase 3 study is a randomized, placebo-controlled study of vosoritide in approximately 110 children with achondroplasia ages five to 14 for 52 weeks.
  • Children in this study will have completed a minimum six-month baseline study to determine their respective baseline growth velocity prior to entering the Phase 3 study.
  • The primary endpoint of the study is the change in growth velocity from baseline over one year in children treated compared to placebo.

BioMarin Announces First Quarter 2019 Financial Results

Retrieved on: 
Thursday, April 25, 2019
Health, Orphan drugs, Organic compounds, BioMarin Pharmaceutical, Achondroplasia, Vosoritide, Amifampridine, Tetrahydrobiopterin, Elosulfase alfa, Cerliponase alfa, Pharmaceutical industry, Medication

The complete Phase 3 GENEr8-1 study will include 130 participants, and is expected to be fully enrolled in the third quarter of 2019.

Key Points: 
  • The complete Phase 3 GENEr8-1 study will include 130 participants, and is expected to be fully enrolled in the third quarter of 2019.
  • Vosoritide for children with achondroplasia: The Company expects top line results from the ongoing global, Phase 3 study by year-end 2019.
  • BioMarin will host a conference call and webcast to discuss first quarter 2019 financial results today, Thursday, April 25, 2019 at 4:30 p.m.
  • BioMarin, Brineura, Firdapse, Kuvan,Naglazyme, Palynziq and Vimizim are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates.