New Data to be Presented for BioMarin's VOXZOGO
BioMarin`s dwarfism therapy gets FDA nod for expanded use
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.
SAN RAFAEL, Calif., Sept. 15, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending marketing authorization to expand the indication for VOXZOGO® (vosoritide) for injection to treat children with achondroplasia aged 4 months and older whose epiphyses (growth plates) are not closed. The diagnosis of achondroplasia should be confirmed by appropriate genetic testing. A final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission in the fourth quarter of 2023. VOXZOGO is currently approved in Europe in children with achondroplasia who are 2 years of age and older with open growth plates.
BioMarin is touting “record revenues” from the first half of 2023, spearheaded by the jump in sales of its achondroplasia drug Voxzogo — so much so that it’s putting a strain on supply.
SAN RAFAEL, Calif., March 14, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, will present updated data demonstrating the long-term benefit of treatment with VOXZOGO® (vosoritide) and new observational data on disease burden in children with achondroplasia. These data will be shared this week in an oral presentation and five posters at the 2023 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting in Salt Lake City, Utah.
The Food and Drug Administration will take another three months to complete its second review of BioMarin’s gene therapy for hemophilia A, now promising an answer by June 30.
SAN RAFAEL, Calif., March 7, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, announced today that the U.S. Food and Drug Administration (FDA) has accepted the company's supplemental New Drug Application (sNDA) for VOXZOGO® (vosoritide) for injection to expand treatment in the United States to include children with achondroplasia under the age of 5. Achondroplasia is the most common form of disproportionate short stature.
EMA Validates Application for Extension of Indication for VOXZOGO
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the Ministry of Health, Labor and Welfare (MHLW) in Japan granted approval of the...