BioMarin Report Results of Vosoritide in P-III OLE Study for the Treatment of Children with Achondroplasia at ENDO 2021

Shots:

• The ongoing P-III OLE study involves assessing Vosoritide (15 mcg/kg) vs PBO in 119 children with achondroplasia. The CHMP opinion is expected in Europe in June 2021
• The results demonstrated increased AGV, baseline mean AGV of 4.28 cm/year, sustained restoration of a major portion of the growing deficit and improved height z-score through the second year of treatment, Similar efficacy after one year of treatment, crossed over to treatment with baseline mean AGV of 3.99 cm/year, mean AGV was 5.65 cm/year
• Vosoritide has also received ODD from FDA and EMA for the treatment of children with achondroplasia. The NDA filing is under review from the FDA with a PDUFA date of August 20, 2021

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