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BioMarin Receives Positive CHMP Opinion in Europe for Vosoritide for the Treatment of Children with Achondroplasia from Age 2 Until Growth Plates Close
European Commission Decision expected Q3 2021 Temporary Authorization for Use (ATU) Granted in France to Allow Access and Reimbursement of Vosoritide to Begin
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"European Commission Decision expected Q3 2021\n Temporary Authorization for Use (ATU) Granted in France to Allow Access and Reimbursement of Vosoritide to Begin Immediately\n Vosoritide is Potentially the First Medicine to be Approved to Treat Children with Achondroplasia in Europe\n Estimated Over 11,000 Children Across Europe, Middle East, and Africa Affected by Achondroplasia and Eligible for Treatment if Approved\n FDA Review of Vosoritide NDA Ongoing, PDUFA Target Action Date Nov. 20, 2021\n\n\nSAN RAFAEL, Calif., June 25, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending marketing authorization for vosoritide, a once daily injection analog of C-type Natriuretic Peptide (CNP) to treat achondroplasia in children from the age of 2 until growth plates are closed, which occurs after puberty when children reach final adult height. Achondroplasia is the most common form of disproportionate short stature in humans. A final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission in Q3 2021. Vosoritide is potentially the first medicine to be approved to treat children with achondroplasia in Europe and would be marketed under the brand name VOXZOGO™ (vosoritide). \n\n \n \n \n \n \n \n\n \nIt is estimated that over 11,000 children across Europe, Middle East, and Africa are affected by achondroplasia and eligible for treatment with vosoritide, if approved by a health authority. Approximately a third of this population are in countries authorized to sell under the EMA license. BioMarin anticipates additional patient access through named patient sales based on an EMA approval in countries in the Middle East and Africa and expects additional market registrations to be facilitated by an anticipated EMA license. \nAlso, the French National Agency for Medicines and Health Products Safety (ANSM) granted an Autorisation Temporaire d'Utilisation de cohorte (ATU cohort), or Temporary Authorization for Use to allow access and reimbursement of vosoritide to begin immediately under an authorized process. An ATU allows access to drugs not yet approved in France, when provided for rare diseases, with no alternative options, and when the benefit/risk is presume...