Business
BioMarin Expands Vosoritide Clinical Program
- Enrolls First Child in Phase 2 Study for Children with Achondroplasia at Risk of Life-Threatening Foramen Magnum Compression - Supports
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"- Enrolls First Child in Phase 2 Study for Children with Achondroplasia at Risk of Life-Threatening Foramen Magnum Compression\n - Supports Investigator-Initiated Phase 2 Study by Children's National Hospital to Explore Vosoritide in Selected Genetic Forms of Short Stature, Potential New Indications\n\n\nSAN RAFAEL, Calif., Nov. 9, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that the Company is expanding its clinical program for vosoritide, an investigational analog of C-type Natriuretic Peptide (CNP), with two new Phase 2 studies. The first study is sponsored by BioMarin to investigate the safety of vosoritide in infants with achondroplasia at risk of life-threatening foramen magnum compression. The second study is an investigator-initiated study sponsored by Children's National Hospital in Washington, D.C. to investigate vosoritide in children with selected genetic forms of short stature, which together represent addressable patient populations of approximately 275,000.\nPhase 2 Study for Children at Risk of Life-Threatening Foramen Magnum Compression\nBioMarin announced today that the Company has enrolled the first child in a Phase 2 randomized, controlled, open-label clinical trial with an open-label extension to investigate the safety of vosoritide in infants with achondroplasia at risk of requiring cervicomedullary decompression surgery to alleviate compression at the foramen magnum, the opening in the base of the skull through which the spinal cord passes. In addition, the study will also measure a secondary endpoint to evaluate the effect of vosoritide on growth of the foramen magnum volume through MRI scans. \nForamen magnum compression is the foremost life-threatening complications of achondroplasia in infants. This study investigates the safety of vosoritide in infants aged 0 -1 years of age with achondroplasia who have evidence of foramen magnum compression at-risk for requiring cervicomedullary decompression surgery. Those infants are under close observation for the appearance of neurological signs of progressive foramen magnum compression, and the current standard of care is palliative with many eventually requiring surgery. The study aims to enroll approximately 20 infants, who will be randomized to either current standard of care plus vosoritide treatment or current standard of...