BioMarin Pharmaceutical announced 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.
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.
Foramen 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 care alone for a two-year period. After the two-year randomized period, children in the study would be eligible to receive vosoritide in an open-label, 3-year additional extension period. The study will examine the incidence of adverse events between the two groups, volume MRI measurements of the foramen magnum, skull and spine, and progression to requiring decompression surgery.
"We continue to expand our clinical program of vosoritide for the treatment of achondroplasia with the first study specifically looking at the safety of vosoritide administered to infants at risk of serious and potentially fatal medical complications resulting from achondroplasia," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. "We are committed to understanding whether vosoritide can safely reduce the number of adverse outcomes in this high-risk population and are pleased to support this study as part of our commitment to advancing the standard of care for children affected by achondroplasia."
This investigator-initiated study will enroll approximately 35 children with short stature in specific genetic categories and will follow them for a six-month observation period to obtain baseline growth velocity, safety profile and quality of life assessment. Study participants will then be treated with vosoritide for 12 months and will be assessed for safety and improvement in growth outcomes. The primary endpoints include incidence of treatment emergent adverse events, change from baseline in annualized growth velocity and change from baseline in height Z-score.
"Many patients who present with short stature likely have genetic mutations in genes involved in growth plate physiology. Those patients with selected causes of short stature that interact with the CNP pathway may benefit from treatment with vosoritide, which directly targets the growth plate," said Andrew Dauber, M.D., MMSc, Chief of Endocrinology at Children's National Hospital, Washington D.C. and lead investigator of this clinical study. "In this ...
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