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Biomarin Pharmaceutical Says U.S. FDA Placed Clinical Hold On BMN 307 Phearless Phase 1/2 Study

Published: 09/06/2021 06:15 GMT
Biomarin Pharmaceutical Inc. - Registered Shares (BMRN) - FDA Placed a Clinical Hold on Bmn 307 Phearless Phase 1/2 Study.
Biomarin Pharma- Phearless Study is Evaluating Bmn 307, Investigational Aav5-phenylalanine Hydroxylase Gene Therapy, in Adults With Phenylketonuria.
FDA's Clinical Hold Was Based on Interim Safety Findings From a Pre-clinical, Non-glp Pharmacology Study.
Biomarin Also Pausing Further Enrollment of Additional Participants Outside U.S. in Phearless Phase 1/2 Study.
Working With FDA and Other Health Authorities and Will Communicate Next Steps When Available.
Biomarin -of 63 Animals Treated, 6 of 7animals Administered Bmn 307 at Highest Dose Group Had Tumors on Liver Necropsy 52 Weeks After Dosing in Study.
Translatability of These Findings to Humans is Uncertain and Under Further Investigation.