WebSep 8, 2024 · The Food and Drug Administration has placed a clinical hold on the phase 1/2 Phearless study evaluating BMN 307 for the treatment of phenylketonuria in adults. … WebSep 6, 2024 · U.S. FDA Placed a Clinical Hold on BMN 307 Phearless Phase 1/2 Gene Therapy Study in Adults with PKU Based on Interim Pre-clinical Study Findings PRESS …
FDA Asks For More Data To Resolve BioMarin
WebFeb 17, 2024 · In February 2024, the Company received additional requests from the Food and Drug Administration (FDA) for information needed to resolve the clinical hold of the PHEARLESS Phase 1/2 study of BMN 307 issued in September 2024. BMN 307 is an AAV5-human phenylalanine hydroxylase (hPAH) gene therapy being studied in adults … WebJul 21, 2024 · Primary Purpose: Treatment. Official Title: A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno … forumbyprometour
FDA Places Clinical Hold on Investigational Gene Therapy for
WebOct 27, 2024 · On September 5, 2024, BioMarin issued a press release announcing, "that the [FDA] placed a clinical hold on the BMN 307 Phearless Phase 1/2 study", which "is evaluating BMN 307, an investigational ... WebSep 7, 2024 · The Phase 1/2 Phearless study is evaluating BMN 307, an AAV5-phenylalanine hydroxylase (PAH) gene therapy for PKU. ... The clinical hold for the PKU program comes after BioMarin’s hemophilia A ... WebSep 28, 2024 · The clinical hold follows preclinical data which highlighted a potential issue in mice models of PKU. When treated with high BMN 307 doses, 85.7% of the mice … forumbyavanti.com