Based on interim safety findings from a pre-clinical, non-GLP pharmacology study, FDA has placed a clinical hold on the BMN 307 Phearless Phase 1/2 study, BioMarin Pharmaceutical Inc. (BMRN) said. The study is evaluating BMN 307, an investigational AAV5-phenylalanine hydroxylase gene therapy in adults with phenylketonuria.
The company carried out the pre-clinical study to understand the durability of BMN 307 activity in mice bearing two germline mutations. Of 63 animals treated, six of seven animals administered BMN 307 at the highest dose group had tumors on liver necropsy 52 weeks after dosing.
BioMarin said the clinical significance of these pre-clinical rodent findings has not been established. However, the company is pausing further enrollment into this global Phase 1/2 study until the investigation of these findings is completed.
For comments and feedback contact: editorial@rttnews.com
April 17, 2026 15:29 ET The ongoing conflict in the Middle East continues to raise concerns for policymakers who worry about the impact of the supply shock and high energy prices on the real economy. Producer price data and various survey results on the housing market were the main news from the U.S. this week. In Europe, industrial production data for the euro area gained attention. GDP figures out of China and the policy move by the Singapore central bank were in focus in Asia.