FDA Asks For More Data To Resolve BioMarin's Gene Therapy Clinical Hold

BioMarin Pharmaceutical Inc (NASDAQ:BMRN) said that the FDA had requested additional information regarding the clinical hold on PHEARLESS Phase 1/2 study of BMN 307 issued in September 2021. 

• BMN 307 is an AAV5-human phenylalanine hydroxylase (hPAH) gene therapy studied in adults with phenylketonuria (PKU). 
• The FDA has requested data from additional non-clinical studies to assess the theoretical oncogenic risk to human study participants, expected to take several quarters. 
• Related: FDA Slaps Clinical Hold on BioMarin's Gene Therapy For Genetic Metabolism Disorder.
• The Company will communicate the next steps for the program when available.
• The Company has also completed enrollment in the ongoing Phase 3 Study 270-303 to evaluate the safety and effectiveness of valoctocogene roxaparvovec, an AAV5 gene therapy, in combination with prophylactic corticosteroids in severe hemophilia A. 
• Results from the 52-week analysis from this study are expected in 1H 2023. 
• Price Action: BMRN shares are down 1.44% at $89.00 during the premarket session on the last check Friday.