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

BioMarin Pharmaceutical Inc 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.