BioMarin’s PKU Gene Therapy Clinical Hold To Last ‘Several Quarters’

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• By Mandy Jackson
  
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BMN 307 for phenylketonuria was put on a clinical hold in September due to liver cancer observed in mice and was expected to lift in Q1 of 2022, but the US FDA has requested additional preclinical studies. 

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Homology also now has a gene therapy for PKU on a clinical hold • Source: Alamy

BioMarin Pharmaceutical Inc. expects it to take “several quarters” to complete new preclinical studies requested by the US Food and Drug Administration as the agency determines whether to lift a clinical hold on the Phase I/II PHEARLESS clinical trial of gene therapy BMN 307 in adults with phenylketonuria (PKU). The company rapidly responded to the hold last year, so analysts expected PHEARLESS to restart in the first quarter of 2022, but that was before BioMarin’s 17 February update on its gene therapy programs.

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