FDA's extension of clinical hold for PKU drug hits BioMarin

18 February 2022

US biotech BioMarin Pharmaceutical (Nasdaq: BMRN) opened Friday’s trading 2% lower.

This followed news that the US Food and Drug Administration (FDA) needs more information to resolve the clinical hold of the PHEARLESS Phase I/II study of BMN 307 issued in September 2021.

BMN 307 is an AAV5-human phenylalanine hydroxylase gene therapy being 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, which is expected to take several quarters.

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