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ARUP Labs Seek PMA for Hemophilia Gene Therapy CDx

NEW YORK – ARUP Laboratories said on Thursday that the US Food and Drug Administration has accepted its premarket approval submission for an AAV5 total antibody assay intended as a companion diagnostic for an investigational severe hemophilia gene therapy treatment being developed by BioMarin Pharmaceutical.

The assay is being developed for use with valoctocogene roxaparvovec, which was accepted for priority review by the FDA, BioMarin said separately on Thursday, noting that it is the first acceptance by the agency for marketing application for a gene therapy product for any type of hemophilia in the US. The pharma company partnered with Salt Lake City-based ARUP to develop the companion diagnostic test, which is blood-based and intended to identify those patients most likely to respond to AAV5-based gene therapy.

BioMarin noted that it implemented the test in multiple clinical studies which evaluated valoctocogene roxaparvovec as a treatment for hemophilia A patients without antibodies to AAV5.