Liv­er tu­mors in an­i­mals trig­ger clin­i­cal hold for Bio­Marin gene ther­a­py — high­light­ing a re­cur­ring con­cern

Hours af­ter the FDA wrapped its marathon lis­ten­ing ses­sion on the safe­ty risks of gene ther­a­py, Bio­Marin of­fered a re­minder of why the ad­vi­so­ry com­mit­tee meet­ing was con­vened in the first place.

Bio­Marin is halt­ing world­wide en­roll­ment to a Phase I/II study of its ex­per­i­men­tal gene ther­a­py for phenylke­tonuria fol­low­ing a clin­i­cal hold based on in­ter­im safe­ty find­ings from a pre­clin­i­cal phar­ma­col­o­gy study — name­ly, tu­mors found in the liv­er of cer­tain an­i­mals giv­en the drug.

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