BioMarin Files IND for PEG-PAL

BioMarin Pharmaceutical has filed an investigational new drug application (IND) for polyethylene glycol-phenylalanine ammonia lyase (PEG-PAL) for the treatment of phenylketonuria, a genetic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase.

The Phase I study will assess the safety and pharmacokinetics of single injections of PEG-PAL in approximately 35 patients in a series of escalating dose cohorts.

Participants will be allowed to continue in a Phase II study to evaluate the safety and efficacy of weekly injections for eight weeks followed by a dose titration period.

BioMarin said it expects to begin studies of PEG-PAL in the first quarter of 2008.