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news.BioMarin Pharmaceutical has announced that the first patient has initiated treatment in the Phase I clinical study of PEG-PAL for the treatment of phenylketonuria. The study is expected to conclude enrollment in the fourth quarter of 2008.
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The Phase I clinical trial is an open-label, multi-center study to be conducted in approximately 35 phenylketonuria (PKU) patients in a series of seven dose- escalating cohorts ranging from 0.001 to 1mg/kg, with each cohort receiving a single dose, and a six-week follow-up period.
The primary objective of the study is to assess the safety and tolerability of single, subcutaneous injections of PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase) in subjects with PKU. The secondary objectives of the study are to evaluate the pharmacokinetics of single, subcutaneous injections of PEG-PAL administered at escalating doses and to evaluate the effect of PEG-PAL on blood Phe concentrations in subjects with PKU.
Jean-Jacques Bienaime, CEO of BioMarin, said: “We believe PEG-PAL holds tremendous potential to bring blood Phe down to normal levels and may help patients who either do not respond to Kuvan or who wish to reduce blood Phe levels beyond what is possible with Kuvan.”