NOVATO, Calif., Aug. 17 /PRNewswire-FirstCall/ — BioMarin
Pharmaceutical Inc. (Nasdaq:
BMRN) announced today that the first subject has initiated
treatment in a Phase 3b study (PKU-016) to evaluate the effects of
Kuvan (sapropterin dihydrochloride) on neuropsychiatric symptoms in
subjects with phenylketonuria (PKU). BioMarin expects to
report results in the first half of 2012.
“We hope that this trial will provide quantitative data
regarding the neuropsychiatric effects of Kuvan for PKU patients,
and if successful, will continue to build its value proposition and
to drive the continued expansion of the market,” said Jean-Jacques
Bienaime, Chief Executive Officer of BioMarin. “Although we have
heard anecdotal accounts from patients and physicians regarding
Kuvan’s impact beyond blood Phe level reduction, BioMarin has never
formally evaluated these endpoints in a company trial due to the
extremely short development time for Kuvan. We are excited to
have this important study underway, and if the results are
positive, we plan to file for a label amendment. We also look
forward to upcoming data from several investigator-sponsored
studies on the neurocognitive effects of Kuvan at the SSIEM meeting
in Istanbul in late August.”
The Phase 3b study is a double-blind, placebo-controlled,
randomized 28-week study (2-week screening period, 13-week
randomized treatment period, 13-week open-label treatment period)
to evaluate the safety and therapeutic effects of sapropterin
dihydrochloride on neuropsychiatric symptoms in subjects with PKU.
The study will enroll approximately 200 subjects ages twelve
and above at approximately 40 sites in the U.S. and Canada.
The primary efficacy variable for symptoms of attention deficit
hyperactivity disorder (ADHD) will be evaluated using the
Attention-Deficit Hyperactivity Disorder Rating Scale (ASRS) in
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