BioMarin Pharmaceutical
Inc. BMRN announced today results from the PKU-016 ASCEND study, the
largest randomized controlled trial evaluating neuropsychiatric outcomes in
phenylketonuria (PKU) patients treated with the approved drug Kuvan
(sapropterin dihydrochloride).
The study evaluated medically important symptoms similar to attention deficit
hyperactivity disorder (ADHD) in PKU patients whose blood levels of
phenylalanine (Phe) are reduced by Kuvan. The primary endpoint of the study
was evaluated using an attention deficit hyperactivity rating scale (ADHD-RS),
commonly used to evaluate symptoms of inattentiveness and hyperactivity. Kuvan
improved the ADHD-RS (p=0.085), driven by a statistically significant change
in the inattention component of the score (p=0.036). PKU patients have a lower
degree of hyperactivity as compared to ADHD patients, which likely resulted in
the less prominent benefits in this domain. The neurocognitive benefit of
Kuvan in patients who respond to Kuvan was corroborated by the Behavior Rating
Inventory of Executive Function, or BRIEF, in children who were rated by their
parents. Moderate to severe inattention deficit affected approximately
one-third of patients in the study. Preliminary analysis of safety indicates
that it is consistent with the general experience with Kuvan.
"We are encouraged to see the positive trend in the predetermined ADHD
primary endpoint, which was driven by a statistically significant change in
the sub-domain of inattentiveness," said Hank Fuchs, M.D., Chief Medical
Officer of BioMarin. "We believe these results will provide useful information
to the medical community on clinically relevant benefits of Kuvan and
facilitate adoption of the product. Further, the results will help inform the
design of the Phase 3 PEG-PAL program with key learnings about the measurement
tools. We are encouraged by the results and plan to discuss the submission of
this data with the FDA for possible inclusion in the Kuvan label."
In addition to the ADHD-RS and BRIEF instruments, other exploratory tests in
the PKU-016 study of neuropsychiatric deficit in PKU patients demonstrated a
higher level of impairment in PKU patients than the normal population. Further
analyses are underway to complete the evaluation of Kuvan on these outcome
measures.
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