Potential Chorea Treatment Valbenazine Under Review in US
Sold under brand name Ingrezza, therapy is approved in the US for tardive dyskinesia
The U.S. Food and Drug Administration (FDA) has agreed to review Neurocrine Biosciences’ supplemental new drug application (sNDA) for valbenazine to treat chorea caused by Huntington’s disease.
“This sNDA filing advances our effort to bring a potential new treatment option to the many thousands of people experiencing chorea associated with Huntington disease in the U.S.,” Eiry W. Roberts, MD, Neurocrine’s chief medical officer, said in a company press release.
The FDA set a prescription drug user fee act target action date for Aug. 20, meaning a decision is expected by then.
“We look forward to working with the FDA as it reviews our filing,” Roberts said.
Valbenazine, sold under the brand name Ingrezza, is approved in the U.S. for treating tardive dyskinesia, a movement disorder marked by facial tics. An sNDA lets a company request changes in the packaging, labelling, dosage, ingredients, or indications of an already approved therapy.
About 90% of adults with Huntington’s have chorea — uncontrolled, jerky movements that can affect several body parts and negatively impact motor coordination, walking ability, and posture, as well as swallowing and speech.
Valbenazine is an oral therapy that works by blocking the activity of vesicular monoamine transporter-2, a protein that controls the activity of dopamine in the brain. Dopamine is a major signaling molecule and is important for coordinating movement. Lowering its activity is believed to reduce involuntary movements.
Valbenazine was granted orphan drug designation by the FDA last year to control chorea in people with Huntington’s. Such a status is meant to accelerate the therapy’s clinical development and regulatory approval.
Neurocrine’s sNDA is supported by data from the completed Phase 3 KINECT-HD clinical trial (NCT04102579) and an ongoing open-label extension trial called KINECT-HD2 (NCT04400331). The latter is still enrolling at sites in the U.S. and Canada.
Results from a pharmacokinetics study — an evaluation of a therapy’s movement into, through, and out of the body — were also included.
KINECT-HD investigated the safety, tolerability, and effectiveness of valbenazine in 128 adults, aged 18–75, who had chorea symptoms due to Huntington’s. Participants were randomly assigned either valbenazine or a placebo capsule every day for 12 weeks (about three months).
Previously presented results reported that valbenazine significantly eased chorea symptoms relative to a placebo. Also, a greater proportion of valbenazine-treated patients showed an improvement in their overall health status relative to those given a placebo. The superiority of the therapy over a placebo in these outcomes was observed as early as two weeks after starting treatment.
KINECT-HD2 is assessing valbenazine’s long-term safety and effectiveness in patients who completed the KINECT-HD study as well as other adult Huntington’s patients with chorea symptoms. The trial is expected to enroll 150 patients who will receive the therapy for up to 156 weeks (nearly three years).
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