Ingrezza now approved by FDA for chorea in Huntington’s disease
New treatment seen to improve life quality for patients in clinical trials
The once-daily oral therapy Ingrezza (valbenazine) — already marketed in the U.S. as a treatment for a movement disorder — has now been approved by the U.S. Food and Drug Administration (FDA) for chorea in adults with Huntington’s disease.
Chorea, characterized by uncontrolled, jerky movements that can affect any part of the body, affects as many as 90% of adults with Huntington’s.
“We are proud to bring Ingrezza to people living with HD [Huntington’s disease] and their caregivers who now have the option of a one-capsule, once-daily treatment that has demonstrated significant improvement in HD chorea in clinical studies,” Kevin C. Gorman, CEO of Neurocrine Biosciences, which markets Ingrezza, said in a company press release.
Louise Vetter, president and CEO of the Huntington’s Disease Society of America, hailed the approval, noting that “chorea associated with HD can significantly affect the quality of life of a person living with HD by impacting their daily activities, social life, independence and overall well-being.”
“The approval of Ingrezza for HD chorea means that people living with [the neurodegenerative disease] have a new treatment option to help manage their chorea symptoms, which is a welcomed milestone in efforts to improve care for families affected by HD,” Vetter said.
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Ingrezza is available in oral capsules at doses of 40, 60, and 80 mg. According to Neurocrine, healthcare providers can adjust the therapy’s starting dose for each patient, based on the individual’s response and tolerability.
Neurocrine already had offered a support program called INBRACE to help prescribed patients access its therapy. With Ingrezza now approved for Huntington’s, that program also will be available for these new patients. The program includes copay assistance for individuals with commercial insurance as well as assistance for uninsured patients.
The FDA’s decision comes about eight months after the agency agreed to review the company’s application asking to add Huntington’s-associated chorea to Ingrezza’s label, which already covered tardive dyskinesia, a movement disorder characterized by uncontrolled facial tics.
The therapy had been granted orphan drug status by the FDA for Huntington’s-associated chorea, which was meant to accelerate its clinical development and regulatory approval.
Ingrezza is thought to work by reducing the activity of dopamine in the brain. Dopamine is a neurotransmitter — a chemical that nerve cells use to communicate with each other — that plays important roles in regulating movement. By lowering dopamine signaling, Ingrezza is expected to ease uncontrolled motor movements.
Its approval for chorea was supported by data from two clinical trials: the completed Phase 3 KINECT-HD trial (NCT04102579) and the ongoing open-label extension KINECT-HD2 trial (NCT04400331). The Huntington Study Group, a clinical research network, is a collaborator in both of them. KINECT-HD2 is slated to wrap up in December.
KINECT-HD tested the therapy against a placebo in 128 adults with chorea associated with Hungtington’s. Participants were started on a daily dose of 40 mg, which was gradually increased up to 80 mg, based on patients’ tolerability.
The results showed that Ingrezza significantly outperformed the placebo, by about threefold, in easing chorea symptoms after about three months of treatment. Nearly half of patients given Ingrezza experienced a reduction of more than 40% in chorea severity.
Clinical results that led to this important approval showed reduction in the severity of chorea as early as two weeks after starting Ingrezza at an initial dose of 40 mg, with consistently greater improvements versus placebo seen at all subsequent visits.
Patient- and physician-reported chorea reductions were more frequent in the Ingrezza group than in the placebo group.
The most common side effects of treatment were sleepiness and fatigue.
“Clinical results that led to this important approval showed reduction in the severity of chorea as early as two weeks after starting Ingrezza at an initial dose of 40 mg, with consistently greater improvements versus placebo seen at all subsequent visits,” said Erin Furr Stimming, MD, a neurologist at UTHealth Houston and the trial’s principal investigator on behalf of the Huntington Study Group.
“Data also demonstrated Ingrezza was generally well tolerated and showed clinically meaningful improvement in adults with chorea associated with HD,” Stimming added.
KINECT-HD2 is evaluating the safety and efficacy of up to three years of treatment with Ingrezza in patients who completed the KINECT-HD study and other adults with Huntington’s-associated chorea.
“We are thankful for those in the HD community who helped contribute to this important milestone, and we remain committed to bringing medicines to patients with unmet medical needs for debilitating neurological disorders,” Gorman said.