FDA Names Valbenazine an Orphan Drug for Chorea With Huntington’s

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration (FDA) has designated valbenazine an orphan drug as a potential treatment to control the involuntary, jerky movements known as chorea in people with Huntington’s disease.

Orphan drug status is given to therapies with the potential to improve medical care for rare disease. It confers several incentives to the therapy’s developer, Neurocrine Biosciences, including exemption from FDA application fees and the potential for seven years of market exclusivity should the agency approve the therapy.

Neurocrine plans to apply for FDA approval of valbenazine for Huntington’s patients this year, with its request supported by data from the Phase 3 KINECT-HD clinical trial and an enrolling, open-label separate study underway in the U.S. and Canada.

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“Receiving an FDA Orphan Drug Designation validates our continued commitment to developing new treatment options that could benefit the lives of patients living with rare diseases, including those impacted by HD [Huntington’s disease],” Kevin Gorman, PhD, Neurocrine’s CEO, said in a press release.

Chorea is a common motor symptom of Huntington’s that is characterized by jerky, uncontrolled movements. Valbenazine works by blocking the activity of vesicular monoamine transporter-2 (VMAT-2), a protein that controls the activity of dopamine in the brain. Lowering dopamine activity — dopamine is a signaling molecule important for coordinating movement — is believed to lessen involuntary movements.

Valbenazine, an oral therapy, is FDA-approved and marketed under the brand name Ingrezza to treat tardive dyskinesia, a movement disorder characterized by facial tics.

Neurocrine sponsored KINECT-HD (NCT04102579) to test valbenazine in Huntington’s-associated chorea. The study enrolled 128 adults, who were randomly assigned to valbenazine or a placebo capsule, taken every day for 12 weeks (around three months).

Top-line results, announced last year, showed that treatment with valbenazine significantly eased chorea symptoms relative to placebo, with an effect seen after about two weeks of treatment.

An ongoing, open-label extension trial called KINECT-HD2 (NCT04400331) is further exploring the long-term safety and efficacy of valbenazine in adults, up to age 75, with Huntington’s-associated chorea. KINECT-HD2 is enrolling people who completed the original KINECT-HD study and is actively recruiting additional participants at sites in the U.S. and Canada.

“We are in the process of completing data analysis from the KINECT-HD and the ongoing KINECT-HD2 studies, which will form the basis of our supplemental new drug application (sNDA) for submission to the FDA later this year,” seeking valbenazine’s approval for Huntington’s patients, Gorman said.