In a recent announcement, the FDA approved an additional new drug from Neurocrine Biosciences, valbenazine (Ingrezza), a selective vesicular monoamine transporter 2 (VMAT2) inhibitor, for the treatment of chorea associated with Huntington’s disease (HD). Your application (sNDA) has been received. The agency plans to make a decision regarding agents by August 20, 2023.1
The sNDA is supported by results from the double-blind, placebo-controlled Phase 3 KINECT-HD trial (NCT04102579) and the ongoing open-label KINECT-HD2 trial (NCT04400331). Each study included adults aged 18 to her 75 who were diagnosed with Manifest HD or Motor Manifest HD with adequate chorea symptoms.
Eiry W. Roberts, MD, chief medical officer at Neurocrine Biosciences, said in a statement: We look forward to working with the FDA as it reviews our application. ”1
In KINECT-HD, a total of 128 HD patients were randomized 1:1 to either valbenazine or placebo for a 12-week treatment period, titrated for the first 8 weeks, followed by a 4-week maintenance period. continued. At the end of the study, agents achieved their primary endpoint. This is represented by a statistically significant placebo-adjusted reduction of her 3.2 units in the Unified Huntington’s Disease Rating Scale (UHDRS) total maximal chorea (TMC) score (P. <.00001) from baseline to weeks 10 and 12.2
For secondary endpoints, the investigators observed a 42.9% and 52.7% reduction in clinical global impression of change and patient global impression of change, respectively, in valbenazine-treated patients, compared with 13.2% decrease (P. <.001) and 26.4% (P. <.01) for placebo. Another secondary endpoint used to assess upper and lower extremity function, quality of life on the Neuropathy Scale, was included in the study but did not reach statistical significance. .
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Treatment-emergent adverse events (TEAEs) were found in 76.6% in the valbenazine group and 63.5% in the placebo group, with serious TEAEs reported in 1.6% and 3.2% of these groups, respectively. The most common TEAEs, including somnolence, fatigue, falls, and akathisia, were mild to moderate in nature, consistent with previous safety findings. Specifically, no patient exhibited suicidal behavior or thoughts while taking the study drug.
An open-label study, KINECT-HD2, will include approximately 150 adults with exercise manifest HD and will be evaluated for up to 156 weeks. Similar to KINECT-HD, in this study he will use change from baseline to maintenance in the UHDRS TMC score as the primary endpoint. Although still open for recruitment, the study spans a 104-week study period.
History was made in April 2017 when valbenazine became the first FDA-approved treatment for patients with TD. Later that year, a capsule-strength form of the drug was also approved. The drug’s approval is based on more than 20 clinical studies involving more than 1000 people. Data from these studies showed that valbenazine 80 mg significantly, rapidly and significantly improved her TD severity compared to placebo at 6 weeks (–3.2 vs –0.1; P. ≤.001) separation was seen as early as 2 weeks, and a continued decrease was documented through 48 weeks of treatment.3