Orphan Drug Designation Granted to Valbenazine for Tourette Syndrome
The FDA has granted Orphan Drug Designation to Ingrezza for the treatment of Tourette syndrome in children.
Valbenazine (Ingrezza; Neurocrine Biosciences) has been granted Orphan Drug Designation by the Food and Drug Administration (FDA) for the treatment of Tourette syndrome in pediatric patients.
Neurocrine Biosciences also announced that it has initiated a Phase 2b clinical trial, T-Force GOLD, in children and adolescents with Tourette syndrome. The T-Force GOLD study is a multicenter, randomized, double-blind, placebo-controlled, parallel group, Phase 2b study evaluating the safety, tolerability, efficacy and optimal dose of valbenazine in up to 120 pediatric patients with moderate to severe Tourette syndrome. Patients will receive either valbenazine or placebo for 12 weeks followed by 2 weeks off-drug.
The primary endpoint of the study is the change from baseline of the Yale Global Tic Severity Scale between placebo and active treatment groups at the end of week 12. Tourette symptoms will also be evaluated using the Premonitory Urge for Tics Scale as well as Clinical Global Impression Scales. Top-line data from this study is expected in late 2018.
Valbenazine, a selective vesicular monoamine transporter 2 (VMAT2) inhibitor, is already approved in the US for the treatment of adults with tardive dyskinesia.
Ingrezza™ (Valbenazine) capsules: an investigational treatment of Tourette syndrome [news release]. San Diego, CA: Neurocrine Biosciences. Accessed November 8, 2017.