Austedo (Deutetrabenazine) Indications
Austedo (deutetrabenazine) is FDA-approved to treat two specific conditions: chorea associated with Huntington's disease and tardive dyskinesia in adults. 11
FDA-Approved Indications
1. Chorea Associated with Huntington's Disease
Deutetrabenazine demonstrated statistically significant efficacy in reducing involuntary choreiform movements in Huntington's disease patients. In the pivotal 12-week randomized controlled trial (Study 1), patients receiving Austedo showed a 4.4-unit improvement in Total Maximal Chorea Score compared to 1.9 units with placebo (treatment effect -2.5 units, p<0.0001). 1 Notably, 51% of patients rated their symptoms as "Much Improved" or "Very Much Improved" versus only 20% with placebo. The mean effective dose was 40 mg per day, with titration up to a maximum of 48 mg daily. 1
2. Tardive Dyskinesia
The medication is approved for treating tardive dyskinesia in adults, a condition caused by chronic use of dopamine receptor antagonists. This indication was established through two 12-week randomized, double-blind, placebo-controlled trials involving 335 adult patients. 11
Mechanism and Clinical Context
Deutetrabenazine functions as a vesicular monoamine transporter 2 (VMAT-2) inhibitor, representing one of the major pharmacological treatments for alleviating chorea alongside tetrabenazine and valbenazine. 2 The deuterated formulation offers pharmacokinetic advantages over the original tetrabenazine.
Important Clinical Considerations
The therapeutic effect is symptomatic only—deutetrabenazine does not modify the underlying disease progression of Huntington's disease. When discontinued, chorea scores return to baseline within one week, as demonstrated in the pivotal trial where Week 13 follow-up (after 1-week washout) showed return to baseline chorea levels. 1
Both formulations are available: the twice-daily tablet (Austedo) and once-daily extended-release formulation (Austedo XR), which have demonstrated bioequivalence at steady state. 3 The medication can be taken with or without food, as no clinically significant food effect was observed. 4
Dosing is individualized through careful titration starting at 6 mg per day, increasing weekly in 6 mg increments until optimal chorea control is achieved, side effects become intolerable, or the maximum dose of 48 mg daily (72 mg in some cases under close monitoring) is reached. 15