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Drug Trials Snapshots: AUSTEDO

HOW TO USE THIS SNAPSHOT
The information provided in Snapshots highlights who participated in the clinical trials that supported the FDA approval of this drug, and whether there were differences among sex, race, and age groups. The “MORE INFO” bar shows more detailed, technical content for each section. The Snapshot is intended as one tool for consumers to use when discussing the risks and benefits of the drugs.

LIMITATIONS OF THIS SNAPSHOT:
Do not rely on Snapshots to make decisions regarding medical care. Always speak to your health provider about the risks and benefits of a drug. Refer to the AUSTEDO Prescribing Information for complete information.

AUSTEDO (deutetrabenazine)
(aw-STED-oh)
Teva Pharmaceuticals
Approval date: April 3, 2017


DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

AUSTEDO is a drug used for the treatment of chorea in patients with Huntington’s disease.

Chorea is a condition characterized by uncontrolled, involuntary movements and is one of many symptoms that occur in patients with Huntington’s disease.

How is this drug used?

AUSTEDO is a tablet that is taken with food once or twice a day depending on the daily dose.

What are the benefits of this drug?

AUSTEDO reduced chorea in Huntington’s disease patients.

What are the benefits of this drug?

The table below summarizes the data on the primary efficacy endpoint. This was based on the Total Maximal Chorea Score, an item of the Unified Huntington's Disease Rating Scale (UHDRS). On this scale, chorea is rated from 0 to 4 (with 0 representing no chorea) for 7 different parts of the body. The total score ranges from 0 to 28. The study compared the score at baseline to the average score at Weeks 9 and 12. The average score at Weeks 9 and 12 was called the “Maintenance Therapy” score or “Maintenance” score.

Table 2. Change from Baseline to Maintenance Therapy in Total Maximal Chorea (TMC)a Score in Patients Treated with AUSTEDO in Trial 1

Motor EndpointAUSTEDO
N = 45
Placebo
N = 45
p-value
Change in Total Chorea Scorea from Baseline to Maintenance Therapyb-4.4-1.9<>

aTMC is a subscale of the Unified Huntington's Disease Rating Scale (UHDRS)
bPrimary efficacy endpoint
AUSTEDO Prescribing Information

Were there any differences in how well the drug worked in clinical trials among sex, race and age?

Subgroup analyses were conducted for sex and age. Analysis for different racial groups was not done because majority of patients were White.

  • Sex: AUSTEDO worked similarly in men and women.
  • Age: AUSTEDO worked similarly in patients below and above 65 years of age.

Were there any differences in how well the drug worked in clinical trials among sex, and race groups?

The tables below summarize the responses to AUSTEDO by sex and age subgroups for the mITT population which includes all randomized patients who received at least one dose of study drug and had at least one post-baseline assessment of the TMC score.

Table 3. Subgroup Analysis of Primary Endpoint by Sex (mITT population)

SexChange from Baseline to maintenance in TMC scoreAUSTEDOPlacebo
WomenN2317
Means (SD)a-4.91 (2.45)-2.65 (2.69)
MenN2228
Means (SD)a-3.80 (3.36)-1.59 (2.62)
mITT: modified intent-to-treat; N: number of mITT patients; SD: standard deviation; TMC: total maximal chorea.
a Obtained from all observations in the gender specific mITT population at maintenance, with the last available value method for missing data.

Adapted from FDA Statistical review

Table 4. Subgroup Analysis of Primary Endpoint by Age Group (mITT population)

AgeChange from Baseline to maintenance in TMC scoreAUSTEDOPlacebo
< 65="">N3835
Means (SD)a-4.22 (2.84)-2.17 (2.81)
≥ 65 yearsN710
Means (SD)a-5.14 (3.64)-1.35 (2.08)
mITT: modified intent-to-treat; N: number of mITT patients; SD: standard deviation; TMC: total maximal chorea.
aObtained from all observations in the age group specific mITT population at maintenance, with the last available value method for missing data.

Adapted from FDA Statistical review

What are the possible side effects?

AUSTEDO may increase the risk of depression and suicide. Other serious side effects that AUSTEDO may cause include life threatening condition called neuroleptic malignant syndrome, restlessness (akathisia) and excessive sleepiness.

The most common side effects of AUSTEDO are sleepiness, diarrhea, dry mouth and tiredness.

What are the possible side effects?

The table below summarizes most common adverse reactions in the trial.

Table 5. Adverse Reactions in Trial 1 Experienced by at Least 4% of Patients on AUSTEDO and with a Greater Incidence than on Placebo

 

Adverse Reactions

AUSTEDO
(N = 45)
%

Placebo
(N = 45)
%

Somnolence

11

4

Diarrhea

9

0

Dry mouth

9

7

Fatigue

9

4

Urinary tract infection

7

2

Insomnia

7

4

Anxiety

4

2

Constipation

4

2

Contusion

4

2

AUSTEDO Prescribing Information

Were there any differences in side effects among sex, race and age?

Overall the number of patients in the trial was small, and the number of patients who experienced side effects in each subgroup was limited; therefore differences in side effect among sex, race and age subgroups could not be determined.

Were there any differences in side effects of the clinical trials among sex, and race groups?

The occurrence of side effects per demographic subgroup was low to allow for meaningful analysis.

WHO WAS IN THE CLINICAL TRIALS?

Who participated in the clinical trials?

The FDA approved AUSTEDO based primarily on evidence from one clinical trial that enrolled 90 patients with chorea associated with Huntington’s disease. The trial was conducted in 34 centers in the United States and Canada.

The figure below summarizes how many men and women were in the clinical trial.

Figure 1. Baseline Demographics by Sex

Pie chart summarizing how many men and women were in the clinical trial of the drug AUSTEDO . In total, 50 men (56%) and  40 women (44%) participated in the clinical trial.

Adapted from FDA Statistical review

The figure and table below summarize the percentage of patients by race in the clinical trial.

Figure 2. Baseline Demographics by Race

Pie chart summarizing the percentage of patients by race in clinical trial. In total, 83 Whites (92%), 5 Blacks or African Americans (6%), and  Multiple  (2%), participated in the clinical trial.

Adapted from FDA Statistical review

Table 1. Demographics by Race

Race

Number of patients

Percentage of patients

  White

83

92

  Black or African American

5

6

  Multiple

2

2

Adapted from FDA Statistical review

Figure 3. Baseline Demographics by Age

Pie charts summarizing how many individuals of certain age groups were in the clinical trial. In total, 73 patients  were younger than 65 years (81%), and  17 patients were  65 years and older (19 %)

Adapted from FDA Statistical review

Who participated in the trials?

The table below summarizes baseline demographics for the randomized population.

Table 6. Baseline Demographics for the Trial 1

Demographic ParameterAUSTEDO
(N=45)
Placebo
(N=45)
Total
(N=90)
Age (years)
   Mean (SD)55.4 (10.3)52.1 (13.4)53.7 (12.0)
   Min, Max23,7430,7323,74
Age Group (years), n (%)
   < 65="">38(84)35 (78)73 (81)
   ≥65 years7 (16)10 (22)17(19)
Sex, n (%)
   Men22 (49)28 (62)50 (56)
   Women23 (51)17 (38)40 (44)
Race, n ( %)
   White45 (100)38 (84)83 (92)
   Black or African American05 (11)5 (6)
   Multiple02 (4)2 (2)
Ethnicity, n (%)
   Non-Hispanic or Latino45 (100)45 (100)45 (100)

Adapted from FDA Statistical review

How were the trials designed?

There was one trial that evaluated the benefit and side effects of AUSTEDO. In the trial, patients with chorea associated with Huntington’s disease were randomly assigned to receive either AUSTEDO or placebo for 12 weeks. Neither the patients nor the health care providers knew which treatment was being given until after the trials were completed.

The trials measured overall change in chorea using Total Maximal Chorea Score. On this scale, chorea is rated from 0 to 4 (with 0 representing no chorea) for 7 different parts of the body. The total score ranges from 0 to 28.

How were the trials designed?

AUSTEDO was studied in a 12-week, randomized, double-blind, placebo controlled trial of ambulatory patients with manifest chorea associated with Huntington’s disease.

AUSTEDO was started at 6 mg per day and titrated upward, at weekly intervals, in 6 mg increments until satisfactory treatment of chorea was achieved, intolerable side effects occurred, or until a maximal dose of 48 mg per day was reached.

The primary endpoint was the change from baseline to maintenance therapy in the Total Maximal Chorea Score, an item of the Unified Huntington's Disease Rating Scale (UHDRS). On this scale, chorea is rated from 0 to 4 (with 0 representing no chorea) for 7 different parts of the body. The total score ranges from 0 to 28.

GLOSSARY

CLINICAL TRIAL: Voluntary research studies conducted in people and designed to answer specific questions about the safety or effectiveness of drugs, vaccines, other therapies, or new ways of using existing treatments.
COMPARATOR: A previously available treatment or placebo used in clinical trials that is compared to the actual drug being tested.
EFFICACY: How well the drug achieves the desired response when it is taken as described in a controlled clinical setting, such as during a clinical trial.
PLACEBO: An inactive substance or “sugar pill” that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo.
SUBGROUP: A subset of the population studied in a clinical trial. Demographic subsets include sex, race, and age groups.

PRESCRIBING INFORMATION

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