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  5. Drug Trials Snapshots: SYMDEKO
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Drug Trials Snapshots: SYMDEKO

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 SYMDEKO Package Insert for complete information.

SYMDEKO (tezacaftor/ivacaftor)
{sim-DECK-oh}
Vertex Pharmaceuticals
Approval date: February 12, 2018


DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

SYMDEKO is a drug for the treatment of cystic fibrosis (CF) in patients 12 years and older, who have specific gene mutations.

CF is a serious genetic disorder that results in the formation of thick mucus that builds up in the lungs and other parts of the body. This can lead to severe breathing problems.

How is this drug used?

SYMDEKO is packaged as two tablets. One tablet, a combination of two drugs (tezacaftor and ivacaftor) is taken by mouth in the morning. The second tablet (ivacaftor) is taken by mouth, in the evening.

What are the benefits of this drug?

SYMDEKO improved lung function by allowing air to move easier. The amount of air that can be forcibly blown out in one second [percent predicted forced expiratory volume (ppFEV1)] was increased.

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

  • Sex: SYMDEKO worked similarly in males and females.
  • Race: Most of the patients were White. Differences in how well the drug worked among races could not be determined because of the small number of patients in other races.
  • Age: SYMDEKO worked similarly in patients younger and older than 18 years of age.

What are the possible side effects?

SYMDEKO may cause serious side effects including increased liver enzymes and clouding of the lens in the eye (cataracts).

The most commonly reported side effects associated with SYMDEKO are headache, nasopharyngitis, nausea, sinus congestion, and dizziness.

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

  • Sex: The risk of side effects was similar in males and females.
  • Race: Most of the patients in the trials were White. Differences in the occurrence of side effects among races could not be determined, because of the small number of patients in other races.
  • Age: The risk of side effects was similar in patients less than and older than 18 years of age.

WHO WAS IN THE CLINICAL TRIALS?

Who participated in the clinical trials?

The FDA approved SYMDEKO based on evidence from 3 clinical trials of 916 patients with cystic fibrosis.

Trial 1 (NCT02347657) was conducted in CF patients, who have two copies of the F508del mutation. This trial was conducted at 91 sites in the following 12 countries: Canada, Denmark, France, Germany, Ireland, Italy, Netherlands, Spain, Sweden, Switzerland, and the United Kingdom.

Trial 2 (NCT02392234) was conducted in CF patients, who have the F508del mutation and a second mutation that is predicted to respond to tezacaftor and/or ivacaftor therapy. This trial was conducted at 81 sites in the following 10 countries: Australia, Belgium, Canada, Germany, France, Israel, Italy, Netherlands, and the United Kingdom.

Trial 3 (NCT02516410) was conducted in CF patients, who have the F508del mutation and a second mutation that is predicted to be unresponsive to tezacaftor and/or ivacaftor therapy. This trial was conducted 38 sites in the following 7 countries: Australia, Austria, Canada, France, Israel, and Spain.

Figures 1 – 3 below summarize by sex, race and age how many patients participated in the combined clinical trials.

Figure 1. Baseline Demographics by Sex

Pie chart summarizing how many males and females were in the clinical trials. In total, 455 males (50%) and 461 women (50%) participated in the clinical trials.

FDA Review

Figure 2. Baseline Demographics by Race

FDA Review

Table 1. Demographics of Efficacy Trials by Race

Race Number of Patients Percentage of Patients
White 891 97%
Black or African American 7 1%
Asian 2 less than 1%
Other 7 1%
Not reported* 9 1%

*Data for race was not collected due to local regulations

FDA Review

Figure 3. Baseline Demographics by Age

Pie chart summarizes how many individuals of certain age groups were in the clinical trials. In total, 187 patients were 12 to 17 years old (20%) and 729 patients were 18 years and older (80%).

FDA Review

How were the trials designed?

The benefit and side effects of SYMDEKO were evaluated in three trials. Each trial had a different design and treatment duration.

Trial 1: Patients were randomly assigned to receive SYMDEKO, or placebo twice daily. The SYMDEKO morning dose was one table containing tezacaftor and ivacaftor; and the evening dose was one tablet containing ivacaftor only. Neither the patients nor the health care providers knew which treatment was being given until after the trials were completed. ppFEV1, the amount of air that can be forcibly blown out in one second, was measured after 24 weeks of treatment. SYMDEKO was compared to placebo.

Trial 2: Patients were randomly assigned to one of three treatments. Treatment one was SYMDEKO, in the morning and evening. The SYMDEKO morning dose was one table containing tezacaftor and ivacaftor; and the evening dose was one tablet containing ivacaftor only. Treatment two was ivacaftor twice daily. Treatment three was placebo twice daily. Neither the patients nor the health care providers knew which treatment was being given until after the trials were completed. In this trial, the benefit of SYMDEKO was assessed by taking the average of week 4 and week 8 measurements of ppFEV1 and compared to placebo and ivacaftor.

Trial 3: Patients were randomly assigned to receive SYMDEKO or placebo twice daily. The SYMDEKO morning dose was one table containing tezacaftor and ivacaftor; and the evening dose was one tablet containing ivacaftor only. Neither the patients nor the health care providers knew which treatment was being given until after the trials were completed. ppFEV1 was measured after 12 weeks of treatment. SYMDEKO was compared to placebo. This trial also had pre-planned analysis of some data, before the trial was completed.

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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