A new DRUG TRIALS SNAPSHOT is now available
Drug Trials Snapshots: GALAFOLD
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.
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 GALAFOLD Package Insert for complete information.
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 GALAFOLD Package Insert for complete information.
GALAFOLD (migalastat hydrochloride)
gal-a-fold
Amicus Therapeutics U.S., Inc.
Approval date: August 10, 2018
gal-a-fold
Amicus Therapeutics U.S., Inc.
Approval date: August 10, 2018
DRUG TRIALS SNAPSHOT SUMMARY:
What is the drug for?
GALAFOLD is a drug for treatment of Fabry disease in adults. It is used in patients who have a specific change in the gene that causes Fabry disease. Change in the gene causes the corresponding enzyme to be a good target for therapy with GALAFOLD based on laboratory data.
Fabry disease is a rare, inherited disease that results from buildup of fat deposits called globotriaosylceramide (GL-3) in the body’s cells (including blood vessels and kidney) because of decreased activity of an enzyme called alpha galactosidase A (GLA).
How is this drug used?
GALAFOLD is taken as a capsule by mouth once every other day.
What are the benefits of this drug?
After six months of treatment, patients treated with GALAFOLD had a greater decrease in fat deposits in blood vessels of the kidney when compared to patients on placebo.
GALAFOLD was approved under FDA’s accelerated approval program, which provides earlier patient access to a promising new drug for serious condition while the company continues to conduct clinical trials to confirm that the drug works well.
Were there any differences in how well the drug worked in clinical trials among sex, race and age?
- Sex: GALAFOLD appeared to work better in males than in females.
- Race: The majority of patients in the trial was White. Differences in how the drug works among races could not be determined.
- Age: The majority of patients in the trial was 18-65 years of age. Differences in how the drug works among different age groups could not be determined.
What are the possible side effects?
The most common side effects are headache, nasal and throat irritation (nasopharyngitis), urinary tract infection, nausea, and fever (pyrexia).
Were there any differences in side effects among sex, race and age?
- Sex: The occurrence of side effects was similar among males and females with the exception of urinary tract infection which was seen more frequently in females than in males.
- Race: The majority of patients in the trial was White. Differences in the occurrence of side effects among races could not be determined.
- Age: The majority of patients in the trial was 18-65 years of age. Differences in the occurrence of side effects among different age groups could not be determined.
WHO WAS IN THE CLINICAL TRIALS?
Who participated in the clinical trials?
The FDA approved GALAFOLD based primarily on evidence from one clinical trial (NCT00925301/Trial 1) of 67 adults with Fabry disease. The trial was conducted at 13 sites in Canada, Europe, Latin America, and the United States.
All 67 patients were evaluated for side effects (safety population) and are presented below. Out of these 67 patients, 45 patients were evaluated for benefit (efficacy population) and are presented in Table 8, under the MORE INFO section.
Figure 1 summarizes the percentage of patients by sex in the clinical trial.
Figure 1. Baseline Demographics by Sex (safety population)
FDA Review
Figure 2 summarizes the percentage of patients by race in the clinical trial.
Figure 2. Baseline Demographics by Race (safety population)
FDA Review
Table 1. Baseline Demographics by Race (safety population)
| Race | Number of Patients | Percentage of Patients |
|---|---|---|
| White | 65 | 97% |
| Other | 2 | 3% |
FDA Review
Figure 3 summarizes the percentage of patients by age in the clinical trial used to evaluate safety.
Figure 3. Baseline Demographics by Age (safety population)
FDA Review
How were the trials designed?
The benefit and side effects of GALAFOLD were evaluated primarily in one clinical trial. Trial 1 enrolled adults with Fabry disease who had a genetic change and corresponding enzyme change. The enzyme change was determined to be a good target for therapy with GALAFOLD based on laboratory data. The patients never received treatment to replace the missing enzyme or had not received treatment to replace the missing enzyme for at least 6 months. Patients were randomly assigned to receive GALAFOLD or placebo once every other day for 6 months. Neither the patients nor the health care providers knew which treatment was being given until after the trial was completed.
The benefit of GALAFOLD was assessed after 6 months of treatment, by determining the number of fat deposits (GL-3) in blood vessel cells of the kidneys (by kidney biopsy). The proportion of patients with a 50% or more reduction in the average number of fat deposits (GL 3) after receiving GALAFOLD was compared to the proportion of patients with a 50% or more reduction in the average number of fat deposits (GL 3) after receiving the placebo.
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.
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.
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