MEPSEVII is used to treat children and adult patients with mucopolysaccharidosis VII (also known as MPS VII or Sly Syndrome). Mucopolysaccharidosis VII is a rare, inherited disease. It is caused by a low level of an enzyme (β-glucuronidase). The lack of an enzyme causes mucopolysaccharides (long chains of sugar molecules) to build up in the tissues and organs of the body, causing progressive damage.
MEPSEVII is an enzyme replacement therapy that works by replacing the missing enzyme. MEPSEVII is given by a healthcare provider directly, into the bloodstream, through a needle in the vein, intravenous (IV) infusion every two weeks. MEPSEVII is infused over 4 hours. The amount of drug used depends on patient’s weight.
Drug Trial Snapshot: MEPSEVII
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 MEPSEVII 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 MEPSEVII Package Insert for complete information.
MEPSEVII (vestronidase alfa-vjbk)
mep-SEV-ee
Ultragenyx Pharmaceuticals Inc.
Approval date: November 15, 2017
mep-SEV-ee
Ultragenyx Pharmaceuticals Inc.
Approval date: November 15, 2017
DRUG TRIALS SNAPSHOT SUMMARY:
What is the drug for?
MEPSEVII is used to treat children and adult patients with mucopolysaccharidosis VII (also known as MPS VII or Sly Syndrome).
Mucopolysaccharidosis VII is a rare, inherited disease. It is caused by a low level of an enzyme (β-glucuronidase). The lack of an enzyme causes mucopolysaccharides (long chains of sugar molecules) to build up in the tissues and organs of the body, causing progressive damage.
MEPSEVII is an enzyme replacement therapy that works by replacing the missing enzyme.
How is this drug used?
MEPSEVII is given by a healthcare provider directly, into the bloodstream, through a needle in the vein, intravenous (IV) infusion every two weeks. MEPSEVII is infused over 4 hours.
The amount of drug used depends on patient’s weight.
What are the benefits of this drug?
After 24 weeks of treatment, patients who received MEPSEVII walked a longer distance (about 18 meters) in comparison to placebo treatment during the 6-minute walk test (6MWT).
Two additional patients in the MEPSEVII development program experienced marked improvement in lung function.
Were there any differences in how well the drug worked in clinical trials among sex, race and age?
The number of patients in the trial was too small (12 patients) to determine differences in sex, race, and age subgroups.
What are the possible side effects?
MEPSEVII may cause a serious allergic reaction (anaphylaxis). Anaphylaxis may include difficulty breathing, low blood pressure, and slow heartbeat.
The most common side effects of MEPSEVII are leakage of drug from the vein into surrounding tissue, diarrhea, rash, swelling in arms and legs, itching, severe allergic reaction(anaphylaxis), infusion site swelling, and headache.
Were there any differences in side effects among sex, race and age?
The number of patients in the trial was too small (12 patients) to determine differences in sex, race, and age subgroups.
WHO WAS IN THE CLINICAL TRIALS?
Who participated in the clinical trials?
The FDA approved MEPSEVII based primarily on evidence from one clinical trial (NCT02230566) of 12 patients with mucopolysaccharidosis VII. The trial was conducted at 4 sites in the United States.
Figure 1 summarizes how many patients were enrolled in the clinical trial.
Figure 1. Baseline Demographics by Sex
FDA Review
Figure 2 and Table 1 summarize the percentage of patients by race enrolled in the clinical trial.
Figure 2. Baseline Demographics by Race
FDA Review
Table 1. Baseline Demographics by Race
| Race | Number of Patients | Percentage |
|---|---|---|
| White | 9 | 75% |
| Other/Unknown | 3 | 25% |
Figure 3 summarizes the percentage of patients by age group in the trial.
Figure 3. Baseline Demographics by Age
FDA Review
How were the trials designed?
The benefit and side effects of MEPSEVII were based primarily on one trial. Patients were randomly assigned to four groups. Three groups of patients received placebo treatment before starting MEPSEVII treatment and one group received MEPSEVII only. MEPSEVII or placebo were given once every two weeks as intravenous (IV) infusions. Neither patients nor healthcare providers knew which treatment was given until after the trial was competed.
The benefit of 24 weeks of MEPSEVII treatment was primarily evaluated by the 6-minute walking test (6MWT) and compared to placebo treatment in ten patients who could perform the test. The 6MWT measured the distance a patient could walk on a flat surface in 6 minutes. An additional follow-up using 6MWT was done for up to 120 weeks.
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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