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Drug Trials Snapshots: Kanuma

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 KANUMA Prescribing Information for complete information.

KANUMA (sebelipase alfa)
Kuh-new-muh
Alexion
Approval date: December 8, 2015


DRUG TRIALS SNAPSHOT SUMMARY: 

What is the drug for?

KANUMA is a treatment for patients with a rare disease known as Lysosomal Acid Lipase (LAL) deficiency. Patients with LAL deficiency have no or little LAL enzyme activity. This results in a build-up of fats within the cells of various tissues that can lead to liver, heart, and blood vessel disease and other complications.

LAL deficiency has two forms. The first type (referred to as Wolman disease) is seen in infants (around 2 to 4 months of age) and is often fatal in the first year of life. The second form (referred to as cholesteryl ester storage disease, or CESD) is milder with an onset in early childhood or later in life. Life expectancy of patients with CESD depends on the severity of the disease and complications from the disease.

How is this drug used?

KANUMA is given by a health care professional through a needle in the vein (intravenous). KANUMA is given once every week to patients who present with LAL deficiency in the first six months of life. It is given once every other week to other patients.

What are the benefits of this drug?

In patients with Wolman disease, six of nine (67%) treated with KANUMA were alive at 12 months of age. This was compared to a group from another study (called a historical control) of 21 infants who were not treated. In this untreated group, none were alive at 12 months of age.

In patients with CESD treated with KANUMA, there was an improvement in levels of low-density lipoprotein (LDL) cholesterol and other disease-related measures compared to placebo after 20 weeks of treatment.

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

Subgroup analyses were conducted for sex, race, and age for the CESD trial in children and adults.

  • Sex:  KANUMA worked similarly in males and females.
  • Race:  The majority of patients in the trials were white. Differences in response to KANUMA among races could not be determined.
  • Age:  KANUMA worked similarly in all age groups studied.

What are the possible side effects?

The most common side effects observed in patients treated with KANUMA are diarrhea, vomiting, fever, rhinitis, anemia, cough, headache, constipation, and nausea.

KANUMA may cause severe allergic reactions including a life-threatening reaction called anaphylaxis.

Were there any differences in side effects among sex, race, and age? Subgroup analyses were conducted for sex, race, and age for the CESD trial in children and adults.

  • Sex:  The risk of side effects was similar in males and females.
  • Race:  The majority of patients in the trials were white. Differences in side effects among races could not be determined.
  • Age: The risk of side effects was similar in all age groups studied.

 

WHO WAS IN THE CLINICAL TRIALS?

Who participated in the clinical trials?

The FDA approved KANUMA based on evidence from 2 clinical trials of 75 patients with LAL deficiency. One trial enrolled 9 infants 1-6 months of age with Wolman disease, and the other trial enrolled 66 children and adults 4 years to 58 years of age with CESD. The trials were conducted in the United States and 16 other countries around the world.

Figures 1 and 2 summarize how many patients, by sex, were enrolled in the clinical trials. The two trials are shown separately.

Figure 1. Baseline Demographics by SexClinical Trial in Infants*
 

Pie chart summarizing how many male and female infants were in the clinical trial of the drug KANUMA. In total, 5 male (56%) and 4 female (44%) patients participated in the clinical trial used to evaluate the drug KANUMA.

*Based on clinical trial data

Figure 2. Baseline Demographics by SexClinical Trial in Children and Adults with CESD*

Pie chart summarizing how many male and female patients were in the clinical trial of the drug KANUMA. In total, 33 male (50%) and 33 female (50%) patients participated in the clinical trial in children and adults with CESD used to evaluate the drug KANUMA.*Based on clinical trial data

Figure 2 and Table 1 summarize the number and percentage of patients by race in the clinical trial in infants.

Figure 2. Baseline Demographics by Race—Clinical Trial in Infants*

Alt-Tag: Pie chart summarizing the percentage of infants by race enrolled in the KANUMA clinical trial. In total, 4 White (44%), 1 Black (11%), 1 Asian (11%) 3 Other (33%)  patients participated in the clinical trial used to evaluate the drug KANUMA.

*Based on clinical trial data
 

Table 1. Baseline Demographics by Race—Clinical Trial in Infants*

Race Number of Patients Percentage
White 4 44%
Black or African American 1 11%
Asian 1 11%
Other 3 33%

*Based on clinical trial data
 
Figure 3 and Table 2 summarize the number and percentage of patients by race in the clinical trial in children and adults.

Figure 3. Baseline Demographics by Race—Clinical Trial in Children and Adults with CESD*

Pie chart summarizing the percentage of children and adults by race who participated in the KANUMA clinical trial. In total, 55 White (83%), 1 Black (2%), 3 Asian (5%) and 7 Other (11%)  patients participated in the clinical trial in children and adults with CESD used to evaluate the drug KANUMA

*Based on clinical trial data

Table 2. Baseline Demographics by Race—Clinical Trial in Children and Adults with CESD*

Race Number of Patients Percentage
White 55 83%
Black or African American 1 2%
Asian 3 5%
Other 7 11%

*Based on clinical trial data
 

Figure 3 summarizes the number and percentage of patients by age group in the clinical trial in children and adults.

Figure 3. Baseline Demographics by Age—Clinical Trial in Children and Adults with CESD*

Pie chart summarizing how many children and adults of certain age groups were in the KANUMA clinical trial. In total, 24 patients (36%) were less than 12 years old, 23 patients (35%) were between 12 and 17 years old and 19 patients(29%) were 18 years and older. *Based on clinical trial data

How were the trials designed?

There were two trials that evaluated the benefits and side effects of KANUMA.

The first trial was done in 9 infants with Wolman disease. All babies were treated with intravenous KANUMA once every week. The benefit was evaluated by measuring how many infants were alive at the age of 12 months. This was compared to a historical cohort, which is a group of Wolman disease patients from another study with similar clinical characteristics who were not treated with KANUMA.

The second trial was done in 66 children and adults with LAL deficiency. All participants in the trial were treated with intravenous KANUMA or placebo once every other week. Neither the patients nor the health care providers knew which treatment was being given until after the trials were completed. The benefit of KANUMA was measured at 20 weeks although some patients continued to receive KANUMA for up to 36 weeks.

The trial measured improvement in LDL cholesterol levels and compared KANUMA to placebo at Week 20.

 

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.
 

DRUG PRESCRIBING INFORMATION

MEDICAL REVIEW

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