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  5. Drugs Trials Snapshot: Danyelza
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Drugs Trials Snapshot: Danyelza

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 DANYELZA Prescribing Information for complete information.

DANYELZA (naxitamab-gqgk)
(dan-YEL-zah)
Y-mAbs Therapeutics, Inc.
Approval date: November 25, 2020


DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

DANYELZA is a drug used in combination with a medicine called granulocyte-macrophage colony-stimulating factor (GM-CSF) to treat children 1 year of age and older and adults with high-risk neuroblastoma in bone or bone marrow whose tumor

  • did not respond to or has come back after previous treatments and
  • has shown a partial response, minor response, or stable disease to prior therapy.

Neuroblastoma is a rare cancer that develops from nerve cells anywhere in the body and most often occurs in young children.

How is this drug used?

DANYELZA is an injection given by a healthcare professional directly into a vein (intravenous infusion) on Days 1, 3 and 5 of each treatment cycle. GM-CSF is given under the skin (subcutaneously) daily starting 5 days prior to DANYELZA and continuing until the last day of DANYELZA administration of each cycle.

What are the benefits of this drug?

In one trial, approximately 45 percent of patients receiving DANYELZA with GM-CSF experienced complete or partial shrinkage of their cancer; for 30% of those patients, shrinkage lasted 6 months or longer.. In another trial, 34% patients receiving DANYELZA with GM-CSF experienced complete or partial shrinkage of their cancer; for 23% of those patients, shrinkage lasted 6 months or longer for.

DANYELZA was approved under FDA’s accelerated approval program, which provides earlier patient access to a promising new drug 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: DANYELZA was similarly effective in boys and girls.
  • Race: The majority of patients were White; therefore, differences in response to DANYELZA among different race groups could not be determined.
  • Age: The majority of patients were 12 years or younger, therefore, differences in response to DANYELZA among patients of different age groups could not be determined.

What are the possible side effects?

DANYELZA may cause serious side effects including

  • life-threatening reactions related to the infusion,
  • severe pain and nerve damage,
  • inflammation of the spinal cord,
  • brain damage called Reversible Posterior Leukoencephalopathy,
  • increased blood pressure,
  • harm to an unborn baby.

The common side effects that occurred in more than a half of patients who received DANYELZA were infusion-related reactions, pain, fast heart beats, vomiting, cough, nausea, decreased appetite, and diarrhea.

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

  • Sex: The occurence of side effects were similar in males and females.
  • Race: The majority of patients were White. Therefore, differences in side effects among races could not be determined.
  • Age: The majority of patients were 12 years or younger. Therefore, differences in side effects among patients of different age groups could not be determined.

WHO WAS IN THE CLINICAL TRIALS?

Who participated in the clinical trials?

The FDA approved DANYELZA based on two clinical trials (Trial 1/NCT03363373 and Trial 2/NCT01757626) 97 patients with high-risk neuroblastoma in bone or bone marrow. The trials were conducted at 4 centers in the United States and in Spain.

Figure 1 summarizes how many boys and girls were in the clinical trials.

Figure 1. Demographics by Sex

Pie chart summarizing how many men and women were in the clinical trials. In

Adapted from FDA Review

Figure 2 summarizes by race how many patients were in the clinical trials.

Figure 2. Demographics by Race

Pie chart summarizing how many patients of different races were in the clinical trial. In total, 67 patients were White (69%), 3 patients were Black or African American (3%), 17 patients were Asian (18%), 2 patients were American Indian or Alaska Native (2%), and 8 patients were Other/Not reported (8%).)

Adapted from FDA Review

The figure below summarizes the age groups of patients in the clinical trials.

Figure 3. Demographics by Age

Pie chart summarizing how many individuals of certain age groups were in the clinical trial.  In total, 90 patients were less than 12 years old (93%), 5 patients were between 12-17 years old (5%), and 2 patients were 18 years and older (2%).

Adapted from FDA Review

The figure below summarizes the patients by ethnicity in the clinical trials.

Figure 4. Demographics by Ethnicity

Pie chart summarizing how many individuals of certain ethnicity groups were in the clinical trial.  In total, 6 patients were Hispanic or Latino (6%), 84 patients were not Hispanic or Latino (87%), and for 7 patients (7%) ethnicity has not been reported).

Adapted from FDA Review

How were the trials designed?

Both trials enrolled patients who were previously treated for high-risk neuroblastoma in the bone or bone marrow. Some patients were not responding to the previous therapies anymore and some patients experienced the return of the cancer. Patients with cancer that was actively growing after their last therapy were not included in the trial. All patients received DANYELZA in combination with GM-CSF according to the trial schedule.

The trials measured the percent of patients whose tumors completely or partially shrank, and whose bone marrow disease partially or completely responded, after treatment (overall response rate) and how long that shrinkage lasted.

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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