Drug Trials Snapshot: NIKTIMVO
HOW TO USE THIS SNAPSHOT
The information provided in Snapshots highlights who participated in the key clinical trials that supported the original FDA approval of this drug, and whether there were differences among sex, race, age, and ethnic 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 healthcare provider about the benefits and risks of a drug.
Some of the information in this Snapshot is for presentation purposes and does not represent the approved conditions of use of this drug. Refer to the NIKTIMVO Prescribing Information for all of the approved conditions of use of this drug (e.g., indication(s), population(s), dosing regimen(s), safety information).
Snapshots are limited to the information available at the time of the original approval of the drug and do not provide information on who participated in clinical trials that supported later approvals for additional uses of the drug (if applicable).
NIKTIMVO (axatilimab-csfr)
nik tim' voe
Incyte Corporation
Original Approval date: August 14, 202
DRUG TRIALS SNAPSHOT SUMMARY:
What is the drug for?
NIKTIMVO is a drug used to treat adults and children who weigh at least 88.2 pounds (40 kg) with chronic graft-versus-host disease (GVHD) who have received at least two prior treatments (systemic therapy) and required additional treatment.
Chronic GVHD is a complication that can occur after someone has received a bone marrow or stem cell transplantation from another person.
How is this drug used?
NIKTIMVO is a solution for intravenous infusion that is taken at a dose of 0.3 mg/kg of body weight, every two weeks in patients weighing up to 40 kg.
Who participated in the clinical trials?
The FDA approved NIKTIMVO based on evidence of safety and efficacy from a clinical trial which included a total of 79 patients with chronic GVHD after failure of two prior lines of systemic therapy. The trial was conducted at 55 sites in 13 countries, including Australia, Belgium, Canada, Germany, Greece, France, Israel, Italy, Spain, South Korea, Taiwan, the United Kingdom, and the United States. There were 24 sites in the United States. Out of 79 patients, at the approved dose of 0.3 mg/kg every two weeks, 37 patients were enrolled in the United States, and 43 patients at the trial sites outside of the United States.
How were the trials designed?
NIKTIMVO was evaluated in an open-label single arm clinical trial of 79 patients with chronic GVHD who had received at least two prior systemic treatments and required additional treatment. All patients received NIKTIMVO 0.3 mg/kg every two weeks, until chronic GVHD progression or unacceptable toxicity.
The benefit of NIKTIMVO was evaluated by measuring how many patients had responded to the treatment.
How were the trials designed?
The primary endpoint of the trial was the overall chronic GVHD response rate in the first 6 cycles (by Cycle 7 Day 1), defined according to the 2014 National Institutes of Health Consensus Development Project on Criteria for Clinical trials in chronic GVHD.
Improvement in chronic GVHD symptom burden measured by the modified Lee Symptom Scale score (mLSS) was the key secondary endpoint.
DEMOGRAPHICS SNAPSHOT
Figure 1 summarizes how many male and female patients were enrolled in the clinical trial used to evaluate the efficacy of NIKTIMVO.
Figure 1. Baseline Demographics by Sex
Source: Adapted from FDA Review
Figure 2 summarizes how many patients by race were enrolled in the clinical trial used to evaluate the efficacy of NIKTIMVO.
Figure 2. Baseline Demographics by Race
Source: Adapted from FDA Review
Figure 3 summarizes how many patients by age were enrolled in the clinical trial used to evaluate the efficacy of NIKTIMVO.
Figure 3. Baseline Demographics by Age
Source: Adapted from FDA Review
Figure 4 summarizes how many patients by ethnicity were enrolled in the clinical trial used to evaluate the efficacy of NIKTIMVO.
Figure 4. how many patients by ethnicity were enrolled in the clinical trial used to evaluate the efficacy of NIKTIMVO.
Source: Adapted from FDA Review
Who participated in the trials?
Table 1. Baseline Demographics
| Demographic | NIKTIMVO N=79 |
| Sex, n (%) | |
| Male | 46 (58) |
| Female | 33 (42) |
| Age, years | |
| Mean (SD) | 49.4 (17.0) |
| Median | 50 |
| Range (min, max) | 7, 76 |
| Age group, years, n (%) | |
| <17 | 4 (5) |
| ≥17 to <65 | 54 (68) |
| ≥65 | 21 (27) |
| Race, n (%) | |
| White | 67 (85) |
| Asian or Pacific Islander | 4 (5) |
| Black or African American | 2 (3) |
| Other/Not reported | 6 (8) |
| Ethnicity, n (%) | |
| Hispanic or Latino | 5 (6) |
| Not Hispanic or Latino | 72 (91) |
| Other | 2 (3) |
Source: Adapted from FDA Review
Abbreviations: SD, standard deviation
What are the benefits of this drug?
Seventy-five percent (75%) of patients treated with NIKTIMVO had their chronic GVHD respond to treatment. In addition, an exploratory analysis showed that 56% patients reported a meaningful decrease in the chronic GVHD symptom bother.
What are the benefits of this drug (results of trials used to assess efficacy)?
Table 2. Efficacy Results, Efficacy Population
| Endpoint | NIKTIMVO N=79 |
| Overall response rate, n (%) | 59 (75) |
| 95% CI | 64, 84 |
| Complete response, n (%) | 0 (0) |
| Partial response, n (%) | 59 (75) |
Source: NIKTIMVO Prescribing Information
Abbreviations: CI, confidence interva
Were there any differences in how well the drug worked in clinical trials among sex, race, and age?
- Sex: NIKTIMVO worked similarly in males or females.
- Race: NIKTIMVO worked similarly in White and patients of other races. However, the sample size for races other than White was relatively small. Therefore, differences in how NIKTIMVO worked among races could not be determined.
- Age: NIKTIMVO worked similarly in patients of all ages. However, the sample size for patients younger than 18 years of age was relatively small. Therefore, differences in how NIKTIMVO worked among age groups could not be determined.
- Ethnicity: NIKTIMVO worked similarly in Hispanic and patients of other ethnicities. However, the sample size for ethnicity other than not Hispanic was relatively small. Therefore, differences in how NIKTIMVO worked among ethnicities could not be determined.
What are the possible side effects?
The most common side effects of NIKTIMVO are infections; increased blood level of liver enzymes; decreased blood level of phosphate; low red blood cell count (anemia); muscle, bone, or joint pain; increased blood level of pancreatic enzymes; low energy; increased blood level of calcium; increased blood level of a muscle enzyme; increased blood level of a bone enzyme; nausea; headache; diarrhea; cough; fever; shortness of breath; and infusion related reactions. Infusion-related reactions are common with NIKTIMVO and can be serious.
Based on its mechanism of action, NIKTIMVO can be harmful to an unborn baby when given to pregnant patients.
What are the possible side effects? (results of trials used to assess safety)?
Table 3. Adverse Reactions in ≥10% of Patients With Chronic GVHD Who Received NIKTIMVO
| Adverse Reaction | All Grades % |
Grades 3 or 4 % |
|---|---|---|
| Infections and infestations | ||
| Infection (pathogen unspecified) | 57 | 14 |
| Viral infection | 43 | 15 |
| Bacterial infection | 15 | 8 |
| Musculoskeletal and connective tissue disorders | ||
| Musculoskeletal pain | 35 | 3 |
| General disorders and administration site conditions | ||
| Fatigue | 32 | 4 |
| Pyrexia | 15 | 1 |
| Edema | 13 | 1 |
| Gastrointestinal disorders | ||
| Nausea | 23 | 3 |
| Diarrhea | 18 | 5 |
| Nervous system disorders | ||
| Headache | 20 | 1 |
| Dizziness | 11 | 0 |
| Respiratory, thoracic and mediastinal disorders | ||
| Cough | 18 | 0 |
| Dyspnea | 15 | 3 |
| Immune system disorders | ||
| Drug hypersensitivity | 13 | 3 |
| Metabolism and nutrition disorders | ||
| Decreased appetite | 11 | 4 |
| Vascular disorders | ||
| Hemorrhage | 11 | 1 |
| Skin and subcutaneous tissue disorders | ||
| Rash | 10 | 0 |
Source: NIKTIMVO Prescribing Information
Abbreviations: GVHD, graft-versus-host disease
Table 4. Selected Laboratory Abnormalities in Patients With Chronic GVHD Who Received NIKTIMVO
| NIKTIMVO, N=79 | ||
|---|---|---|
| Laboratory Abnormality | All Grades % |
Grade 3 or 4 % |
| Hematology | ||
| Decreased hemoglobin | 48 | 4 |
| Chemistry | ||
| Increased aspartate aminotransferase | 61 | 5 |
| Increased alanine aminotransferase | 51 | 3 |
| Decreased phosphate | 51 | NA |
| Increased gamma glutamyl transferase | 39 | 4 |
| Increased lipase | 34 | 3 |
| Increased amylase | 32 | 0 |
| Increased calcium | 31 | 1 |
| Increased alkaline phosphatase | 28 | 0 |
| Increased creatine phosphokinase | 25 | 0 |
Source: NIKTIMVO Prescribing Information
Abbreviations: GVHD, graft-versus-host disease
Were there any differences in side effects among sex, race, and age?
- Sex: The occurrence of side effects was similar in males and females.
- Race: The occurrence of side effects was similar in White and patients of other races, including Black or African American, Asian, other, and not reported.
- Age: The occurrence of side effects on blood, lungs, and liver was higher among patients older than 65 years of age and infection was higher among patients 65 years of age and younger.
- Ethnicity: Safety by ethnicity was not analyzed, because there were only five Hispanic or Latino patients.
Although certain adverse reactions were more frequent in some subgroups, because of the small subgroup sample sizes, it cannot be concluded that these differences are clinically meaningful.
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.