Drug Trials Snapshot: IMAAVY
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 IMAAVY Prescribing Information for all 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).
IMAAVY (nipocalimab-aahu)
im-AH-vee
Janssen Biotech, Inc.
Original Approval date: April 29, 2025
DRUG TRIALS SNAPSHOT SUMMARY:
What is the drug for?
IMAAVY is a drug for the treatment of generalized myasthenia gravis (gMG) in adult and pediatric patients 12 years of age and older. It is used by patients whose blood has antibodies against the acetylcholine receptor (AChR) or muscle-specific kinase (MuSK).
Myasthenia gravis is a rare disease that causes weakness in muscles, especially those muscles that control the eyes, face, neck, mouth, swallowing, breathing, and limbs.
How is this drug used?
A healthcare provider injects IMAAVY directly into the blood stream through a needle in the vein. This is known as an intravenous, or IV infusion.
It takes about 30 minutes to receive the first IMAAVY infusion and at least 15 minutes to receive a maintenance infusion administered 2 weeks after the first infusion. IMAAVY maintenance infusion is given every 2 weeks thereafter.
The amount of IMAAVY used depends on the patient’s weight.
Who participated in the clinical trials?
The FDA approved IMAAVY based on evidence from Study 1 in 196 adult patients with gMG. This study was conducted at 82 sites in 17 countries in North America, Europe, and Asia (Austrialia, Belgium, Canada, China, Czech Republic, Denmark, France, Germany, Italy, Mexico, Japan, Poland, South Korea, Spain, Sweden, Taiwan, and the United States).
An additional study evaluated the side effects of IMAAVY in 7 pediatric patients ages 12 to 16 years with gMG (Study 2). This study was conducted at 15 sites in 4 countries (Japan, Poland, the Netherlands, and the United States).
How were the trials designed?
The benefit and side-effects of IMAAVY were established in Study 1, a 24-week, multicenter, randomized, double-blind, placebo-controlled study. The study evaluated IMAAVY for the treatment of gMG in adult patients whose blood had antibodies against the AChR and MuSK.
Patients were randomized to receive IMAAVY 30 mg/kg loading dose followed by maintenance dose of 15mg/kg two weeks after the first dose, and then continued every 2 weeks thereafter for 24 weeks, or placebo.
The primary efficacy endpoint was the comparison of the average change from baseline at weeks 22, 23, and 24 of treatment on the Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score between patients treated with IMAAVY and patients who received placebo. The MG-ADL is a scale reported by patients that assesses the impact of gMG on daily function.
How were the trials designed?
The efficacy and safety of IMAAVY for the treatment of gMG in adult patients who are anti-AChR antibody positive or anti-MuSK antibody positive was established in a multicenter, randomized, double-blind, placebo-controlled study (Study 1, NCT04951622).
All patients had gMG defined as Myasthenia Gravis Foundation of America (MGFA) Clinical Classification Class II to IV and MG-ADL total score of at least 6 on stable dose of standard of care MG therapy including acetylcholinesterase inhibitors, steroids, or non-steroidal immunosuppressive therapies, either in combination or alone.
In Study 1, patients received IMAAVY equivalent to 30 mg/kg loading dose followed by maintenance dose of 15mg/kg two weeks after the first dose, and then continued every 2 weeks thereafter for 24 weeks, or placebo.
The primary efficacy outcome measure for Study 1 was the comparison of the average change from baseline at weeks 22, 23, and 24 in the MG-ADL total score between IMAAVY and placebo groups. The MG-ADL is a scale reported by patients that assesses the impact of gMG on daily function.
DEMOGRAPHICS SNAPSHOT
Figure 1 summarizes how many females and males were enrolled in Study 1 used to evaluate the efficacy and safety of IMAAVY.
Figure 1. Baseline Demographics by Sex (Efficacy population [n=153])
Source: Adapted from FDA Review
Figure 2 summarizes how many patients by race enrolled in Study 1 used to evaluate the efficacy and safety of IMAAVY.
Figure 2. Baseline Demographics by Race (Efficacy Population [n=153])
Source: Adapted from FDA Review
Figure 3 summarizes how many patients by age enrolled in Study 1 used to evaluate the efficacy and safety of IMAAVY.
Figure 3. Baseline Demographics by Age (Efficacy Population [n=153])
Source: Adapted from FDA Review
Figure 4 summarizes how many patients by ethnicity enrolled in Study 1 used to evaluate the efficacy and safety of IMAAVY.
Figure 4. Baseline Demographics by Ethnicity (Efficacy Population [n=153])
Source: Adapted from FDA Review
Who participated in the trials?
Table 1 summarizes the demographic data for patients enrolled in Study 1 used to evaluate the efficacy and safety of IMAAVY.
Table 1. Baseline Demographics for Study 1
| Demographic Parameters | IMAAVY (N=77) |
Placebo (N=76) |
All (N=153) |
|---|---|---|---|
| Age, n (%) | |||
18-64 |
59 (77) | 57 (75) | 116 (76) |
≥65 |
18 (23) | 19 (25) | 37 (24) |
| Sex, n (%) | |||
Female |
50 (65) | 42 (55) | 92 (60) |
Male |
27 (35) | 34 (45) | 61 (40) |
| Race, n (%) | |||
American Indian or Alaskan Native |
1 (1) | 0 | 1 (<1) |
Asian |
24 (31) | 25 (33) | 49 (32) |
Black or African American |
1 (1) | 1 (1) | 2 (1) |
White |
49 (64) | 47 (62) | 96 (63) |
Not reported |
2 (3) | 3 (4) | 5 (3) |
| Region, n (%) | |||
Asia |
24 (31) | 24 (32) | 48 (31) |
Rest of World |
44 (57) | 43 (57) | 87 (57) |
United States |
9 (12) | 9 (12) | 18 (12) |
| Ethnicity, n (%) | |||
Hispanic or Latino |
8 (10) | 4 (5) | 12 (8) |
Not Hispanic or Latino |
68 (88) | 69 (91) | 137 (90) |
Not reported |
1 (1) | 3 (4) | 4 (3) |
| Weight, kg | |||
Mean (SD) |
76 (17) | 81 (20) | 79 (19) |
Median |
76 | 78 | 76 |
| BMI, kg/m2 | |||
Mean (SD) |
28 (5) | 29 (6) | 28 (6) |
Median |
28 | 28 | 28 |
Source: Adapted from FDA Review
Abbreviations: kg, kilograms; N, number of patients in each arm; n, number of patients in each subgroup; SD, standard deviation.
What are the benefits of this drug?
The patients who received IMAAVY experienced less weakness affecting their activities of daily living compared to those receiving the placebo infusions.
What are the benefits of this drug (results of trials used to assess efficacy)?
Table 2 summarizes the efficacy results for the patients who were evaluated in Study 1 of IMAAVY. The primary efficacy endpoint was the comparison of the average change from baseline in the MG-ADL total score to Weeks 22, 23, and 24 between IMAAVY and placebo. The MG-ADL measures how patients feel about the effect of the disease on their daily activities. A total score ranges from 0 to 24. Higher scores represent greater severity of the disease.
The secondary outcome of the study was the average change from baseline to week 22 and 24 in the Quantitative Myasthenia Gravis (QMG) total score. The QMG assesses muscle weakness. A total score ranges from 0 to 39. Higher scores indicate more severe weakness.
Table 2. Change from Baseline to Week 24 in MG-ADL and QMG Total Scores in Adult Patients With gMG Who Are Anti-AChR or anti-MuSK Antibody Positive in Study 1
| Efficacy Endpoints | IMAAVY N = 77 LS Mean (SE) |
Placebo N = 76 LS Mean (SE) |
IMAAVY Change Relative to Placebo LS Mean Difference (95% CI) |
p-value |
|---|---|---|---|---|
| Primary Endpoint | ||||
MG-ADL Total Score1 |
-4.7 (0.33) | -3.3 (0.34) | -1.5 (-2.4, -0.5) | 0.002 |
| Secondary Endpoint | ||||
QMG Total Score2 |
-4.9 (0.5) | Placebo N = 76 LS Mean (SE) |
-2.8 (-4.2, -1.4) | <0.001 |
Source: IMAAVY Prescribing Information
1 Mean change from baseline over weeks 22, 23, and 24.
2 Mean change from baseline over weeks 22 and 24
Abbreviations: CI=confidence interval; gMG, generalized myasthenia gravis; MG-ADL = myasthenia gravis – activities of daily living scale; QMG = quantitative myasthenia gravis; LS mean = least squares mean; N, number of patients with data in each arm; SE = standard error
Figure 5 shows the average change in the total MG-ADL score over time from the start of treatment in Study 1. The bottom curve shows the response in IMAAVY-treated patients.
Figure 5. Average Change from Baseline in MG-ADL Total Score From the Beginning of Treatment Over Time in Study 1
Source: IMAAVY Prescribing Information
Were there any differences in how well the drug worked in clinical trials among sex, race, and age?
- Sex: The majority of patients in the Study 1 were female because myasthenia gravis affects females more frequently than males. IMAAVY worked similarly in males and females.
- Race: The number of patients of races other than White and Asian was small in Study 1; therefore, differences in how IMAAVY worked among races could not be determined.
- Age: The number of patients ages 65 and older was small in Study 1; therefore, differences in how IMAAVY worked among older age groups could not be determined. The benefit of IMAAVY in pediatric patients aged 12 to 16 years was derived based on the observed and expected similarity in drug exposure levels and effect of IMAAVY on anti-AChR or anti-MuSK antibody levels between pediatric and adult patients. All patients were 12 years of age and older; therefore, differences in how the drug worked among younger age groups could not be determined.
Were there any differences in how well the drug worked in clinical trials among sex, race and age groups?
Table 3 summarizes the results of the subgroup analyses by age and sex in Study 1. IMAAVY worked similarly between males and females. Differences in how IMAAVY worked among age groups and races could not be determined because of the small number of patients in some age and racial categories.
Table 3. Subgroup Analysis of Average Change From Baseline to Week 22, 23, and 24 in MG-ADL total Score in Study 1
| Subgroup | Treatment Arm | No. of Subjects at Baseline (N) |
LS Mean Difference From Placebo (95% CI) |
|---|---|---|---|
| Age | |||
18 to <45 |
Placebo | 30 | -- |
| Nipocalimab | 21 | -2.547 (-4.143, -0.951) | |
≥45 to <65 |
Placebo | 27 | -- |
| Nipocalimab | 38 | -1.279 (-2.749, 0.191) | |
≥65 years |
Placebo | 19 | -- |
| Nipocalimab | 18 | -0.004 (-1.985, 1.978) | |
| Sex | |||
Female |
Placebo | 42 | -- |
| Nipocalimab | 50 | -0.857 (-2.059, 0.345) | |
Male |
Placebo | 34 | -- |
| Nipocalimab | 27 | -2.392 (-3.871, -0.912) | |
| Race | |||
Asian |
Placebo | 25 | -- |
| Nipocalimab | 24 | -1.20 (-2.870, 0.471) | |
White |
Placebo | 47 | -- |
| Nipocalimab | 49 | -1.259 (-2.466, -0.052) | |
Source: Adapted from FDA Review
Abbreviations: LS: least square mean; CI: confidence interval; MG-ADL, myasthenia gravis-activities of daily living; N, number of patients with data in each arm
What are the possible side effects?
IMAAVY may increase the risk for infections. IMAAVY is associated with hypersensitivity reactions, including angioedema and rash, and infusion-related reactions, including headache and influenza-like illness.
The most common side effects of IMAAVY were infections, peripheral edema, muscle spasms, hypersensitivity reaction, abdominal pain, back pain, pyrexia (fever), diarrhea, cough, anemia, dizziness, nausea, hypertension (high blood pressure), and insomnia.
What are the possible side effects (results of trials used to assess safety)?
Table 4 summarizes the most common side effects of IMAAVY that occurred in Study 1.
Table 4. Adverse Reactions in at Least 5% of Patients Treated with IMAAVY and More Frequently Than in Patients Who Received Placebo in Study 1
| Adverse Reaction | IMAAVY N=98 % |
Placebo N=98 % |
|---|---|---|
| Infection | ||
Respiratory tract infectiona |
18 | 13 |
Urinary tract infectionb |
6 | 3 |
Herpes zoster and Herpes simplex |
6 | 2 |
Oral infectionc |
5 | 3 |
| Peripheral edema | 12 | 2 |
| Muscle spasm | 12 | 3 |
| Hypersensitivity reactiond | 8 | 7 |
| Abdominal pain | 8 | 3 |
| Back pain | 8 | 5 |
| Pyrexia (fever) | 7 | 1 |
| Diarrhea | 7 | 3 |
| Cough | 7 | 3 |
| Anemiab | 6 | 4 |
| Dizziness | 5 | 1 |
| Nausea | 5 | 2 |
| Hypertension | 5 | 2 |
| Insomnia | 5 | 2 |
Includes the following reported in patients treated with IMAAVY:
a COVID-19 (and other related terms), pneumonia, bronchitis, pneumonia bacteria
b other related terms
c glossitis, oral candidiasis, pericoronitis, pulpitis dental, tooth abscess, tooth infection
d angioedema, dermatitis atopic, eczema, gingival swelling, rash (and other related terms), urticaria
Source: IMAAVY Prescribing Information
Were there any differences in side effects among sex, race, and age?
- Sex: The occurrence of side effects was similar in males and females in Study 1.
- Race: The number of patients of races other than White and Asian was small; therefore, differences in side effects among races could not be determined.
- Age: The number of patients aged 65 years and older was small; therefore, the differences in side effects among older age groups could not be determined. Side effects observed in 7 pediatric patients aged 12 to 16 years in Study 2 were consistent with those seen in adult patients from Study 1. All patients were 12 years of age and older; therefore, differences in side effects among younger age groups could not be determined.
Were there any differences in side effects of the clinical trials among sex, race, and age groups?
Table 5 summarizes the results of the subgroup analysis by age, sex, race, and ethnicity. The majority of patients in Study 1 were female. Differences in side effects among some age, racial, and ethnic groups could not be determined due to small number of patients enrolled in the study.
Table 5. Subgroup Analysis of Side Effects Occurring in at Least 5% of Patients Treated with IMAAVY and More Frequently Than Placebo in Study 1
| Characteristic | Nipocalimab (N=98) n/Ns (%) |
Placebo (N=98) n/Ns (%) |
|---|---|---|
| Sex | ||
Female |
55/66 (83.3) | 46/56 (82.1) |
Male |
27/32 (84.4) | 36/42 (85.7) |
| Age group, years | ||
≥18 to 44 |
23/27 (85.2) | 27/33 (81.8) |
≥45 to 64 |
38/46 (82.6) | 33/41 (80.5) |
≥65 |
21/25 (84.0) | 22/24 (91.7) |
| Age group ≥75, years | ||
≥75 |
7/8 (87.5) | 11/12 (91.7) |
| Race | ||
American Indian or Alaska Native |
1/1 (100) | 0/0 (NA) |
Asian |
26/28 (92.9) | 26/29 (89.7) |
Black or African American |
1/1 (100) | 0/1 (0) |
White |
52/66 (78.8) | 53/65 (81.5) |
Not reported |
2/2 (100) | 3/3 (100) |
| Ethnicity | ||
Hispanic or Latino |
9/10 (90.0) | 6/6 (100) |
Not Hispanic or Latino |
72/87 (82.8) | 73/89 (82.0) |
Not reported |
1/1 (100) | 3/3 (100) |
Source: Adapted from FDA Review
Abbreviations: N, number of patients in the safety population; n, number of patients with adverse event; Ns, total number of patients for each specific subgroup and were assigned to that specific arm
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.
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