Drug Trials Snapshot: RHAPSIDO
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 RHAPSIDO 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).
RHAPSIDO (remibrutinib)
RAP-si-doe
Novartis Pharmaceuticals Corp
Original Approval date: September 30, 2025
DRUG TRIALS SNAPSHOT SUMMARY:
What is the drug for?
RHAPSIDO is a kinase inhibitor that is approved for the treatment of chronic spontaneous urticaria (CSU) in adult patients who remain symptomatic despite H1-antihistamine treatment.
How is this drug used?
RHAPSIDO is a tablet taken by mouth twice daily.
Who participated in the clinical trial?
The FDA approved RHAPSIDO based on evidence from two clinical trials of 912 adult patients with CSU. The trials were conducted at 237 sites in 18 countries including the United States, Europe, Asia, Latin America, Caribbean, Canada, and Japan. There were 200 patients enrolled from site in the United States and 712 patients from sites outside the United States. The same trials were used to assess both efficacy and safety.
How were the trials designed?
RHAPSIDO was studied in two identical clinical trials with 912 patients who had CSU that was not well controlled by antihistamine medicines. Patients were randomly assigned to receive either RHAPSIDO 25 mg twice daily or a placebo (sugar pill) for 24 weeks. The main goals were to see if RHAPSIDO reduced itching and hives better than placebo after 12 weeks of treatment.
The trials (REMIX-1 and REMIX-2) were randomized, double-blind, placebo-controlled studies. Patients had to have chronic spontaneous urticaria for at least six months with ongoing symptoms despite antihistamine treatment. All patients continued their background antihistamine therapy. The co-primary endpoints were the change from baseline in weekly itch severity score (ISS7) and weekly hives severity score (HSS7) at Week 12.
DEMOGRAPHICS SNAPSHOT
Figure 1 shows how many male and female patients were enrolled in the clinical trial used to evaluate the efficacy of RHAPSIDO.
Figure 1. Baseline Demographics by Sex, Efficacy Population
Source: Adapted from FDA Review
Figure 2 shows how many patients by race were enrolled in the clinical trial used to evaluate the efficacy of RHAPSIDO.
Figure 2. Baseline Demographics by Race, Efficacy Population
Source: Adapted from FDA Review
*Other includes: More than one race (12 participants), unknown race (4 participants) and Native Hawaiian or Pacific Islander (1 participant).
Figure 3 shows how many patients by age were enrolled in the clinical trial used to evaluate the efficacy of RHAPSIDO.
Figure 3. Baseline Demographics by Age, Efficacy Population
Source: Adapted from FDA Review
Figure 4 shows how many patients by ethnicity were enrolled in the clinical trial used to evaluate the efficacy of RHAPSIDO.
Figure 4. Baseline Demographics by Ethnicity, Efficacy Population
Source: Adapted from FDA Review
Who participated in the trials?
Table 1. Baseline Demographics of Efficacy Trials by Age, Race, Sex, and Ethnicity
| Demographic | REMIX-1 |
REMIX-2 |
||||
|---|---|---|---|---|---|---|
| RHAPSIDO N=309 |
Placebo N=153 |
Total N=462 |
RHAPSIDO N=297 |
Placebo N=153 |
Total N=450 |
|
| Age group, years, n (%) | ||||||
≥18 to <65 |
279 (90) | 140 (92) | 419 (91) | 274 (92) | 142 (93) | 416 (92) |
≥65 to <85 |
30 (10) | 13 (8) | 43 (9) | 23 (8) | 11 (7) | 34 (8) |
| Age, years | ||||||
Mean (SD) |
44.5 (14.3) | 46.0 (13.3) | 45.0 (13.9) | 42.0 (14.4) | 41.3 (14.6) | 41.7 (14.5) |
Median |
44 | 47 | 45 | 41 | 40 | 41 |
Min, max |
18, 79 | 18, 79 | 18, 79 | 18, 80 | 18, 81 | 18, 81 |
| Sex, n (%) | ||||||
Female |
208 (67) | 105 (69) | 313 (68) | 195 (66) | 99 (65) | 294 (65) |
Male |
101 (33) | 48 (31) | 149 (32) | 102 (34) | 54 (35) | 156 (35) |
| Race, n (%) | ||||||
White |
184 (60) | 86 (56) | 270 (58) | 157 (53) | 77 (50) | 234 (52) |
Asian |
94 (30) | 45 (29) | 139 (30) | 129 (43) | 72 (47) | 201 (45) |
American Indian or Alaska Native |
12 (4) | 14 (9) | 26 (6) | 0 | 0 | 0 |
Black or African American |
12 (4) | 3 (2) | 15 (3) | 7 (2) | 3 (2) | 10 (2) |
More than one race |
6 (2) | 2 (1) | 8 (2) | 3 (1) | 1 (1) | 4 (1) |
Unknown |
1 (0) | 2 (1) | 3 (1) | 1 (0) | 0 | 1 (0) |
Native Hawaiian or Pacific Islander |
0 (0) | 1 (1) | 1 (0) | 0 | 0 | 0 |
| Ethnicity, n (%) | ||||||
Not Hispanic or Latino |
235 (76) | 111 (73) | 346 (75) | 281 (95) | 147 (96) | 428 (95) |
Hispanic or Latino |
74 (24) | 42 (27) | 116 (25) | 16 (5) | 5 (3) | 21 (5) |
Unknown |
0 (0) | 0 (0) | 0 (0) | 0 (0) | 1 (1) | 1 (<1) |
| Geographic region, n (%) | ||||||
Asia, Middle East and Africa |
99 (32) | 49 (32) | 148 (32) | 93 (31) | 50 (33) | 143 (32) |
United States |
77 (25) | 38 (25) | 115 (25) | 56 (19) | 29 (19) | 85 (19) |
Europe |
63 (20) | 31 (20) | 94 (20) | 79 (27) | 39 (25) | 118 (26) |
Latin America, Caribbean, and Canada |
51 (17) | 27 (18) | 78 (17) | 26 (9) | 13 (8) | 39 (9) |
Japan |
19 (6) | 8 (5) | 27 (6) | 43 (14) | 22 (14) | 65 (14) |
| Body mass index | ||||||
Mean (SD) |
27.8 (6.4) | 28.4 (6.5) | 28.0 (6.4) | 27.0 (6.5) | 26.9 (5.9) | 27.0 (6.3) |
Median |
26.9 | 27.5 | 27.1 | 25.7 | 25.7 | 25.7 |
Min, max |
16, 58 | 16, 54 | 16, 58 | 15, 53 | 18, 51 | 15, 53 |
Missing |
0 | 1 | 1 | 0 | 0 | 0 |
| Body mass index group, n (%) | ||||||
<25 (kg/m2) |
119 (39) | 56 (37) | 175 (38) | 137 (46) | 69 (45) | 206 (46) |
25 to <30 (kg/m2) |
95 (31) | 43 (28) | 138 (30) | 84 (28) | 50 (33) | 134 (30) |
≥30 (kg/m2) |
95 (31) | 53 (35) | 148 (32) | 76 (26) | 34 (22) | 110 (24) |
Source: Adapted from FDA Review
Abbreviations: SD, standard deviation
What are the benefits of this drug?
RHAPSIDO helped reduce itching and hives in adults with CSU. In both studies, patients taking RHAPSIDO had significantly less itching and fewer hives compared to patients taking placebo after 12 weeks. About half of patients taking RHAPSIDO had well-controlled symptoms (minimal itching and hives), and about 30% had complete relief from symptoms.
What are the benefits of this drug (results of trials used to assess efficacy)?
In both REMIX-1 and REMIX-2 trials, RHAPSIDO demonstrated improvements in the weekly itch severity score (ISS7) and weekly hives severity score (HSS7) at Week 12 compared to placebo. RHAPSIDO also showed improvement in disease control (UAS7 ≤6) and complete symptom relief (UAS7 = 0).
Table 2. HSS7 Efficacy Results, Efficacy Population
| HSS7 (Range 0 to 21) | REMIX-1 |
REMIX-2 |
||
|---|---|---|---|---|
| RHAPSIDO N=309 |
Placebo N=153 |
RHAPSIDO N=297 |
Placebo N=153 |
|
| Baseline, mean (SD) | 16.0 (4.5) | 15.5 (4.5) | 16.0 (4.6) | 15.7 (4.4) |
| Change from baseline, LS mean (SE) | -10.47 (0.40) | -6.86 (0.55) | -10.47 (0.39) | -6.00 (0.53) |
| Treatment difference LS mean (95% CI) | -3.61 (-4.85, -2.36) |
-4.47 (-5.71, -3.23) |
||
| p-Value | <0.001 |
<0.001 |
||
Source: Adapted from FDA Review
Abbreviations: CI, confidence interval; HSS7, weekly hives severity score; LS, least squares; SD, standard deviation; SE, standard error
Table 3. ISS7 Efficacy Results, Efficacy Population
| ISS7 (Range 0 to 21) | REMIX-1 |
REMIX-2 |
||
|---|---|---|---|---|
| RHAPSIDO N=309 |
Placebo N=153 |
RHAPSIDO N=297 |
Placebo N=153 |
|
| Baseline, mean (SD) | 14.8 (4.1) | 14.4 (4.0) | 14.4 (4.3) | 13.9 (4.1) |
| Change from baseline, LS mean (SE) | -9.52 (0.34) | -6.89 (0.47) | -8.95 (0.34) | -5.72 (0.45) |
| Treatment difference LS mean (95% CI) | -2.63 (-3.70, -1.56) |
-3.23 (-4.29, -2.16) |
||
| p-Value | <0.001 |
<0.001 |
||
Source: Adapted from FDA Review
Abbreviations: CI, confidence interval; ISS7, weekly itch severity score; LS, least squares; SD, standard deviation; SE, standard error
Were there any differences in how well the drug worked in clinical trials among sex, race, and age?
- Sex: RHAPSIDO worked similarly in males and females.
- Race: RHAPSIDO worked similarly in White and Asian patients. The number of patients of other races was small; therefore, differences in how RHAPSIDO worked among other races could not be determined.
- Age: RHAPSIDO worked similarly in patients younger and older than 65 years of age.
- Ethnicity: RHAPSIDO worked similarly in Hispanic or Latino and not Hispanic or Latino patients.
Were there any differences in how well the drug worked in clinical trials among sex, race and age groups?
Subgroup analyses of the primary endpoint based on sex, race, age, and ethnicity were performed. In general, there were no significant differences in the efficacy of RHAPSIDO based on the demographic subgroups, but some subgroups were small; therefore, differences in certain subgroups could not be determined.
Table 4. Efficacy Results by Sex, Race, and Age, Efficacy Population
| Subgroup | RHAPSIDO N |
Placebo N |
Treatment Difference HSS7 (95% CI) | Treatment Difference ISS7 (95% CI) |
|---|---|---|---|---|
| Sex | ||||
Female |
403 | 204 | -3.86 (-4.74, -2.98) | -2.51 (-3.44, -1.58) |
Male |
203 | 102 | -4.80 (-6.31, -3.29) | -3.77 (-5.06, -2.47) |
| Race | ||||
White |
341 | 163 | -4.94 (-6.13, -3.74) | -3.37 (-4.40, -2.35) |
Asian |
223 | 117 | -2.82 (-4.23, -1.40) | -2.09 (-3.31, -0.88) |
Black or African American |
19 | 6 | -3.63 (-9.84, 2.58) | -3.78 (-9.11, 1.55) |
American Indian or Alaska Native |
12 | 14 | -2.95 (-7.81, 1.91) | -3.49 (-7.66, 0.67) |
Multiple |
11 | 5 | -3.95 (-10.56, 2.67) | -3.81 (-9.48, 1.86) |
| Age, years | ||||
<65 |
553 | 282 | -4.11 (-5.02, -3.19) | -3.00 (-3.79, -2.21) |
≥65 |
53 | 24 | -3.21 (-6.27, -0.14) | -1.99 (-4.61, 0.63) |
| Ethnicity | ||||
Hispanic or Latino |
90 | 47 | -3.26 (-5.56, -0.97) | -2.54 (-4.50, -0.57) |
Not Hispanic or Latino |
516 | 258 | -4.18 (-5.13, -3.23) | -2.99 (-3.81, -2.18) |
Source: Adapted from FDA Review
Negative values indicate improvement (reduction in symptoms).
Abbreviations: CI, confidence interval; HSS7, weekly hives severity score; ISS7, weekly itch severity score
What are the possible side effects?
The most common side effects of RHAPSIDO include upper respiratory tract infections (including common cold), bleeding under the skin (such as bruising, small red spots), headache, nausea, and stomach pain. RHAPSIDO may increase the risk of bleeding due to its effect on blood platelets.
What are the possible side effects (results of trials used to assess safety)?
Table 5. Safety Results, Safety Population
| Adverse Reaction | RHAPSIDO N=606 n (%) |
Placebo N=306 n (%) |
|---|---|---|
| Nasopharyngitisa | 67 (11) | 27 (9) |
| Bleedingb | 55 (9) | 6 (2) |
| Headachec | 41 (7) | 19 (6) |
| Nausea | 18 (3) | 5 (2) |
| Abdominal paind | 18 (3) | 6 (2) |
Source: Adapted from RHAPSIDO Prescribing Information
a Includes acute sinusitis, chronic sinusitis, nasopharyngitis, pharyngitis, pharyngitis streptococcal, rhinitis, rhinitis allergic, and upper respiratory tract infection
b Includes conjunctival bleeding, contusion, ecchymosis, epistaxis, gingival bleeding, hematoma, hematuria, hemorrhagic ovarian cyst, intermenstrual bleeding, petechiae, purpura, and urinary occult blood
c Includes headache and migraine
d Includes abdominal discomfort, abdominal distention, abdominal pain, and abdominal pain upper
Were there any differences in side effects among sex, race, and age?
- Sex: The occurrence of side effects was similar in males and females, though bleeding-related side effects were more common in females (11.4%) compared to males (4.4%).
- Race: The occurrence of side effects was similar in White and Asian patients. The number of patients of other races was small; therefore, differences in side effects among other races could not be determined.
- Age: The occurrence of side effects was similar in patients younger and older than 65 years of age.
- Ethnicity: The occurrence of side effects was similar in Hispanic or Latino and not Hispanic or Latino patients.
Were there any differences in side effects of the clinical trials among sex, race, and age groups?
Table 6. Side Effects by Sex, Race, Age, and Ethnicity, Safety Population
| Subgroup | Any AE n/Ns (%) |
Bleeding n/Ns (%) |
Nasopharyngitis n/Ns (%) |
|---|---|---|---|
| Sex | |||
| Female | |||
RHAPSIDO |
271/403 (67) | 46/403 (11.4) | 52/403 (12.9) |
Placebo |
136/204 (67) | 6/204 (2.9) | 15/204 (7.4) |
| Male | |||
RHAPSIDO |
122/203 (60) | 9/203 (4.4) | 15/203 (7.4) |
Placebo |
62/102 (61) | 1/102 (1.0) | 12/102 (11.8) |
| Age, years | |||
| <65 | |||
RHAPSIDO |
355/553 (64) | 50/553 (9.0) | 61/553 (11.0) |
Placebo |
182/282 (65) | 7/282 (2.5) | 25/282 (8.9) |
| ≥65 | |||
RHAPSIDO |
38/53 (72) | 5/53 (9.4) | 6/53 (11.3) |
Placebo |
16/24 (67) | 0/24 (0) | 2/24 (8.3) |
| Race | |||
| White | |||
RHAPSIDO |
221/341 (65) | 34/341 (10.0) | 47/341 (13.8) |
Placebo |
112/163 (69) | 6/163 (3.7) | 18/163 (11.0) |
| Asian | |||
RHAPSIDO |
142/223 (64) | 18/223 (8.1) | 14/223 (6.3) |
Placebo |
71/117 (61) | 1/117 (0.9) | 9/117 (7.7) |
| Black or African American | |||
RHAPSIDO |
15/19 (79) | 2/19 (10.5) | 1/19 (5.3) |
Placebo |
4/6 (67) | 0/6 (0) | 0/6 (0) |
| American Indian or Alaska Native | |||
RHAPSIDO |
7/12 (58) | 0/12 (0) | 3/12 (25.0) |
Placebo |
7/14 (50) | 0/14 (0) | 0/14 (0) |
| Multiple/Other | |||
RHAPSIDO |
8/11 (73) | 1/11 (9.1) | 2/11 (18.2) |
Placebo |
3/6 (50) | 0/6 (0) | 0/6 (0) |
| Ethnicity | |||
| Hispanic or Latino | |||
RHAPSIDO |
54/90 (60) | 5/90 (5.6) | 13/90 (14.4) |
Placebo |
26/47 (55) | 0/47 (0) | 1/47 (2.1) |
| Not Hispanic or Latino | |||
RHAPSIDO |
339/516 (66) | 50/516 (9.7) | 54/516 (10.5) |
Placebo |
171/258 (66) | 7/258 (2.7) | 26/258 (10.1) |
Source: Adapted from FDA Review
Abbreviations: AE, adverse event; n, number of patients with adverse event; Ns, total number of patients for each specific subgroup and were assigned to that specific arm
GLOSSARY
ADVERSE REACTION: A harmful or unpleasant effect that happens when taking a medicine.
CHRONIC SPONTANEOUS URTICARIA (CSU): A skin condition that causes itchy hives (raised, red bumps) that come and go for at least 6 weeks without a known cause.
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.
CONFIDENCE INTERVAL (CI): A range of values that shows how certain we are about a study result. A narrower range means we are more confident in the result.
DOUBLE-BLIND: A type of study where neither the patients nor the doctors know which treatment the patient is receiving (the actual drug or placebo).
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.
ENDPOINT: The main result that researchers measure in a clinical trial to see if a treatment works.
H1-ANTIHISTAMINE: A type of allergy medicine commonly used to treat hives and itching.
HIVES: Raised, red, itchy bumps on the skin that can appear and disappear quickly.
KINASE INHIBITOR: A type of medicine that blocks certain proteins (called kinases) that send signals inside cells.
LEAST SQUARES (LS) MEAN: A statistical method used to calculate the average effect of a treatment while accounting for other factors that might influence the results.
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.
RANDOMIZED: A method of assigning patients to different treatment groups by chance, like flipping a coin, to make sure the groups are similar.
STANDARD DEVIATION (SD): A number that shows how much the individual results vary from the average result.
STANDARD ERROR (SE): A measure of how precise an average result is likely to be.
SUBGROUP: A subset of the population studied in a clinical trial. Demographic subsets include sex, race, and age groups.
TREATMENT DIFFERENCE: The difference in results between patients who received the study drug and those who received placebo.
URTICARIA ACTIVITY SCORE (UAS7): A way to measure how severe hives and itching are over a 7-day period, combining both itch and hives scores.
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