Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Omalizumab · 9 trials · 5 indications
Total NPS ranges from 0 to 8 (sum of 0-4 for left and right nasal passage scores per the following criteria), with a lower score indicating smaller-sized nasal polyps: 0 = No polyps; 1 = Small polyps in the middle meatus not reaching below the inferior border of the middle turbinate; 2 = Polyps reaching below the lower border of the middle turbinate (modified to accommodate those with a middle turbinectomy, such that polyp must have reached the top of the inferior turbinate.); 3 = Large polyps reaching the lower border of the inferior turbinate or polyps medial to the middle turbinate; and 4 = Large polyps causing complete obstruction of the inferior nasal cavity. Two blinded primary independent expert readers reviewed every post-screening recorded video endoscopy for a given participant to determine total NPS. A third reader chose one of the two scores to be used for analysis in cases where there was any discrepancy in total NPS assigned between the two primary readers.
The Nasal Congestion Score (NCS) was assessed daily by the participant via an electronic diary as the response to the following question: Is your nose blocked? The four available response options were scored from 0 (no symptoms) to 3 (severe symptoms): 0 = Not at all; 1 = Mild; 2 = Moderate; and 3 = Severe. For each study day, a score was calculated using an average of the prior 7 days among the available days within the pre-specified window (For Week 24: Study Days 155 to 186), excluding the study day itself, if a value had been recorded by the participant on at least 4 of the prior 7 days; otherwise, the 7-day prior average for that study day was to be considered missing. One calculated (non-missing) 7-day prior average was selected for analysis according to the study day with nearest proximity to Week 24 (Study Day 168), with the earlier selected in the case of a tie. Baseline was defined as the (non-missing) 7-day interval ending on the latest day prior to randomization.
A serious adverse event was defined as any adverse event that met any of the following criteria: was fatal; was life-threatening; required or prolonged inpatient hospitalization; resulted in persistent or significant disability/incapacity; was a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the study drug; or, was a significant medical event in the investigator's judgment. Multiple occurrences of the same serious adverse event in one individual were counted once.
A serious adverse event was defined as any adverse event that met any of the following criteria: was fatal; was life-threatening; required or prolonged inpatient hospitalization; resulted in persistent or significant disability/incapacity; was a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the study drug; or, was a significant medical event in the investigator's judgment. Multiple occurrences of the same serious adverse event in one individual were counted once.
Total NPS ranges from 0 to 8 (sum of 0-4 for left and right nasal passage scores per the following criteria), with a lower score indicating smaller-sized nasal polyps: 0 = No polyps; 1 = Small polyps in the middle meatus not reaching below the inferior border of the middle turbinate; 2 = Polyps reaching below the lower border of the middle turbinate (modified to accommodate those with a middle turbinectomy, such that polyp must have reached the top of the inferior turbinate.); 3 = Large polyps reaching the lower border of the inferior turbinate or polyps medial to the middle turbinate; and 4 = Large polyps causing complete obstruction of the inferior nasal cavity. Two blinded primary independent expert readers reviewed every post-screening recorded video endoscopy for a given participant to determine total NPS. A third reader chose one of the two scores to be used for analysis in cases where there was any discrepancy in total NPS assigned between the two primary readers.
The Nasal Congestion Score (NCS) was assessed daily by the participant via an electronic diary as the response to the following question: Is your nose blocked? The four available response options were scored from 0 (no symptoms) to 3 (severe symptoms): 0 = Not at all; 1 = Mild; 2 = Moderate; and 3 = Severe. For each study day, a score was calculated using an average of the prior 7 days among the available days within the pre-specified window (For Week 24: Study Days 155 to 186), excluding the study day itself, if a value had been recorded by the participant on at least 4 of the prior 7 days; otherwise, the 7-day prior average for that study day was to be considered missing. One calculated (non-missing) 7-day prior average was selected for analysis according to the study day with nearest proximity to Week 24 (Study Day 168), with the earlier selected in the case of a tie. Baseline was defined as the (non-missing) 7-day interval ending on the latest day prior to randomization.
The weekly itch severity score is the sum of the daily itch severity scores over 7 days and ranges from 0 to 21. The daily itch severity score is the average of the morning and evening scores on a scale of 0 (none) to 3 (severe). The Baseline weekly itch severity score is the sum of the daily itch severity scores over the 7 days prior to the first treatment. A higher itch severity score indicates more severe itching. A negative change score indicates improvement.
The percentage of participants with serious adverse events and other adverse events is summarized by MedDRA preferred terms and organ classes in the Reported Adverse Events section below.
The weekly itch severity score is the sum of the daily itch severity scores over 7 days and ranges from 0 to 21. The daily itch severity score is the average of the morning and evening scores on a scale of 0 (none) to 3 (severe). The Baseline weekly itch severity score is the sum of the daily itch severity scores over the 7 days prior to the first treatment. A higher itch severity score indicates more severe itching. A negative change score indicates improvement.
The UAS is a composite diary-recorded score, which is the sum of the numeric severity intensity ratings (0 = none to 3 = intense) for 1) the number of wheals (hives) and 2) the intensity of the pruritus (itch). The UAS7 is the sum of the daily average UAS (morning and evening values) for 7 days. The maximum UAS7 score is 42.
The primary analysis included two tests: a test for superiority of the lyophilized formulation of omalizumab compared with placebo in the change of allergen concentration and a test for the superiority of the aged liquid omalizumab compared with placebo. The difference for the change in the allergen concentration between the lyophilized formulation of omalizumab and placebo, and between the aged liquid omalizumab and placebo were assessed by the exact Wilcoxon-Mann-Whitney test.
All grades according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), v3.0. An SAE is defined as an adverse event that results in death, is life threatening, requires hospitalization, results in significant disability, results in birth defect, or is considered a significant medical event by the investigator
| Arm | Type | Description |
|---|---|---|
| Cohort A: Study GA39688 Omalizumab | EXPERIMENTAL | Participants who received omalizumab once every 2 weeks (Q2W) or once every 4 weeks (Q4W) in Study GA39688 will continue to receive omalizumab at Week 24 at the same dosing schedule. |
| Cohort A: Study GA39688 Placebo | EXPERIMENTAL | Participants who received placebo Q2W or Q4W in Study GA39688 will start receiving omalizumab Q2W or Q4W at Week 24 at the same dosing schedule. |
| Cohort B: Study GA39855 Omalizumab | EXPERIMENTAL | Participants who received omalizumab Q2W or Q4W in Study GA39855 will continue to receive omalizumab at Week 24 at the same dosing schedule. |
| Cohort B: Study GA39855 Placebo | EXPERIMENTAL | Participants who received placebo Q2W or Q4W in Study GA39855 will start receiving omalizumab Q2W or Q4W at Week 24 at the same dosing schedule. |
| Omalizumab | EXPERIMENTAL | Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy. |
| Placebo | PLACEBO_COMPARATOR | Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy. |
| Omalizumab 75 mg | EXPERIMENTAL | Omalizumab 75 mg sc every 4 weeks |
| Omalizumab 150 mg | EXPERIMENTAL | Omalizumab 150 mg sc every 4 weeks |
| Omalizumab 300 mg | EXPERIMENTAL | Omalizumab 300 mg sc every 4 weeks. |
| Omalizumab 600 mg | EXPERIMENTAL | Omalizumab (Xolair) was administered subcutaneously on Day 0 of the study. Participants remained on stable doses of their pre-allocation Chronic Idiopathic Urticaria (CIU) H1 antihistamine treatment throughout the study, and were provided diphenhydramine as rescue medication for pruritus relief on an as-needed basis. |
| 1 | EXPERIMENTAL | - |
| 2 | EXPERIMENTAL | - |
| 3 | PLACEBO_COMPARATOR | - |
| Name | Type | Description |
|---|---|---|
| Omalizumab | DRUG | Omalizumab will be administered as a subcutaneous (SC) injection Q2W or Q4W. |
| Placebo | DRUG | Participants will not be receiving placebo in this OLE study. Participants who were randomized to the placebo arms for 24 weeks in studies GA39688/GA39855 and then enter this OLE study will receive omalizumab, but they will be placed in separate analysis cohorts. |
| H1 antihistamine, H2 antihistamine, leukotriene receptor antagonist | DRUG | Participants were required to maintain stable doses of their pre-randomization combination therapy with an H1 antihistamine and either an H2 blocker or leukotriene receptor antagonist, or all 3 drugs in combination, throughout the 24-week treatment period and 16-week follow-up period of the 40-week study. |
| Diphenhydramine | DRUG | Participants were provided with diphenhydramine 25 mg for itch relief on an as-needed basis, up to a maximum of 3 doses within 24 hours for the duration of the 40-week study. |
| H1 antihistamines | DRUG | Patients received one of the following: Cetirizine 10 mg once per day (QD), Levocetirizine dihydrochloride 5 mg QD, Fexofenadine 60 mg twice per day or 180 mg QD, Loratadine 10 mg QD or Desloratadine 5 mg QD |
Inclusion Criteria: * Ability to comply with the study protocol, in the investigator's judgment * Participation in Study GA39688 or GA39855, including completion of endoscopy and other assessments at Week 24, without discontinuation of study drug * Completion of eDiary daily assessments for at leas...
Omalizumab is an investigational drug being studied for peanut hypersensitivity, chronic idiopathic urticaria, nasal polyps, asthma, and allergic asthma. It is developed by Roche Holding AG (RHHBY) and is currently in Phase 3 clinical development. The drug has not been approved by the FDA and remains under investigation.
Omalizumab targets immunoglobulin E (IgE), a key driver of allergic responses. By binding to IgE, it is designed to reduce allergic inflammation. This mechanism is relevant across its studied indications, including allergic asthma and nasal polyps.
Omalizumab is developed by Roche Holding AG, a biopharmaceutical company traded under the ticker RHHBY. Roche is conducting clinical trials to evaluate the drug for multiple allergic and inflammatory conditions.
Omalizumab is in Phase 3 clinical development. It is an investigational drug and has not been approved by the FDA. Four clinical trials have been completed, with no active trials currently ongoing.
Omalizumab has completed four clinical trials, including NCT00434434 in mild allergic asthma, NCT03280537 and NCT03280550 in chronic rhinosinusitis with nasal polyps, and NCT03478930, an extension study in the same condition. All trials are completed.
Omalizumab is also known as Xolair, a brand name for the drug. It is being studied for conditions such as nasal polyps and allergic asthma, and is developed by Roche Holding AG.