TABLE 1.

Summary of studies examining the impact of omalizumab added to conventional therapy in patients with uncontrolled asthma

Study (n)	Characteristics of patients at baseline	Study duration and type	Concomitant therapy	Primary efficacy end point	Rate with treatment vs control	Additional significant results (omalizumab vs comparator)
Solèr et al (24) (n=546)	Age 12 to 75 years Moderate to severe allergic asthma BDP 769/772 μg/day (treatment/placebo groups)	28 weeks (SSP 16, SRP 8, MP 4) Multicentre, randomized, double-blind, placebo-controlled, parallel group	BDP at dose required for stability in SSP In SRP, BDP reduced by 25% of baseline at each visit* Salbutamol 100 μg/puff for rescue	Exacerbation rate (episodes per subject per year)	SSP: 0.28 vs 0.66, P<0.001 SRP: 0.36 vs 0.75, P<0.001	Median daily ICS dose 100 μg vs 300 μg, P<0.001 ICS dose reduced by ≥50% in 79% vs 55% of patients Withdrawal of ICS in 43% vs 19% Significantly lower use of rescue medication, P<0.001
Buhl et al (40)† (n=483)	Age 12 to 75 years Moderate to severe allergic asthma BDP 766/773 μg/day (treatment/placebo groups)	24 weeks Multicentre, randomized, double-blind, placebo-controlled, parallel group	BDP or other ICS at lowest effective dose Other asthma medications as required	Exacerbation rate (episodes per subject per year)	0.48 vs 1.14, P<0.001	24% vs 40.6% of patients had ≥1 exacerbation, P<0.001 Mean BDP equivalent dose 253 μg/day vs 434 μg/day ~35% vs ~15% required no ICS Lower use of concomitant therapies
Busse et al (37) (n=525)	Age 12 to 75 years Moderate to severe allergic asthma BDP 568/570 μg/day (treatment/placebo groups)	28 weeks (SSP 16, SRP 8, MP 4) Randomized, double-blind, placebo-controlled, multicentre, parallel group	BDP at dose required for stability in SSP; in SRP, dose reduced by 25% of baseline at each visit* Albuterol: 2 puffs (90 μg/puff) as needed (maximum 8 puffs daily) for rescue Immunotherapy and nonasthma medication maintained	Exacerbation rate (episodes per subject per year)	SSP: 0.28 vs 0.54, P=0.006 SRP: 0.39 vs 0.66, P=0.009	Median reduction in ICS dose 75% vs 50%, P<0.001 ≥ 50% reduction in BDP dose achieved by 72.4% vs 54.9%, P<0.001 BDP discontinued in 39.6% vs 19.1%, P<0.001
Lanier et al (41)‡ (n=460)	Age 12 to 75 years Moderate to severe allergic asthma BDP 565/552 μg/day (treatment/placebo groups)	24 weeks Double-blind, placebo-controlled	BDP or other ICS at lowest effective dose Other asthma medications as required	Exacerbation rate (episodes per subject per year)	0.6 vs 0.83, P=0.023	31.8% vs 42.8% of patients had ≥1 exacerbation, P=0.015 Mean BDP equivalent dose 227 μg/day vs 335 g/day, P<0.001 Significantly more treated patients achieved =50% ICS reduction or stopped ICS Lower use of concomitant therapies
INNOVATE (38) (n=419)	Age 12 to 75 years Recent history of exacerbations Inadequately controlled despite high-dose ICS (>2300 μg/day) and LABA	28 weeks Randomized, double-blind, parallel group, multicentre Omalizumab added to existing treatment	High-dose ICS and LABA 2/3 of patients received additional controller medications, including oral corticosteroids in 22%	Exacerbation rate (episodes per subject per year)	0.68 vs 0.91, P=0.042	50% reduction in severe exacerbations, P=0.002 44% reduction in total emergency visits, P=0.038
ETOPA (39) (n=312)	Age 12 to 73 years Persistent, moderate to severe allergic asthma ICS ≥400 μg/day (adolescents) or ≥800 μg/day (adults)	52 weeks Randomized, open-label, multicentre, parallel group Omalizumab added to best standard care	ICS daily: 1000 μg (30.1%), 2000 μg (38.8%), 4000 μg (16%) LABA 78% Antileukotrienes 28% SCS 21.2% Salbutamol for rescue	Rate of asthma-related deterioration incidents per patient-year	4.92 vs 9.76, P<0.001	Clinically significant exacerbations reduced by 60.8%, P<0.001 Change in mean daily dose of ICS: −342 μg/day vs +68 μg/day, P<0.001 Median rescue bronchodilator use 0.6 vs 3 puffs/day, P<0.001
Holgate et al (43) (n=246)	Age 12 to 75 years Severe allergic asthma ≥ 1000 μg/dayfluticasone Mean daily dose of1362/1375 μg/day (placebo/treatment groups)	32 weeks (16 add-on, 16 SRP) Randomized, double-blind, multicentre, placebo-controlled Omalizumab added to existing therapy	LABA (43.3% and 49.2% in treatment and placebo groups, respectively) Short-acting beta-agonists as needed	Per cent reduction in ICS use from baseline	Median ICS reduction 60% vs 50%, P=0.003 Mean 57.2% vs 43.3%, P=0.003	≥50% ICS reduction in 73.8% vs 50.8% of patients, P=0.001 Reduction to ≤500 μg/day in 60.3% vs 45.8% of patients, P=0.026

^*For eight weeks until total elimination or until forced expiratory volume in 1 s declined by 20% or more, or asthma worsened;

^†Extension of study by Solèr et al (24);

^‡Extension of study by Busse et al (37). BDP Beclomethasone dipropionate; ETOPA Efficacy and Tolerability of Omalizumab in Poorly controlled Asthma; ICS Inhaled corticosteroids; INNOVATE Investigation of Omalizumab in Severe Asthma Treatment; LABA Long-acting beta₂-agonists; MP Maintenance phase; SCS Systemic corticosteroids; SRP Steroid reduction phase; SSP Stable steroid phase; vs Versus