Efficacy

Proven protection against infection

The annual rate per patient-year in the North American clinical study was1:

0.012

0.012 ASBIs (upper limit of 99% CI, 0.024;

P<0.0001*); 1 ASBI occurred†

2.41

2.41 infections of any kind 

(95% CI, 1.89–3.03)

The impact of reduced infections for patients during the study:

Hospital icon with a line through it representing fewer hospital days.

0.06 days

per patient-year spent in the

hospital due to infections 

(95% CI, 0.03-0.11)

School icon with a line through it, representing fewer missed school days.

1.16 days

per patient-year missed from work, school,
or performing daily activities
due to illness or infection
(95% CI, 0.70-1.79)

*Significantly lower (P<0.0001) than the threshold of 1.0 per patient-year which provides substantial evidence of efficacy.

†Pneumonia was reported in a 78-year-old patient who had a specific antibody deficiency and allergic bronchopulmonary aspergillosis.

CI=confidence interval. 

ASBI = Acute serious bacterial infection

North American clinical study1: CUVITRU was studied in 77 patients (≥2 years of age) with PI in a prospective, open-label, noncontrolled, multicenter study. Efficacy was determined in 53 adults aged ≥16 years, 6 adolescents aged 12 to <16 years, and 15 children aged 2 to <12 years. The primary outcome measure was the annualized rate of acute serious bacterial infections (ASBIs). For the overall efficacy population (N=74), the annualized ASBI rate was 0.012 (upper limit of 99% CI, 0.024) during CUVITRU treatment. Median treatment duration was 380.5 days (range, 30–629 days).

Consistent IG levels between infusions vs IVIG

CUVITRU maintained lower peak IgG and higher trough IgG levels than IVIG1

PK results from a prospective, open-label, noncontrolled, multicenter clinical study1*

Median peak IgG levels

Bar graph depicting median peak IgG levels of CUVITRU vs IVIG 10%.

Geometric mean trough IgG levels

Bar graph depicting geometric mean trough IgG levels of CUVITRU vs IVIG 10%.

IVIG=intravenous immune globulin.

In the North American clinical study, pharmacokinetic (PK) parameters were evaluated in 60 patients ≥2 years of age with PI, patients were administered intravenously at 3 to 4 week intervals with [GAMMAGARD LIQUID, Immune Globulin (Human), 10%] and then switched to weekly subcutaneous CUVITRU infusions.

See section above for Study Design information.

CUVITRU can be dosed weekly to every other week and still maintain consistent IG levels2

Consistent total IgG concentrations based on a population PK model

Weekly dosing

Weekly dosing graph comparing CUVITRU and IVIG 10%.

Q4W=every 4 weeks; QW=every week. 

This model is not intended to show any efficacy results.

Every-other-week dosing

Every-other-week dosing line graph comparing CUVITRU and IVIG 10%.

BIW=every other week; Q4W=every 4 weeks.

This model is not intended to show any efficacy results.

A total of 2063 IgG concentrations from 102 evaluable patients enrolled in 2 prospective, open-label, noncontrolled, multicenter clinical studies to determine the efficacy, safety, tolerability, and PK of CUVITRU in patients ≥2 years of age with PI. IgG population PK was characterized by nonlinear mixed-effects modeling and validated using data splitting and a visual predictive check. IgG profiles were simulated for 1000 patients/interval treated with CUVITRU. A CUVITRU adjustment factor of 130% was used to simulate CUVITRU to IVIG AUC ratios for weekly or biweekly CUVITRU dosing intervals and a monthly IVIG 10% dosing interval.

CUVITRU® was shown to significantly improve patient quality of life vs IVIG in terms of convenience (TSQM-9*) and treatment interference (LQI*) 1

Hand with a check mark icon.

Convenience measured as4:

  • Ease of using the medication in its
    current form
  • Ease of planning when to use the
    medication each time
  • Convenience in taking the medication
    as instructed
  • Freedom to take trips or move
  • Scheduled according to patient’s convenience
IV icon.

Treatment interference measured as4:

  • Interference with social/family life
  • Time waiting
  • Treatment is worthwhile
  • Dependence on others

Other Results:

  • In the TSQM-9, there was no significant improvement in the perception of effectiveness and global satisfaction domains in either age group: 2 to 12 years and 13 years and older
  • In the LQI, the change reported in therapy-related problems and therapy setting domains across all age groups was not statistically significant
  • In other assessments, health-related quality of life differences between IVIG and SCIG treatment were not statistically significant

In the North American clinical study, CUVITRU was shown to significantly improve quality of life vs IVIG in terms of convenience (TSQM-9*) and treatment interference (LQI*) in patients with PI. Health-related quality of life was assessed using the Pediatric Quality of Life InventoryTM (PEDS-QL) questionnaire14 (pediatric subjects) or the self-administered SF-36 survey15 (adult subjects). Quality of life was analyzed separately for the age groups 2 to 4 and 5 to 7 years (PEDS- QL, observer: parent), 8 to 12 and 13 years (PEDS-QL, observer: subject) and 14 years and older (SF-36, observer: subject). Treatment Satisfaction Questionnaire for Medication (TSQM-9) and Life Quality Index (LQI) questionnaire were assessed in patients ages 2 to 12 years (observer: parent) and 13 years and older (observer: patient). Each was assessed in 3 domains: Effectiveness, Convenience, and Global Satisfaction for TSQM-9; and Treatment Interference, Therapy-Related Problems, and Therapy Settings for LQI.1

LQI=Life Quality Index; TSQM=Treatment Satisfaction Questionnaire for Medication.

 *TSQM-9, P<0.001; LQI, P=0.008.

Review safety information

Review Important Safety Information, including contraindications and other specific warnings and precautions to consider when prescribing and monitoring patients treated with CUVITRU.

See safety information

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Demonstrated tolerability  

See how CUVITRU had low rates of systemic adverse reactions and low incidence of local adverse reactions (ARs).1,2

Explore safety data

References

  1. CUVITRU. Prescribing information. Baxalta US Inc.; 2021. 
  2. Dumas T, Berry NS, Wolfsegger M, et al. Population pharmacokinetic modeling and simulation of immunoglobulin exposure with varying dosing intervals of subcutaneous immunoglobulin 20% (Ig20Gly) in patients with primary immunodeficiency diseases. Int Immunopharmacol. 2019;71:404-410.
  3. Meckley LM, Wu Y, Ito D, et al. Patient experience with subcutaneous immunoglobulin 20%, Ig20Gly, for primary immunodeficiency diseases: a prespecified post hoc analysis of combined data from 2 pivotal trials. BMC Immunol. 2020;21(1):24. doi:10.1186/s12865-020-00346-z