Prescribing information

 

ADAKVEO® demonstrated reductions in VOCs across all sickle cell disease (SCD) patient types in the SUSTAIN study1*

The SUSTAIN* study assessed the efficacy and safety of ADAKVEO® in preventing recurrent VOCs. The primary endpoint of the study was the annual rate of SCD-related pain crises with ADAKVEO® vs. placebo1

Patients receiving hydroxyurea/hydrocarbamide (HU/HC) were included in the study1

Primary endpoint: A significant 45% reduction (P=0.01) in the median annual rate of VOCs was seen with ADAKVEO® vs. placebo1*

 

Bar graph showing primary endpoint: 45% reduction (P=0.01) in the median annual rate of VOCs† was seen with ADAKVEO® vs. placebo.

Primary endpoint sub-group analysis

In the SUSTAIN study, sub-groups were analysed with the same method as that for the primary endpoint, reviewing the efficacy of ADAKVEO® in the following parameters:

VOC occurrence rate was assessed based on baseline VOC frequency groups:1,2

  • Patients with 2 to 4 VOCs in the previous 12 months experienced a 43% reduction in median annual rate of VOCs* with ADAKVEO® (n=42) vs. placebo (n=41) (1.14 vs. 2.00, P=0.031; HL=3.70; 1.08, 12.65)
  • Patients with 5 to 10 VOCs in the previous 12 months experienced a 63% reduction in median annual rate of VOCs* with ADAKVEO® (n=25) vs. placebo (n=24) (1.97 vs. 5.32; P=0.322; HL=3.14; 0.30, 32.47)

VOC occurrence rate was assessed in patients with or without concomitant HU/HC use:1

A 32% reduction in VOC frequency was observed in ADAKVEO® patients that were taking HU/HC (n=82, 62%) who still experienced VOCs in the last year

Two bar graphs showing the median annual rate of VOCs. A) Patients receiving concomitant HU/HC- 32% redcution. B) Patients not receiving HU/HC-50% reduction

 

It is also important to note, HU/HC is not appropriate for all patients with SCD and not indicated for HbSC, the second most common SCD genotype3

Primary endpoint post-hoc analysis

A post-hoc analysis was conducted with the same method as that for the primary endpoint to assess the number of patients who experienced no VOCs leading to a healthcare visit following 12 months of ADAKVEO® treatment vs. placebo

 

ADAKVEO® doubles patients’ chances of being VOC-free for 1 year vs. placebo1,4

A diagram of two circles showing the primary endpoint post-hos analysis. A) Orange circle with 36% for ADAKVEO n=24/67. B) Grey circle with 17% for placebo n=11/65.

More than one third (36%) of patients with SCD were VOC-free§ for a year with ADAKVEO® vs. 17% on placebo4

 

Image a magnifying glass next to on a red banner title: Access the SUSTAIN study.

 

Banner title: Reduction in VOCs was observed across all patient groups with ADAKVEO, regardless of baseline VOC frequency.

 

ADAKVEO® is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions after authorisation of ADAKVEO® is important to understand the safety profile of the treatment for patients with SCD.

Indication: ADAKVEO® (crizanlizumab) is indicated for the prevention of recurrent VOCs in patients with SCD who are aged 16 years and over. It can be given as an add-on therapy to HU/HC or as monotherapy in patients for whom HU/HC is inappropriate or inadequate.5

ADAKVEO® has a conditional marketing authorisation and further evidence is awaited.

*The SUSTAIN study assessed the efficacy and safety of ADAKVEO® in patients between the ages of 16–65 years at 69 sites in three countries. The SUSTAIN study was a Phase II, double-blind, randomised trial where patients received 5 mg/kg ADAKVEO® (n=67); 2.5 mg/kg ADAKVEO® (n=66); or placebo (n=65), administered intravenously at week 0, 2, 6 and 4 weekly intervals thereafter over a period of 52-weeks.1

Key inclusion criteria:1

  • 16 to 65 years of age
  • Confirmed medical history or diagnosis of SCD (including HbSS, HbSC, HbSβ+-thalassaemia, HbSβ0-thalassaemia or other genotypes)
  • With or without HU/HC
  • Experienced 2–10 VOCs in the preceding 12 months
  • Clinically acceptable medical history, physical examination, vital signs, clinical laboratory tests

VOCs were defined as a sickle cell acute painful episode in people that were hospitalised6

Leading to a healthcare visit1

§A patient was considered VOC-free if the annualised VOC rate was zero, whether or not the patient completed the 12–month treatment period1

Abbreviations: ARR, absolute risk ratio; HC, hydroxycarbamide; HL, Hodges Lehmann; HU, hydroxyurea; OR, odds ratio; SCD, sickle cell disease; VOC, vaso-occlusive crises.

References

  1. Ataga KI, et al. N Engl J Med. 2017;376(5):429–439.
  2. ADAKVEO® EPAR CHMP assessment report. 2020.
  3. Luchtman-Jones L, et al. Am J Hematol. 2016;91(2):238–242.
  4. Kutlar A, et al. Am J Hematol. [Suppl appendix]. 2019;94(1):55–61.
  5. ADAKVEO® Summary of Product Characteristics.
  6. National Institute for Health and Care Excellence. NICE Support for Commissioning for Sickle Cell Acute Painful Episode: 2014 Report. Available at: https://www.nice.org.uk/guidance/qs58/resources/support-for-commissionin.... Date accessed: April 2021
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UK | June 2021 | 104649
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ADAKVEO® has a conditional marketing authorisation and further evidence is awaited.

For more information, refer to the ADAKVEO® ▼ (crizanlizumab) prescribing information available here:  https://www.health.novartis.co.uk/sites/health.novartis.co.uk/files/adak...

Legal Category: POM.

Marketing Authorisation (MA) number, quantities and price: EU/1/20/1476/001  £1,038.00 per 10ml vial

 

Adverse events should be reported. Reporting forms and information can be found at www.mhra.gov.uk/yellowcard. Adverse events should also be reported to Novartis via [email protected] or online through the pharmacovigilance intake (PVI) tool at www.report.novartis.com
If you have a question about the product, please contact Medical Information on 01276 698370 or by email at [email protected]