Clinical Trial Updates (SCD)

Adakveo (crizanlizumab)

Update: 30 September 2022

No update available.


Update: 30 June 2022

No update available.


Update: 25 April 2022

Adakveo (crizanlizumab) is now available on the U.K.’s National Health Service (NHS) for sickle cell disease (SCD) patients 16 and older.

The new treatment, which is delivered by an into-the-vein infusion, can be used alone or as an add-on to hydroxyurea to help reduce the frequency of painful episodes caused by blood vessel obstruction called vaso-occlusive crises (VOCs).

Adakveo is approved under a conditional marketing authorization that requires the company to provide more evidence about its safety and effectiveness. This means that to get access to the NHS-funded treatment, available as of Feb. 1, a managed access agreement was put in place to enable researchers to collect data from patients and determine whether they are benefiting from treatment.



Update: 10 January 2022

No update available.


Update: 27 October 2021

The National Institute for Health and Care Excellence (NICE) has published the Final Appraisal Determination (FAD) recommending Adakveo as an option for preventing recurrent sickle cell crises in people aged 16 or older with sickle cell disease.

Adakveo will be available through a Managed Access Agreement (MAA) to patients aged 16 or above. NICE expects more than 300 people to get Adakveo via the program, growing to more than 450 people later.
The drug showed in a phase 3 trial that it could significantly reduce the annual rate of sickle cell-related pain crisis over placebo. But NICE’s drug appraisal committee pointed to the study’s small sample size and duration of a little over a year, arguing that the long-term effectiveness of Adakveo is unproven.

To resolve the committee’s concerns, the marketing authorisation holder (Novartis) has proposed to initiate a phase 3 U.K.-specific trial called Stand that will study Adakveo, with or without traditional hydroxyurea therapy, in patients aged 12 years and over with a history of vaso-occlusive crisis. The trial is expected to report a primary analysis in 2023 and will provide data for up to three years. NICE has agreed that it will revisit its guidance when the new evidence becomes available.



Update: 30 June 2021

No update available.


Update: 08 January 2021

Data presented at ASH2020 confirm that no new safety signals were identified in the enrolled SCD population treated with crizanlizumab, with no apparent differences between the 5.0 and 7.5 mg/kg doses in the frequency and severity of AEs. For both doses, serum crizanlizumab concentrations rose to a near maximum level shortly after infusion. Consistent with results from the SUSTAIN trial, crizanlizumab reduced from baseline the annualized rate of VOCs leading to a healthcare visit.



Update: 05 November 2020

The European Commission has approved Adakveo (crizanlizumab) as a preventive treatment for recurrent vaso-occlusive crises (VOCs) in people, 16 and older, with sickle cell disease (SCD).

The approval covers the use of Adakveo in combination with hydroxyurea (also known as hydroxycarbamide) and as a stand-alone therapy in patients for whom hydroxyurea treatment is inappropriate.



Update: 25 August 2020

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending conditional marketing authorization of Adakveo® (Crizanlizumab) for the prevention of recurrent vaso-occlusive crises (VOCs), or pain crises, in patients with sickle cell disease aged 16 years and older.

Adakveo can be given as an add-on therapy to hydroxyurea/hydroxycarbamide (HU/HC) or as monotherapy in patients for whom HU/HC is inappropriate or inadequate.



Update: 31 May 2020

  • Continued access within the US.
  • EMA approval expected within the second half of 2020.



Update: 31 March 2020

No update available.


Update: 31 January 2020

In a draft report, the Institute for Clinical and Economic Review (a cost watchdog) concluded that the newly authorised sickle cell disease drugs in the US (from GBT, Novartis and Emmaus Medical) are too expensive to meet traditional cost-effectiveness measures. To fall within one measure of cost-effectiveness—below $150,000 per quality-adjusted life year—the companies would have to dramatically cut their prices.

For GBT’s Oxbryta, the cut would have to be almost 90% – a cost of $9,218 per year would be more appropriate —down from an estimated list price of $84,000 per year. And for Novartis’ Adakveo, ICER’s calculations suggested an annual cost of $25,410, less than one-third of its existing cost of $88,000 per year, as estimated by ICER.



Update: 25 November 2019

  • US Food and Drug Administration (FDA) approved Adakveo® (Crizanlizumab), previously known as SEG101, a targeted biologic, to reduce the frequency of vaso-occlusive crises (VOCs), or pain crises, in adult and pediatric patients aged 16 years and older with sickle cell disease.
  • 45% reduction in VOCs regardless of SCD genotype and/or hydroxyurea use.
  • Fewer hospitalisation days per year (4 vs 6.87)
  • SUSTAIN is a randomized, multicenter, placebo-controlled, double-blind study. A total of 198 patients with any genotype of sickle cell disease (HbSS, HbSC, HbS/beta0-thalassaemia, HbS/beta+-thalassaemia, and others) and a history of 2-10 VOCs in the previous 12 months were eligible for inclusion.


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