EMA’s human medicines committee (CHMP) has recommended revoking the marketing authorisation for Adakveo (crizanlizumab), a medicine for preventing painful crises (called vaso-occlusive crises) in patients aged 16 years and older with sickle cell disease.
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This follows a review by the CHMP, which concluded that the benefits of the medicine did not outweigh its risks.
The review looked at results of the STAND study, which compared the effectiveness and safety of Adakveo with placebo (a dummy treatment) in patients who had previously had painful crises leading to a healthcare visit.
The study showed that Adakveo did not reduce the number of painful crises leading to a healthcare visit.
Patients treated with Adakveo had on average 2.5 painful crises with a subsequent healthcare visit over the first year of treatment, compared with 2.3 crises in the placebo group.
In addition, the average number of crises requiring a healthcare visit or treatment at home was 4.7 with Adakveo compared with 3.9 with placebo.
In its review, the CHMP also looked at data from other studies, a managed access program and real‑world data.
However, the studies had several limitations, such as the lack of a comparator, and could not be used to show the effect of Adakveo or counterbalance the negative results of the STAND study.
In terms of safety, the STAND study did not raise new concerns but showed a higher rate of severe and serious treatment-related side effects for Adakveo compared with placebo.
The CHMP therefore concluded that its benefits do not outweigh the risks.
At the time of marketing authorisation, data showed that Adakveo was effective at reducing the number of painful crises in patients with sickle cell disease. However, the data were limited and there was some uncertainty about the size of the medicine’s effect.
EMA therefore requested the STAND study as a condition for the marketing authorisation of Adakveo, which was granted in October 2020.
As the STAND study results do not confirm the benefits previously seen with Adakveo, the CHMP has now concluded that the benefits do not outweigh the risks and recommended the revocation of its authorisation in the EU.
Following the CHMP’s recommendation, the European Commission will issue a legally binding decision.
Healthcare professionals should not start any new patients on Adakveo. For patients currently on treatment with Adakveo, healthcare professionals should explain to patients that the medicine is being taken off the market and the reasons why, and discuss alternatives with them. ■
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