EU Regulators Call for Withdrawal of Authorization for Novartis’ Sickle Cell Drug Adakveo Following Phase 3 Setback

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EU Regulators Call for Withdrawal of Authorization for Novartis' Sickle Cell Drug Adakveo Following Phase 3 Setback

After the phase 3 trial of Novartis’ sickle cell disease drug, Adakveo, yielded disappointing results, regulators in Europe have revisited their initial approval and are now suggesting that the authorization be withdrawn. The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) arrived at this recommendation by concluding that the benefits of Adakveo did not outweigh the associated risks. The decision was primarily based on the findings of the phase 3 STAND trial, which failed to demonstrate the superiority of Adakveo over a placebo.

β€œWhile we are disappointed by the recommendation to revoke the conditional MA for crizanlizumab in the European Union, we acknowledge that the STAND study did not meet one of the specific obligations of the conditional MA.” 


More specifically, Adakveo (crizanlizumab) did not effectively decrease the number of painful crises that necessitated healthcare visits. Patients who received Adakveo experienced an average of 2.5 painful crises requiring healthcare visits during the first year of treatment, while the placebo group had an average of 2.3. Furthermore, the Adakveo group had an average of 4.7 crises that required home healthcare visits or treatment, compared to 3.9 in the placebo group.

The CHMP also took into account data from other studies, including a managed access program and real-world data. However, these studies were deemed inconclusive due to various limitations, such as the absence of a comparator.

Initially, the European Medicines Agency granted marketing authorization to Adakveo in 2020 with the condition that the STAND study be conducted to address uncertainties arising from the limited available data. The final decision on revoking the authorization will be made by the European Commission, and it is anticipated to be issued within the next two months, potentially resulting in the withdrawal of the drug from the market.

Novartis acquired Adakveo through its acquisition of Selexys Pharmaceuticals for $665 million in 2016. The drug obtained FDA approval in 2019 based on results indicating a 45% reduction in the median rate of crises compared to a placebo.

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