Novartis’ Hopes to Treat Sickle Cell Sufferers Dashed by EU Reversal

Novartis’ much-hyped sickle cell disease therapy Adakveo (crizanlizumab) has received a stunning blow, as the European Commission has officially withdrawn its conditional marketing authorization. The Swiss pharma company made the announcement on Friday, taking a hit to its hopes of providing a groundbreaking treatment for the disease.

The European Commission has issued a legally binding decision to withdraw Novartis’s drug Adakveo from all 27 territories within the European Union and the European Economic Area. This follows a recommendation from the Committee on Medicinal Products for Human Use (CHMP) to pull the approval after an extensive evaluation of additional Phase III data. Countries set to be impacted by this ruling are Germany, Sweden, Spain, Iceland, Norway and Liechtenstein.

At Novartis, we understand that individuals in these territories may be affected by our decision and thus would like to emphasize the importance of discussing alternative treatments with their doctors and healthcare providers. We encourage those in the affected areas to speak with their healthcare professionals to explore the best options for them.

Adakveo is an innovative humanized IgG2 monoclonal antibody that uses a highly specific method to combat sickle cell disease (SCD). By targeting the cell adhesion molecule P-selectin, which is present on the surface of platelets and endothelial cells, Adakveo prevents vaso-occlusion – the process that occurs in SCD which obstructs blood flow. In this way, Adakveo offers a much-needed solution to this debilitating and life-threatening condition.

In November 2019, the FDA officially approved Adakveo – a revolutionary new treatment designed to reduce the frequency of vaso-occlusive crises in adults 16 and over who were afflicted with sickle cell disease.

Nearly a year later, in October 2020, the European Medicines Agency issued a conditional approval to make the product available to a wider market – despite the need for further evidence to support its overall efficacy and safety. With this newfound development, hopeful sufferers of SCD eagerly anticipate a successful future with this extraordinary new solution.

In January this year, Novartis revealed that their Phase III STAND trial of Adakveo, hopeful of securing full approval within the EU, was unable to replicate prior efficacy results. An initial analysis of STAND revealed that Adakveo was disappointingly unable to demonstrate its superiority over placebo when it came to reducing the annualized rates of vaso-occlusive events in SCD patients.

The CHMP took action by launching a thorough review of Adakveo and in May 2023, verified that Novartis’ treatment was not successful in lessening the number of painful episodes requiring medical attention as opposed to a placebo.

The European regulator conducted a thorough review of past studies related to Adakveo, a managed access program, and real-world performance data. Although the studies had several limitations, such as the lack of a comparator, they couldn’t be used to demonstrate the effect of Adakveo and to counterbalance the STAND study’s negative results.

Novartis was pleased to observe that the findings of the STAND trial showed no new safety concerns associated with Adakveo, confirming the safety data established through prior research. Despite a lack of evidence to support its therapeutic benefit, the adverse event profile of Adakveo remains unchanged.

Despite STAND showing higher rates of severe and serious treatment-related side effects, the CHMP ultimately concluded that the benefits of Adakveo did not outweigh its risks.

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