The European Commission has officially withdrawn its approval of Novartis’ treatment for sickle cell disease, Adakveo (crizanlizumab). The Swiss pharmaceutical behemoth announced the government’s reversal Friday with dismay.
Novartis has been dealt a severe blow as the European Commission (EC) has withdrawn the approval of their medication, Adakveo, in all 27 European countries. Following a review of additional Phase III data by the Committee on Medicinal Products for Human Use (CHMP), the EC was forced to issue a legally binding decision, meaning the medication must be pulled from territories including Germany, Sweden, Spain as well as Iceland, Norway, and Liechtenstein.
Novartis has announced that patients in certain territories should consult with their doctors and healthcare providers to explore alternative treatments. This is a great opportunity to consider various options for improving health and overcoming medical issues.
Adakveo is an innovative humanized IgG2 monoclonal antibody that works to therapeutically intervene in sickle cell disease (SCD). Through its action, Adakveo works to inhibit the interaction between a cell adhesion molecule P-selectin – found on the surface of platelets and blood vessel endothelium – and its corresponding ligands, thus reducing and preventing the possibility of vaso-occlusion. This revolutionary new treatment for SCD offers hope for those living with the condition.
In November 2019, the FDA granted its approval to Adakveo, a therapeutic specifically designed to reduce painful vaso-occlusive crises experienced by sufferers of sickle cell disease aged 16 and older. Almost a year later, in October 2020, high hopes were placed in the product when the European Medicines Agency conditionally allowed its authorization, pending additional data advocating its efficacy and safety.
In January, Novartis announced an unexpected setback in the late-stage STAND trial that was looking to support full approval of Adakveo in the EU. Preliminary analysis revealed that, unfortunately, Adakveo was unable to demonstrate differentiating efficacy over placebo in terms of lowering the annualized rates of vaso-occlusive events in SCD patients. This came as a shock to many given the promising outcome of earlier trials.
In May 2023, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) launched its own review of Novartis’ Adakveo, ultimately confirming that the treatment did not significantly reduce the number of painful crises requiring a healthcare visit, in comparison to placebo.
The European regulator studied the efficacy of Adakveo, the managed access program, through its real-world performance and past studies. Unfortunately, it was found that these studies—lacking a comparator—could not be relied upon to show the effects of Adakveo and oppose the disconcerting findings of the STAND study.
Novartis affirmed that Adakveo’s safety profile continues to stay in line with that of prior studies, despite STAND failing to show significant therapeutic results. Nevertheless, no new safety concerns have been brought to light through the study.
Despite the promising results of the STAND study, the CHMP noted that Adakveo had a higher rate of severe and serious treatment-related side effects, and concluded that its benefits did not outweigh its risks.