Novartis’ Hopes for SCD Treatment Dashed by Phase III Trial Results

Novartis’ hope for a therapeutic breakthrough in the treatment of sickle cell disease, Adakveo (crizanlizumab), is currently under review by the European Medicines Agency’s Committee for Medicinal Products for Human Use. Although promising results were seen in a Phase III trial, the drug failed to replicate its previous efficacy, leaving the future of this potential treatment uncertain.

Adakveo has been clinically studied to measure its effectiveness in decreasing the amount of vaso-occlusive crises (VOCs) that lead to healthcare visits for people living with sickle cell disease (SCD). The results of this research demonstrate promising outcomes for those with SCD.

Initial findings showed no significant difference between the placebo and two different doses of Adakveo in reducing the number of healthcare visits triggered by VOCs over the first year of the randomized study.

Adakveo, the first treatment for Sickle Cell Disease (SCD) to receive conditional marketing authorization from the European Medicines Agency (EMA), was granted the nod in October 2020. This will pave the way for the prevention of recurrent VOCs in SCD patients aged 16 and above. The decision was based on data from the Phase III STAND trial, which is a necessary condition for the authorization.

The EMA will thoroughly evaluate the trial results to determine the impact of long-term use of the therapeutic. Their review will be the key to uncovering if the therapeutic has a beneficial effect or not.

Novartis released a statement on Friday confirming that the trial results of Adakveo showed no new safety concerns, reassuring the public that its safety profile remains reliable. The company emphasizes that the same dose available on the market is safe and effective.

Adakveo, an innovative anti-P-selectin antibody, has been given a big thumbs-up by the FDA. Approved in November 2019, the drug is now available to reduce the frequency of VOCs in SCD patients aged 16 and above, offering a ray of hope to those affected by this debilitating condition.

Novartis’ Adakveo has been approved based on the results of the SUSTAIN clinical trial, which showed an impressive 45% reduction in the annual rate of VOCs. Even more impressive, more than one-third of patients treated with Adakveo didn’t experience a single VOC. This breakthrough therapy offers patients a powerful new option for managing VOCs.

Adakveo proved to be an effective treatment for patients, not only reducing the frequency of pain crises but also leading to a 42% reduction in hospitalization days over the course of a year compared to those treated with placebo.

Novartis announced on Friday that it will be collaborating with regulatory bodies, such as the FDA, to decide the best course of action moving forward. The company is currently conducting further analysis of the trial data to gain a comprehensive understanding of the results.

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