EUROPE — A regulatory upheaval has led pharmaceutical giant Novartis to withdraw its sickle cell disease drug, Adakveo, from the European market.

This decision comes in the wake of a recommendation made by a committee associated with the European Medicines Agency in May, which advised against the continued conditional clearance of Adakveo.

This unexpected turn raises concerns for patients and prompts a quest for alternative treatment options.

The crux of this regulatory shift lies in the results of a late-stage clinical trial that compared the drug’s efficacy to a placebo.

Surprisingly, the trial revealed no significant difference in reducing the frequency of pain crises—a common experience for those with sickle cell.

Novartis has urged patients currently using Adakveo to engage in discussions with their physicians about exploring alternative treatment avenues.

While the company did not provide a timeline for the withdrawal process, it emphasized that no fresh safety concerns have emerged regarding the medicine.

The European Commission’s verdict carries far-reaching consequences, encompassing not only all 27 European Union countries but also Iceland, Norway, Liechtenstein, and Northern Ireland (until January 2025).

Adakveo, an antibody drug, was endorsed in Europe in late 2020 to counter vaso-occlusive crises—a hallmark of severe sickle cell.

The treatment functions by binding to P-selectin, a substance found on blood vessel cells, in a bid to thwart the blockage of blood vessels during pain crises.

Global impact and company response

On a global scale, Adakveo has reached around 11,800 individuals grappling with sickle cell, as reported by Novartis.

In the first half of the current year, the company posted slightly over US$100 million in sales for the drug.

In the United States, Adakveo entered the market alongside a drug from Global Blood Therapeutics (now under Pfizer’s ownership).

However, these treatments have seen limited utilization due to their high cost and their inability to provide a definitive cure for sickle cell—a condition driven by the distortion of red blood cells into crescents that obstruct blood vessels.

The setbacks faced by Adakveo and similar treatments coincide with the emergence of powerful genetic therapies for sickle cell disease.

In the near future, two gene-corrective treatments with the potential for curative impact might secure approval from the U.S. Food and Drug Administration.

The medications — one is being developed by Bluebird Bio and the other by a partnership between Vertex Pharmaceuticals and CRISPR Therapeutics — have not yet been approved by the Food and Drug Administration.

As a result, pricing has not been disclosed, but analyst estimates have suggested the treatments may be priced at roughly US$2 million per year.

The significance of these pioneering therapies extends beyond their transformative potential to cure. A deeper dive into the data reveals the economic implications of traditional care methods.

In 2016 alone, the U.S. witnessed a staggering 134,000 hospital stays that collectively cost a substantial US$811.4 million, according to data sourced from the Agency for Healthcare Research and Quality.

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