Oxbryta’s withdrawal is a setback in the fight against sickle cell disease

The therapy was withdrawn from global markets due to safety concerns

Dunstan Nicol-Wilson avatar

by Dunstan Nicol-Wilson |

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Pfizer’s recent decision to withdraw Oxbryta (voxelotor) from global markets feels like a devastating blow for many people with sickle cell disease.

Oxbryta’s conditional approval in 2019 by the U.S. Food and Drug Administration gave hope to many, as few treatments exist for patients with sickle cell disease. The therapy was approved in the European Union in 2022.

As a sickle cell advocate who’s committed to raising awareness, I believe that improving health outcomes and access to treatment is what the fight is all about. This loss of a treatment option is a setback for our community.

For the many patients and families who rely on this medication to manage their condition, the uncertainty about what comes next must be overwhelming. Sickle cell disease is already an unpredictable and painful condition to live with; now, patients who found some relief from Oxbryta must work with their care provider to figure out next steps.

I’ve seen firsthand how important access to effective treatments is — for physical health as well as mental and emotional well-being. I was not eligible for Oxbryta, but I had hoped that as more treatments became available, I would be suitable for one of them. Many people living with sickle cell disease already experience anxiety about their health and future, which can be mentally exhausting.

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A step backward

One of the most pressing concerns in the sickle cell community is whether the wider population, researchers, investors, and healthcare professionals really care about us. Sickle cell primarily affects people of African descent, and historically, this population has faced significant disparities in healthcare access and outcomes. The development and approval of Oxbryta was a win for our community, but its withdrawal feels like a step backward in our efforts to achieve equity.

After reading the reasons behind Pfizer’s decision, I agree that if the therapy’s benefits don’t outweigh its risks, withdrawing it from the market is the right decision. However, this raises questions about the clinical trials and safety testing conducted before the drug was approved. Why weren’t these safety issues raised sooner?

The relationship between Black people and clinical trials is already very fragile. This kind of news is likely to further fracture relationships between the Black community and research and development, as our marginalized population has once again been failed.

Despite this setback, I remain hopeful and determined to focus on the big picture. We should use the data and lessons learned from Oxbryta to develop better and safer therapies for people with sickle cell. There needs to be more research, more treatment options, and better access to care for our neglected patient population.

I urge the sickle cell community, healthcare professionals, and policymakers to come together and demand more viable alternatives. The narrative cannot be that approved treatments are failing sickle cell patients. We need action now.


Note: Sickle Cell Disease News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Sickle Cell Disease News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to sickle cell disease.

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