To Our Sickle Cell Community - On Oxbryta Being Withdrawn and What to do Next
Updated 10/4/24
To the sickle-cell disease community affected by the recent Oxbryta removal:
There’s a lot of uncertainty and so many unknowns right now. We recognize you already had limited therapies available, and the Oxbryta withdrawal means that one of those options was taken away. Our team at Folia has worked with dozens of SCD community members on a study that included lots of people taking Oxbryta, and we know that some people consider Oxbryta a core part of their SCD mitigation strategy.
So - this is frustrating and sudden, and completely out of your control. (Sidebar - does it actually make more sense for therapies like these, with serious adverse events identified, to get a warning and then leave the decisions to individuals and their doctors?) We’re here with you and we feel the frustration too.
A Pfizer representative shared in a town hall with the Sickle Cell Disease Association of America and the Sickle Cell Consortium that Pfizer is currently reviewing any and all data collected on Oxbryta to understand why emerging data differs from the original trial data, meaning they’re trying to understand why there is an increase in vaso-occlusive crises and fatalities. Because of this, they are not going to allow any compassionate use of Oxbryta for those who have seen benefits. They do plan to share what they learn, however, they do not know when that will happen because they are receiving new data daily.
You may still have some options available to you. Here we’ve broken down the top treatments reported by our community.
Because this is happening so quickly, we recommend making an appointment with your doctor ASAP, so you can talk through your options.
Below are the top sickle-cell treatments that our community has tracked on Folia, broken down by type:
Intended to prevent or reduce pain crises:
[Oxbryta]
Adakveo
Endari
Aspirin
Hydroxyurea
Intended to treat pain crises:
Oxycodone
Morphine sulfate
Ibuprofen
Methadone hydrochloride
Norco
Tylenol
Supplements:
Folic acid
Vitamin D3
We’ve recently learned that Pfizer is not offering any guidance around how to discontinue use with Oxbryta or ween down. According to a Pfizer representative, they do not have any efficacy for a dose or protocol for gradual discontinuation, so they recommend you speak with your doctor to advise what the best protocol will be for you. If you experience an adverse event in this process, you can report it to Pfizer at 1-800-438-1985. However, Dr. Sharl Azar, hematologist at Massachusetts General Hospital has said “patients shouldn’t stop taking Oxbryta suddenly, and instead must taper off.”
If you’re navigating this and have tips or advice for those trying to figure out what to do next, please comment and share. If you have questions for us or topics you’d like us to research, please comment below. There are currently lots of unknowns, but we will continue to share as we learn more.
With you in solidarity and working toward a future of SCD care that makes sense and is accessible to everyone,
The Folia team
