Announcement from ASH about Sickle Cell Disease

The American Society of Hematology just released their priorities for sickle cell research. First of all, the President of ASH acknowledged that, despite research and advancements, the current way sickle cell is treated and managed falls short of successful care.

Developed with a wide group of researchers and healthcare providers, seven research areas were identified as priorities in Sickle Cell Research.

1. Organ damage: to better understand the risk and progression to the essential organs of the body (eg, heart, kidneys, etc.)

2. Pain: to better understand what processes are behind the pain, to develop new treatments for pain

3. Therapies: how to best use the therapies available, such as hydroxyurea and blood transfusions, to discover the best medications and doses for individuals living with SCD

4. New drugs to treat SCD that can prevent organ damage

5. Greater investment in a cure, including different new approaches, such as partial transplants and other therapies

6. Guidelines: how useful they are and whether they’re being utilized in the real world

7. Sickle cell trait: including genetic counseling and family planning.

Source: firstwordpharma.com/ Am Soc Hematology

Research is ongoing in sickle cell disease, and you don’t have to search for it alone. Know Rare has a support team that can help guide you. Contact us here.

Pfizer pulls Sickle Cell Disease Drug Off the Market

Following a number of deaths in clinical studies for Oxbryta (voxelotor), which was approved as treatment for sickle cell disease in 35 countries, Pfizer decided to proactively withdraw the drug from being marketed. Pfizer took over the drug when it acquired Global Blood Therapeutics, and the drug works by preventing the clumping together of hemoglobin into the sickle shape and by breaking them down.  

Pfizer’s Chief Medical Officer was responsible for the decision, after a review of the results of two clinical trials testing the drug, one in children who were at higher risk of stroke, and the other in a study of the effect of the drug on leg ulcers, which happens to people with SCD. In the first study of 236 people over age 12, there were 8 deaths in the group taking the drug (vs 2 in those taking placebo), and in the second study, of 88 participants all taking the drug (but may also be taking something else) there were 8 who died.

Although the European Medical Association (EMA) said there was not enough evidence to assume the deaths were caused by the medication alone, Pfizer is voluntarily pulling it off the market, since the medical officer believes the medication’s benefit is not enough to justify the risk of death.

A drug is not approved by the FDA unless it has undergone multiple stages of testing, across many people. What’s interesting about Pfizer’s decision is that the serious effect occurred in a specific population in both cases, and it is not clear whether the same risks would apply to the general SCD population. However, it seems Pfizer believes that, when safety concerns arise, it may be better to take no chances.

This is a setback for people with SCD, since another treatment option has failed. 

If you or your loved one is diagnosed with sickle cell disease and would like to learn about the latest research opportunities, you can get in touch with the Know Rare support team here.