Anemia Treatment for Lower-Risk MDS Receives FDA Orphan Drug Designation

If you are a person with lower-risk myelodysplastic syndromes (MDS), learn how you may be able to receive a new anemia treatment, called tebapivat, through a clinical trial. 

What is Tebapivat for MDS? 

Tebapivat (by Agios) is a new medicine taken by mouth that helps improve anemia for people with lower-risk MDS, a condition in which red blood cell counts are low.  

MDS causes to anemia because stem cells, which can become different types of blood cells, struggle to develop into specialized types. Tebapivat helps support stem cells during this process, boosting the production of healthy red blood cells.

The FDA granted tebapivat orphan drug designation in September 2024. This means that tebapivat will receive the support it needs to move more readily through the clinical trial process and hopefully toward its approval.  

Anemia treatments for lower-risk MDS patients are in demand to help alleviate fatigue, consistent dependence on red blood cell transfusions, and other quality-of-life concerns caused by the condition.

How Effective is Tebapivat, and Where Can I Receive It? 

Early data from tebapivat’s clinical trial showed that several lower-risk MDS patients with anemia could stop red blood cell transfusions and saw an increase in low hemoglobin levels (the protein in red blood cells that carries oxygen). 

If you have lower-risk MDS, click the button below to review your eligibility for tebapivat as an anemia treatment. 

Explore Treatment with Tebapivat

If you want to learn how clinical trials generally work, click here. 

Conclusion 

In summary, tebapivat is a new anemia treatment option in a clinical trial for people with lower-risk MDS. It works to overcome obstacles in healthy blood cell production caused by MDS by assisting stem cell development into specialized types like red blood cells. 

Early data has shown that after taking tebapivat, some patients are able to stop blood cell transfusions. Click on the clinical trial link above if you are interested in reviewing your eligibility to receive this treatment. 

Track your MDS Labs 

Are you interested in keeping track of your MDS labs, such as blood counts, in one place? Click on the link below to access your free account! 

Track My MDS Labs

Sources: 

• Agios Announces FDA Orphan Drug Designation Granted to Tebapivat (AG-946) for Treatment of Myelodysplastic Syndromes (MDS)

• FDA Grants Orphan Drug Designation to Tebapivat for MDS

• A Study of Tebapivat (AG-946) in Participants With Anemia Due to Lower-Risk Myelodysplastic Syndromes (LR-MDS)