Are there new drugs approved specifically for Myelodysplastic Syndromes anemia?

Anemia is a common and challenging symptom in myelodysplastic syndromes (MDS). In recent years, several new drugs have been approved specifically to treat anemia in lower-risk MDS, offering more options for patients who do not respond to standard therapies like erythropoiesis-stimulating agents (ESAs). These include lenalidomide for a specific genetic subtype, luspatercept, and imetelstat. Other promising agents are in clinical trials.

What the research says

Lenalidomide (Revlimid) is approved for transfusion-dependent anemia in adults with low- or intermediate-1-risk MDS who have a deletion 5q abnormality ¹. This was one of the first targeted therapies for MDS anemia. More recently, luspatercept, a TGF-beta ligand trap, was approved for anemia in lower-risk MDS, and it targets ineffective erythropoiesis ⁶. In 2024, the FDA approved imetelstat (Rytelo), a telomerase inhibitor, for transfusion-dependent anemia in low- to intermediate-1 risk MDS patients who require 4 or more red blood cell units over 8 weeks and have not responded to or are ineligible for ESAs ⁴⁶. Other drugs in development include elritercept, zilurgisertib, and DISC-0974, which target different pathways involved in MDS anemia ⁶. However, despite these advances, the overall number of new approvals for MDS remains limited, and allogeneic stem cell transplant is still the only potentially curative option ⁷⁸.

What to ask your doctor

• Could my MDS subtype (e.g., del5q, SF3B1 mutation) make me a candidate for lenalidomide or luspatercept?
• Am I eligible for imetelstat if I have transfusion-dependent anemia and have not responded to ESAs?
• Are there any clinical trials for newer drugs like elritercept or zilurgisertib that I might join?
• How do the side effects of these newer drugs compare to standard treatments for MDS anemia?
• Should I consider genetic testing to identify targets for these newer therapies?