Imetelstat FDA Approval Status
FDA Approved: No
Generic name: imetelstat
Company: Geron Corporation
Treatment for: Myelodysplastic Syndrome
Imetelstat is a novel, first-in-class telomerase inhibitor in development for the treatment of transfusion-dependent anemia in adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) who have failed to respond or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESAs).
- Myelodysplastic syndromes are a group of disorders caused by the malfunctioning of stem cells in the bone marrow. Insufficient red blood cell production causes anemia that can require regular blood transfusions.
- Imetelstat works to treat transfusion-dependent anemia in MDS patients by binding to and inhibiting the activity of the enzyme telomerase to selectively kill the malignant stem and progenitor cells in the bone marrow.
- Imetelstat has been granted Fast Track designation by the U.S. Food and Drug Administration for both the treatment of adult patients with transfusion dependent anemia due to Low or Intermediate-1 risk MDS that is not associated with del(5q) who are refractory or resistant to an erythropoiesis stimulating agent, and for adult patients with Intermediate-2 or High-risk MF whose disease has relapsed after or is refractory to janus associated kinase (JAK) inhibitor treatment.
- Geron submitted a New Drug Application (NDA) in the U.S. in June 2023.
Development timeline for imetelstat
Further information
Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.
