HealthFDA Panel Gives Thumbs Up to Imetelstat for Treating Anemia in MDS...

FDA Panel Gives Thumbs Up to Imetelstat for Treating Anemia in MDS Patients

Oncology/Hematology: FDA Panel Supports Imetelstat for Anemia Treatment

An FDA panel has endorsed investigational imetelstat as a viable treatment for transfusion-dependent anemia in adults with lower-risk myelodysplastic syndromes (MDS) who have not responded to erythropoiesis-stimulating agents (ESAs) or are not eligible for this standard treatment.

Despite initial concerns raised by the FDA staff before the recent meeting of the Oncologic Drugs Advisory Committee (ODAC), the committee members voted 12-2 in favor of imetelstat. They believed that the benefits of transfusion independence, as demonstrated in a phase III study of this telomerase inhibitor, outweighed any potential risks associated with the drug.

Dr. Neil Vasan, a panelist from Columbia University Medical Center in New York City, highlighted that the trial successfully met its primary endpoint and offered a new therapeutic option for patients who had exhausted other treatment options. The improvement in transfusion independence was deemed significant enough to justify the risks of cytopenias in this patient population.

The developer of imetelstat, Geron, is seeking traditional approval for the drug based on the results of the MDS3001 trial, which involved 178 heavily transfused patients who did not respond to ESAs. The study showed that the group treated with imetelstat achieved significantly higher rates of red blood cell transfusion independence for at least 8 weeks compared to the placebo group. Similarly, the 24-week transfusion independence rate was also higher in the imetelstat group.

Despite the higher occurrence of high-grade thrombocytopenia and neutropenia in the imetelstat-treated group, as well as increased use of myeloid growth factors, clinicians can monitor the risks and benefits of the drug and discontinue it if necessary. Dr. Jorge Nieva from the University of Southern California emphasized the importance of clinicians being able to make informed decisions based on the data provided.

Dr. Jacqueline Garcia from the Dana-Farber Cancer Center in Boston highlighted the long-term benefits of reducing reliance on blood transfusions for patients. She noted that many of the cytopenias observed in the imetelstat group were temporary and did not lead to serious infections, indicating the drug’s safety profile.

The ODAC Chair, Dr. Ravi Madan, emphasized the significance of this decision in providing a new treatment option for patients with MDS. This endorsement of imetelstat underscores the potential impact it could have on improving outcomes for individuals with transfusion-dependent anemia.

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