Imetelstat for Treating Anemia in Lower-Risk MDS Patients

On March 14th, 2024, the FDA’s Oncologic Drugs Advisory Committee (ODAC) met to discuss a New Drug Application to treat patients with lower-risk myelodysplastic syndromes (MDS). The ODAC is an independent group of advisors that review and discuss new results available and share their recommendations with the FDA before they announce a decision. However, the recommendation does not determine the final FDA’s decision.

What Is Imetelstat?

Imetelstat (Geron) is the first of a new type of therapy called telomerase inhibitors being tested to treat patients with low- to intermediate-risk MDS who have lost response to or are ineligible for treatment with erythropoietin-stimulating agents.

Can Imetelstat Help Patients with Low-Risk MDS?

The IMerge trial enrolled transfusion-dependent patients with low-risk MDS who could not be treated with erythropoiesis-stimulating agents (ESAs, like Epogen) or had lost response to it. This study showed that after being treated with imetelstat, 39.8% of those patients became transfusion-independent for at least 8 consecutive weeks, and 28% did not need any transfusion for 6 months. However, more than 60% of patients treated with imetelstat also experienced a reduction of platelets and an increased risk of infections, which was resolved in most cases.
 
For more information about imetelstat and the IMerge clinical trial results, see our previous article, ‘The Promising Treatment Of Imetelstat And Transfusion Independence’.

What Did the FDA’s Expert Committee Say About Imetelstat? 

The panel reviewed and discussed all the data available on imetelstat and concluded that the benefits of imetelstat are greater than its risks for the treatment of transfusion-dependant anemia in adult patients with low- to intermediate-1 risk MDS who cannot be treated anymore with erythropoiesis-stimulating agents. 

The Committee was asked to review and discuss the available data around the following key points:

1. The balance between benefits and risks of imetelstat treatment, taking into account the number of responses observed, their duration, and imetelstat’s side effects.
2. The impact of constant transfusions on a patient's life and the role of imetelstat in treating both the symptoms and the underlying disease.
3. The impact of imetelstat's treatment on patient-reported outcomes.
4. The representation of US patients in imetelstat's clinical trials.

With 12 votes in favor and 2 votes against, the ODAC recommended the FDA approve imetelstat for the treatment of MDS patients.

When Will We Know if Imetelstat is Approved for MDS Treatment?

The maker of imetelstat, Geron, announced that it expects a final decision from the FDA in June 2024 unless they ask for more data. In addition, imetelstat is also being reviewed for commercialization in Europe.

To learn more about MDS risk classification and treatment, visit HealthTree University for MDS, where you can find out if new treatments like luspatercept or imetelstat could help you. 

Learn About MDS Treatments Here

Sources:

• March 14, 2024: Meeting of the Oncologic Drugs Advisory Committee Meeting Announcement
• Geron Announces FDA Oncologic Drugs Advisory Committee Votes in Favor of the Clinical Benefit/Risk Profile of Imetelstat for the Treatment of Transfusion-Dependent Anemia in Patients with Lower-Risk MDS