Expanded Access Protocol Makes Imetelstat Available to Patients With MDS

The FDA granted an Expanded Access Protocol to imtelstat to treat certain patients with myelodysplastic syndrome.

Patients with lower risk myelodysplastic syndrome (MDS) — a type of cancer that starts in the bone marrow — may now have access to the experimental cancer drug, imetelstat, thanks to the Food and Drug Administration’s (FDA) Expanded Access Protocol, according to a press release from Geron, the pharmaceutical company manufacturing the drug.

The protocol, which is sometimes referred to as “compassionate use,” helps establish a pathway for patients with serious or life-threatening illnesses to get access to medical products that have not yet been approved. According to the FDA’s website, the program is for situations when there is “no comparable or satisfactory alterative therapy options” available.

"We have heard physicians in both academic and community settings express the need for new treatment options for their lower risk MDS patients, and they will now have an option to access imetelstat through the (Expanded Access Protocol)," Dr. Faye Feller, executive vice president and chief medical officer of Geron, said in the press release.

Thanks to the Expanded Access Protocol, imetelstat will now be available to patients with transfusion-dependent, non-del (5q) lower risk MDS who are refractory to (meaning their disease did not respond to), relapsed after, or ineligible for erythropoiesis-stimulating agents and naïve to lenalidomide and hypomethylating agents.

The FDA’s decision came after findings from the phase 3 IMerge clinical trial showed that after eight weeks of treatment, there was a higher percentage of patients who did not rely on transfusions in a group who received imetelstat compared to a group that received a placebo (inactive drug). The average duration of transfusion independence was nearly a year for the imetelstat group.

READ MORE: Imetelstat Hits Transfusion Independence Goal in Low-Risk Myelodysplastic Syndrome

Based on these findings, Geron announced that it submitted to the FDA New Drug Application, which, according to the agency is, “a type of application in which a pharmaceutical manufacturer or its agent requests permission from the (FDA) for a license to market a drug for one or more specific indications.”

“With positive IMerge Phase 3 results submitted to the FDA, we now plan to be working with clinicians, patients and the MDS patient advocacy community to provide access to imetelstat for eligible patients through our (Expanded Access Protocol) that was accepted by the FDA,” Feller said.

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