Myelodysplastic Syndromes: The FDA Grants Fast Track Designation for Eltanexor


Karyopharm Therapeutics, a pharmaceutical company focusing on novel cancer therapies, has issued a statement via PR Newswire that the FDA has granted regulatory designations to eltanexor, Karyopharm’s Selective Inhibitor of Nuclear Export (SINE).

Eltanexor is a novel SINE compound. It binds with XPO1 creating an accumulation of proteins that suppress tumors in the cell nucleus.

The company also received the FDA’s orphan drug designation in January 2022. Orphan drugs treat rare diseases that affect less than 200,000 people in the U.S. Eltanexor is under consideration as being a promising treatment for rare diseases.

In addition, the European Commission conferred the designation of Orphan Medicinal Product Designation on eltanexor to treat debilitation or life-threatening diseases. The definition of Orphan Drugs in the EU is fewer than 5 in 100,000 people across the European Union. Eltanexor is slated to provide a substantial benefit over and above existing treatments.

Eltanexor (KPT-8602) is being investigated in connection with treatment for patients diagnosed with various levels of refractory (not responding to treatment) and relapsed myelodysplastic syndromes (MDS). SINE molecules bind to and inhibit XPO1 thereby allowing tumor suppressors to accumulate and kill cancer cells.

About MDS

MDS is a group of diseases resulting from poor bone marrow function and accompanied by the risk of progressing to AML (acute myeloid leukemia.) Information about AML is available here.

About the Study

The most recent effort to investigate eltanexor is an open-label Phase 1/2 study of patients who have relapsed or are refractory to MDS drugs. Karyopharm had recently reported data resulting from its Phase 1 segment of the study. Eltanexor was evaluated in patients who had MDS that was refractory to hypomethylating agents (HMAs).

Currently, HMAs are considered to be the standard treatment for patients who are newly diagnosed and at higher risk for MDS. Yet only about forty to sixty percent of these patients respond to treatment. In HMAs, the response generally lasts two years or less.

A Look at 2022

Estimates for the number of new MDS cases in the year 2022 are approximately fifteen thousand people in the United States and fourteen thousand people living in the EU. Expectations are that the level of new diseases will be intermediate to high-risk MDS.

HMA refractory disease currently has no approved therapies. Patients with refractory HMA  are often told that they only have four to six months to live.

These statistics prompted Karyopharn’s CEO, Richard Paulson, to announce the company’s commitment to ongoing clinical trials and to deliver Eltanexor as a much-needed treatment option.

About the Fast Track Designation

The goal of Fast Track designations is to expedite the development and delivery of drugs to fill unmet needs so the drugs will reach patients earlier. After receiving the Fast Track designation, developers are encouraged to communicate with the FDA frequently.

The EC promotes the development of drugs that treat debilitation and life-threatening diseases through its Orphan Medicinal Product Designation. The drugs must be of significant benefit over and above existing treatment. The definition of a rare disease in the EU is a disease that affects fewer than 5 in 100,000 people.

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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