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Eltanexor granted fast track designation by the FDA and orphan medicinal product designation by the EC

Aug 4, 2022
Learning objective: After reading this article, learners will be able to cite a new clinical development in MDS.

On July 20, 2022, eltanexor, a novel oral selective inhibitor of nuclear export, was granted fast track designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with relapsed/refractory intermediate-, high-, or very high-risk myelodysplastic syndromes (MDS).1 The European Commission (EC) also designated eltanexor as an orphan medicinal product for the treatment of MDS in the European Union.1

Eltanexor (KPT-8602) is a novel selective inhibitor of nuclear export compound that binds with, and inhibits, the nuclear export protein XPO1, resulting in the accumulation of tumor suppressor proteins in the cell nucleus. This is believed to lead to the selective induction of apoptosis in cancer cells, leaving normal cells largely unaffected. The FDA previously granted eltanexor orphan drug designation in January 2022.1

A phase I/II open-label study is currently ongoing investigating eltanexor in patients with relapsed/refractory MDS. Eltanexor has been previously covered by the MDS Hub.

  1. Karyopharm Therapeutics. Karyopharm granted regulatory designations for eltanexor for the treatment of myelodysplastic syndromes. Published Jul 20, 2022. Accessed Aug 2, 2022.

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