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Karyopharm Granted Regulatory Designations for Eltanexor for the Treatment of Myelodysplastic Syndromes
– FDA Fast Track Designation and European Commission Orphan Medicinal Product Designation Underscore the Significant Need for New Treatment Options for MDS –
About this update from Karyopharm Therapeutics Inc.
[{"type":"text","content":"– FDA Fast Track Designation and European Commission Orphan Medicinal Product Designation Underscore the Significant Need for New Treatment Options for MDS –\nNEWTON, Mass., July 20, 2022 /PRNewswire/ -- Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, today announced new regulatory designations for eltanexor, a novel oral, Selective Inhibitor of Nuclear Export (SINE) investigational compound being studied for the treatment of myelodysplastic syndromes (MDS): (i) the U.S. Food and Drug Administration (FDA) has granted fast track designation for the development program of eltanexor as monotherapy for the treatment of patients with relapsed or refractory intermediate, high-, or very high-risk MDS; (ii) the European Commission (EC) adopted the Committee for Orphan Medicinal Products (COMP) opinion to designate eltanexor as an orphan medicinal product for the treatment of MDS in the European Union (EU). Karyopharm also received orphan drug designation from the FDA in January 2022. MDS are a group of diseases characterized by ineffective production of the components of the blood due to poor bone marrow function with a risk of progression to acute myeloid leukemia.\nKaryopharm is currently investigating eltanexor in an ongoing open-label Phase 1/2 study in patients with relapsed/refractory MDS. Previously, Karyopharm reported initial data from the Phase 1 portion of this study evaluating single-agent eltanexor in patients with hypomethylating agent (HMA)-refractory MDS.\nApproximately 15,000 people in the U.S.1 and 14,000 people in the EU2 are expected to be diagnosed with intermediate-to-high risk MDS in 2022. HMAs are the current standard of care for newly diagnosed, higher-risk MDS patients. However, only 40-60% of patients respond, with these responses typically lasting less than two years.3 The prognosis in HMA-refractory disease is poor, with a median overall survival of four to six months.4,5 There are currently no approved therapies for HMA- refractory MDS.\n\"These recent designations from the FDA and EC reinforce eltanexor's potential to improve clinical outcomes for patients with relapsed/refractory MDS,\" said Richard Paulson, President and Chief Executive Officer of Karyopharm. \"We are dedicated to advancing our ongoing clinical trials and remain co...