Karyopharm’s Eltanexor Receives the US FDA’s Orphan Drug Designation for the Treatment of Myelodysplastic Syndromes.
- The US FDA has granted ODD for eltanexor, a novel oral, SINE compound that inhibits XPO1 leading to the accumulation of tumor suppressor proteins in the cell nucleus to treat MDS
- The therapy is currently being evaluated in an ongoing P-I/II study as monothx. or in combination with approved & investigational agents in 119 patients with multiple types of hematologic & solid tumor cancers
- In the P-I study, the therapy showed 53% ORR, m-OS (9.9mos.) in patients with HMA-refractory MDS. In preclinical models, the therapy has a broad therapeutic window with minimal penetration of the blood-brain barrier & has the potential for another SINE compound to treat cancer
Ref: PR Newswire | Image: Karyopharm
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