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- Karyopharm Therapeutics (NASDAQ:KPTI) is trading ~2.9% higher in the pre-market after announcing that the FDA granted the orphan drug designation for its experimental oral medication, eltanexor, for the treatment of myelodysplastic syndromes (MDS).
- In addition to tax credits for clinical trial costs and waiver of the user fee for marketing applications, the developers of orphan drugs can claim seven years of marketing exclusivity upon regulatory approval for the treatment.
- An inhibitor of nuclear export protein, XPO1, Eltanexor is undergoing studies as monotherapy or in combination with other approved or investigational drugs in a Phase 1/2 trial involving patients with multiple types of hematologic and solid tumor cancers.
- "We are pleased to receive the FDA's orphan drug designation for eltanexor in MDS and believe it reinforces eltanexor's potential to improve clinical outcomes for patients with HMA-refractory MDS," CEO of Karyopharm (KPTI) Richard Paulson remarked.
Read more on data from a Phase 1/2 trial for eltanexor in patients with hypomethylating agent (HMA) refractory myelodysplastic syndrome (MDS).