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news.US-based pharmaceutical firm Karyopharm Therapeutics has received orphan drug designation from the US Food and Drug Administration (FDA) for its lead drug candidate, selinexor (KPT-330) to treat multiple myeloma.
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Selinexor is a first-in-class, oral Selective Inhibitor of Nuclear Export / SINE compound. It has also secured orphan designation in multiple myeloma from the European Medicines Agency (EMA).
Both the FDA and the EMA have granted orphan designation for selinexor in acute myeloid leukemia (AML) and diffuse large B-cell lymphoma (DLBCL).
Additionally, the EMA has granted orphan designation of selinexor to treat chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL), including Richter’s Transformation.
Karyopharm president and chief scientific officer Dr Sharon Shacham said: "Orphan Drug Designation by the FDA for multiple myeloma is another significant milestone in the selinexor development program.
"We are encouraged by the response and durability data demonstrated to date in patients with multiple myeloma who have received selinexor in Phase I and Phase I/II studies and look forward to the commencement of additional trials with selinexor in patients with multiple myeloma in the near term, including a potential registration-directed trial we expect to commence during the first half of 2015."