The US pharmaceutical company, Karyopharm Therapeutics Inc., has announced that the FDA has granted orphan drug designation for its lead drug candidate, selinexor (KPT-330), as a treatment for myeloma. This follows similar orphan drug designation granted recently by the European Medicines Agency (EMA). Selinexor is a first-in-class oral selective inhibitor of nuclear export which acts by binding to and inhibiting the nuclear export protein XPO1 (also known as CRM1), leading to the accumulation of tumour suppressor proteins in the cell nucleus and amplification of their tumour suppressor function. The new orphan drug status will further support the clinical development of Selinexor for myeloma.

 

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