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Karyopharm Announces Additional Orphan Designations Granted for Selinexor (KPT-330) by European Commission

1.7 from 3 votes
Tuesday, December 02, 2014

Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, announced that its lead drug candidate, Selinexor (KPT-330), has received orphan drug designation from the European Medicines Agency (EMA) of the European Commission for the treatment of chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL), including Richter's Transformation, and for the treatment of multiple myeloma.

Selinexor has previously received orphan designation in acute myeloid leukemia (AML) and diffuse large B-cell lymphoma (DLBLC) from both the EMA and the U.S. Food and Drug Administration (FDA). Orphan designation was created to encourage the development of drugs which may provide significant benefit to patients suffering from rare diseases.

"The granting of orphan designation by the EMA for these hematologic malignancies represents a further significant achievement for our Selinexor development program," commented Dr. Sharon Shacham, President and Chief Scientific Officer of Karyopharm. "There continues to be limited treatment options for patients with these serious blood cancers, particularly for those patients who fail to achieve and maintain responses on available therapies. We are excited about the prospects for oral Selinexor's novel mechanism of action to potentially treat these challenging conditions, either alone or in combination with other therapies."

Orphan designation by the EMA is granted to promote the development of drugs that target rare life-threatening or debilitating conditions and that are expected to provide significant therapeutic advantage over existing treatments. Orphan designation qualifies a company for benefits that include targeted scientific advice from the EMA regarding drug development and ten years of market exclusivity following marketing approval.

About Selinexor

Selinexor (KPT-330) is a first-in-class, oral Selective Inhibitor of Nuclear Export / SINE™ compound. Selinexor functions by inhibiting the nuclear export protein XPO1 (also called CRM1), leading to the accumulation of tumor suppressor proteins in the cell nucleus, which subsequently reinitiates and amplifies their tumor suppressor function. This is believed to lead to the selective induction of apoptosis in cancer cells, while largely sparing normal cells. Over 500 patients have been treated with Selinexor in Phase 1 and Phase 2 clinical trials in advanced hematologic malignancies and solid tumors. Karyopharm has initiated two registration-directed clinical trials of Selinexor, one in older patients with acute myeloid leukemia and the other in patients with Richter's Transformation. Additional registration-directed clinical trials in hematological indications, including one in patients with diffuse large B-cell lymphoma (DLBCL) which is expected to begin in Q4 2014, are planned. At least one additional registration-directed clinical trial in a hematologic or solid tumor indication is also planned for the first half of 2015. Additional Phase 1 and Phase 2 studies are ongoing or currently planned, including multiple investigator-sponsored studies of Selinexor in combination with one or more approved therapies. The latest clinical trial information for Selinexor is available at

About Karyopharm Therapeutics

Karyopharm Therapeutics Inc. (Nasdaq:KPTI) is a clinical-stage pharmaceutical company focused on the discovery and development of novel drugs directed against nuclear transport targets for the treatment of cancer and other major diseases. Karyopharm's SINE™ compounds function by binding with and inhibiting the nuclear export protein XPO1 (or CRM1). SINE™ compounds have also shown biological activity in models of inflammation, autoimmune disease, certain viruses, and wound-healing. Karyopharm was founded by Dr. Sharon Shacham and is located in Newton, Massachusetts. For more information, please visit



Justin Renz

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Gina Nugent

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Source: Karyopharm Therapeutics Inc.
1.7 from 3 votes
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