Pharma Focus Europe

Karyopharm and Menarini Group Announce Orphan Medicinal Product Designation from the European Commission for Selinexor for the Treatment of Myelofibrosis

Tuesday, November 01, 2022

Karyopharm Therapeutics Inc., a pharmaceutical company specializing in innovative cancer therapies, and the Menarini Group, a leading international pharmaceutical company, announced today that the European Commission (EC) has granted orphan medicinal product designation to selinexor for the treatment of myelofibrosis (MF). Selinexor previously received orphan drug designation from the U.S. Food and Drug Administration (FDA) for MF in May 2022. Karyopharm is currently evaluating selinexor, a first-in-class XP01 inhibitor, as a monotherapy in previously treated MF patients and in combination with ruxolitinib in treatment-naïve patients. Under an exclusive licensing agreement signed in December 2021, Menarini is responsible for commercializing NEXPOVIO® in the European Economic Area, United Kingdom, Switzerland, CIS countries, Turkey, and Latin America. Commercialization activities in Europe are led by Stemline Therapeutics B.V., a wholly owned subsidiary of Menarini.

"We are delighted to receive orphan medicinal product designation from the EC for selinexor in the treatment of myelofibrosis," said Dr. Reshma Rangwala, Chief Medical Officer of Karyopharm. "This designation, combined with the FDA's orphan drug designation, underscores the significant unmet need for a novel mechanism of action like selinexor for this devastating disease. We remain on track with our clinical plans and look forward to advancing selinexor's development in MF."

"Myelofibrosis is a complex and challenging bone marrow disorder with limited treatment options, and we are dedicated to providing new therapies to patients through our collaboration with Karyopharm. Pending positive study results and regulatory approval, we are excited about the potential to bring selinexor to myelofibrosis patients in Europe," said Dr. Olivia del Puerto, Head of Medical Affairs Oncology - EMEA at Menarini.

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