Karyopharm Therapeutics and the Menarini Group announced that the European Commission (EC) has granted orphan medicinal product designation for selinexor for the treatment of myelofibrosis (MF).
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Selinexor was granted orphan drug designation in MF by the U. S. Food and Drug Administration (FDA) in May 2022. Karyopharm is currently evaluating selinexor, a first-in-class XP01 inhibitor, as monotherapy in patients with previously treated MF, and in combination with ruxolitinib in treatment-naïve patients.
In December 2021, Karyopharm and Menarini entered into an exclusive licensing agreement whereby Menarini is responsible for commercializing all current and future indications of NEXPOVIO in the European Economic Area, United Kingdom and Switzerland, CIS countries, Turkey and Latin America.
Stemline Therapeutics B.V., a wholly owned subsidiary of Menarini, is leading all commercialization activities in Europe.
"Myelofibrosis is a difficult-to-treat and complex disorder of the bone marrow with limited therapeutic options and we are committed to bringing novel treatments to patients through our collaboration with Karyopharm. We are excited about the potential to bring selinexor to myelofibrosis patients in Europe, pending positive study read-outs and regulatory approval," said Olivia del Puerto, MD LMS, Head of Medical Affairs Oncology - EMEA of Menarini.
MF is a rare type of bone marrow cancer that disrupts the body's normal production of blood cells. It causes extensive scarring of the bone marrow, leading to severe anemia that can cause weakness and fatigue. Bone marrow scarring can also cause a low number of platelets, which increases the risk of bleeding.
MF affects males and females in equal numbers and can occur at any age, although it usually affects individuals 50 years old or older.
According to the National Organization of Rare Diseases (NORD), the incidence is estimated to be 1.5 cases per 100,000 people in the United States and in Northern European countries, based on studies, the incidence is estimated to be 0.5 cases per 100,000 people.
NEXPOVIO, which is marketed as XPOVIO in the U.S., has been approved in the following oncology indications by the European Commission:
(i) in combination with dexamethasone for the treatment of multiple myeloma in adult patients who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, two immunomodulatory agents and an anti-CD38 monoclonal antibody, and who have demonstrated disease progression on the last therapy; and
(ii) in combination with bortezomib and dexamethasone for the treatment of adults with multiple myeloma who have received at least one prior therapy.
The marketing authorization of NEXPOVIO is valid in the EU Member States as well as Iceland, Liechtenstein, Norway, and Northern Ireland. NEXPOVIO has been commercially available in Germany since October 1, 2022.
NEXPOVIO is a first-in-class, oral exportin 1 (XPO1) inhibitor. NEXPOVIO functions by selectively binding to and inhibiting the nuclear export protein exportin 1 (XPO1, also called CRM1).
NEXPOVIO blocks the nuclear export of tumor suppressor, growth regulatory and anti-inflammatory proteins, leading to accumulation of these proteins in the nucleus and enhancing their anti-cancer activity in the cell.
The forced nuclear retention of these proteins can counteract a multitude of the oncogenic pathways that, unchecked, allow cancer cells with severe DNA damage to continue to grow and divide in an unrestrained fashion. ■