On January 8, 2019 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, reported that it has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for selinexor, the Company’s first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound, requesting conditional approval for the treatment of patients with relapsed or refractory multiple myeloma (MM) who have received at least three prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor (PI), one immunomodulatory agent (IMiD), and one anti-CD38 monoclonal antibody (mAb), and to their most recent treatment regimen (penta-refractory MM) (Press release, Karyopharm, JAN 8, 2019, View Source [SID1234532591]). Karyopharm also announced that the selinexor MAA has been granted accelerated assessment by the EMA’s Committee for Medicinal Products for Human Use (CHMP).
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"The MAA submission for selinexor is an important milestone for Karyopharm and the CHMP’s granting of accelerated assessment further underscores the urgent need to improve outcomes for patients with highly refractory multiple myeloma," said Sharon Shacham, PhD, MBA, Founder, President and Chief Scientific Officer of Karyopharm. "The results from the pivotal Phase 2b STORM study provide compelling evidence that selinexor in combination with low-dose dexamethasone has the potential to be an effective new treatment option for patients with this difficult to treat disease. With the filing of the MAA and an accelerated assessment designation from the EMA, we hope to make oral selinexor available as quickly as possible to patients throughout Europe."
An accelerated assessment is granted to products deemed by the CHMP to be of major interest for public health and represent therapeutic innovation. Accelerated assessments can reduce the active review time of an MAA from the standard 210 days down to 150 days once it has been validated by the EMA. Selinexor has also previously received orphan designation in multiple myeloma from the EMA.
A New Drug Application (NDA) seeking accelerated approval for oral selinexor with low dose dexamethasone as a treatment for patients with penta-refractory multiple myeloma is under Priority Review by the U.S. Food and Drug Administration (FDA) with an action date of April 6, 2019, under the Prescription Drug User-Fee Act (PDUFA).
About Selinexor
Selinexor is a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound. Selinexor functions by binding with and inhibiting the nuclear export protein XPO1 (also called CRM1), leading to the accumulation of tumor suppressor proteins in the cell nucleus. This reinitiates and amplifies their tumor suppressor function and is believed to lead to the selective induction of apoptosis in cancer cells, while largely sparing normal cells. In 2018, Karyopharm reported positive data from the Phase 2b STORM study evaluating selinexor in combination with low-dose dexamethasone in patients with penta-refractory multiple myeloma. Selinexor has been granted Orphan Drug Designation in multiple myeloma and Fast Track designation for the patient population evaluated in the STORM study. Karyopharm’s New Drug Application (NDA) has been accepted for filing and granted Priority Review by the FDA, and oral selinexor is currently under review by the FDA as a possible new treatment for patients with penta-refractory multiple myeloma. The Company has also submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) with a request for conditional approval and was granted accelerated assessment. Selinexor is also being evaluated in several other mid-and later-phase clinical trials across multiple cancer indications, including in multiple myeloma in a pivotal, randomized Phase 3 study in combination with Velcade (bortezomib) and low-dose dexamethasone (BOSTON), as a potential backbone therapy in combination with approved therapies (STOMP), in diffuse large B-cell lymphoma (SADAL), liposarcoma (SEAL), and an investigator-sponsored study in endometrial cancer (SIENDO), among others. Additional Phase 1, Phase 2 and Phase 3 studies are ongoing or currently planned, including multiple studies in combination with approved therapies in a variety of tumor types to further inform Karyopharm’s clinical development priorities for selinexor. Additional clinical trial information for selinexor is available at www.clinicaltrials.gov.