Karyopharm Announces Outcome of FDA Advisory Committee Meeting Reviewing Selinexor for the Treatment of Patients with Triple Class Refractory Multiple Myeloma
The ODAC voted 8 to 5 recommending that the
“While we are disappointed with ODAC’s recommendation to delay the potential approval of selinexor, we plan to work with the
The ODAC is an independent panel of experts that evaluates data concerning the efficacy and safety of marketed and investigational products for use in the treatment of cancer and makes appropriate recommendations to the FDA. Although the
Karyopharm’s NDA seeking accelerated approval for oral selinexor in combination with dexamethasone as a treatment for patients with triple class refractory multiple myeloma who have received at least three prior therapies is under Priority Review by the
The selinexor NDA is supported by data from Karyopharm’s Phase 2b STORM (Selinexor Treatment of Refractory Myeloma) study, which evaluated selinexor and low-dose dexamethasone in 122 heavily pretreated patients with triple class refractory multiple myeloma who have been previously exposed to all five of the most commonly prescribed anti-myeloma therapies currently available. The study met its primary objective with an overall response rate (ORR) of 26.2%, which included two stringent complete responses (sCRs), six very good partial responses (VGPRs) and 24 partial responses (PRs). The most common adverse events included thrombocytopenia, nausea/vomiting, fatigue and decreased appetite. Adverse events were generally predictable and manageable with dose adjustments and treatment-emergent AEs leading to treatment discontinuation occurred in 26.8% of patients, and these were considered by the Investigator to be treatment-related in 17.9% of patients. Major organ toxicities were not prominent in this study and safety results were consistent with those previously reported from Part 1 of this STORM study (Vogl et al., J Clin Oncol, 2018) and from other selinexor studies.
The Company has also submitted a Marketing Authorization Application (MAA) to the
Karyopharm will discuss ODAC’s recommendation during the Company’s previously-announced conference call on
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 triple class refractory multiple myeloma who have been previously exposed to all five of the most commonly prescribed anti-myeloma therapies currently available. 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
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding our expectations relating to submissions to, and the review and potential approval of selinexor by, regulatory authorities, including the anticipated timing of such submissions and actions, and the potential availability of accelerated approval pathways, the therapeutic potential of and potential clinical development plans for Karyopharm's drug candidates, especially selinexor, and the plans for commercialization. Such statements are subject to numerous important factors, risks and uncertainties, many of which are beyond Karyopharm’s control, that may cause actual events or results to differ materially from Karyopharm's current expectations. For example, there can be no guarantee that regulators will agree that selinexor qualifies for accelerated approval in the U.S. or conditional approval in the E.U. as a result of the data from the STORM study in patients with triple class refractory myeloma or the SADAL study in patients with relapsed or refractory DLBCL or that any of Karyopharm's drug candidates, including selinexor, will successfully complete necessary clinical development phases or that development of any of Karyopharm's drug candidates will continue. Further, there can be no guarantee that any positive developments in Karyopharm's drug candidate portfolio will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other factors, including the following: Karyopharm's results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the
Velcade® is a registered trademark of
Vice President, Investor and Public Relations
857-297-2241 | firstname.lastname@example.org
212-600-1902 | email@example.com
Source: Karyopharm Therapeutics Inc.