Tuesday, July 18, 2023
Karyopharm Therapeutics Inc., a leading pharmaceutical company specializing in innovative cancer treatments, announced today that the United States Food and Drug Administration (FDA) has granted Fast Track Designation to selinexor for the treatment of myelofibrosis patients. This includes individuals with primary myelofibrosis, post-essential thrombocythemia myelofibrosis, and post-polycythemia vera myelofibrosis.
The Fast Track Designation signifies the potential of selinexor to address the critical medical needs in myelofibrosis, and it marks a significant milestone as Karyopharm progresses its Phase 3 study. Dr. Reshma Rangwala, Chief Medical Officer of Karyopharm, expressed optimism about selinexor's unique mechanism of action, which involves XPO1 inhibition—a novel and potentially fundamental approach in myelofibrosis treatment. Encouraging efficacy and safety data from the Phase 1 study, which evaluated the combination of selinexor and ruxolitinib in treatment-naïve myelofibrosis patients, have fueled confidence in selinexor's potential to revolutionize treatment strategies. Dr. Rangwala also expressed the company's commitment to working closely with the FDA as they advance the development of this promising therapy to address the needs of patients.
Karyopharm initiated a pivotal Phase 3 clinical trial (XPORT-MF-034) in June 2023 to assess the effectiveness and safety of once-weekly selinexor 60 mg in combination with ruxolitinib in myelofibrosis patients who have not received JAK inhibitors (JAKi) before. Updated data from the Phase 1 study were presented at several prestigious medical conferences, showcasing significant and sustained spleen responses as well as notable improvements in symptoms for patients receiving selinexor 60 mg in combination with ruxolitinib. The Phase 3 study is anticipated to produce top-line data in 2025. Karyopharm has plans to expand the clinical development program for selinexor in myelofibrosis by investigating its potential in other JAKi-naïve scenarios, including novel combinations, with the aim of benefiting a broader patient population.
Fast Track Designation is granted to facilitate the development and expedite the review process of drugs aimed at treating severe and life-threatening conditions. It involves regular interactions between the pharmaceutical company and the FDA review team and, if specific criteria are met, can lead to Priority Review and Rolling Review eligibility, enabling an accelerated pathway to market approval.