Tuesday, July 18, 2023
Karyopharm Therapeutics Inc. (Nasdaq: KPTI) recently announced that the United States Food and Drug Administration (FDA) has granted Fast Track Designation to their development program of selinexor for the treatment of patients with myelofibrosis, including primary myelofibrosis, post-essential thrombocythemia myelofibrosis, and post-polycythemia vera myelofibrosis. This designation recognizes the potential of selinexor in addressing the unmet medical needs of myelofibrosis patients. The company's Phase 3 study, XPORT-MF-034 (NCT04562389), commenced in June 2023, evaluating the efficacy and safety of selinexor 60 mg once weekly in combination with ruxolitinib in JAKi-naïve myelofibrosis patients. Preliminary data from the Phase 1 study, presented at the American Association for Cancer Research Annual Meeting 2023, American Society of Clinical Oncology 2023, and European Hematology Association 2023, exhibited positive spleen responses and symptom improvement in patients treated with selinexor 60 mg in combination with ruxolitinib as of the April 10, 2023, cutoff date.
The company anticipates releasing top-line data from the Phase 3 study in 2025. Karyopharm also has plans to expand the clinical development program of selinexor in myelofibrosis by investigating its potential in other JAKi-naïve settings, such as novel combinations, to benefit a broader patient population. Fast Track Designation is a regulatory pathway offered by the FDA to facilitate the development and expedite the review of drugs targeting serious and life-threatening conditions, allowing approved products to reach the market more quickly. It involves frequent interactions with the FDA review team and, if specific criteria are met, eligibility for Priority Review and Rolling Review.
