The European Commission has granted Orphan Drug Designation to NS-018 (ilginatinib) for the treatment of myelofibrosis (MF), according to a press release from the drug’s manufacturer, Nippon Shinyaku Pharma Incorporated.
Orphan Drug Designation will grant 10-year marketing exclusivity for the treatment. The US Food and Drug Administration (FDA) granted Orphan Drug Designation to NS-018 in December 2022.
Read more about MF experimental treatments
“Several gene mutations are associated with MF, and the most common mutation is to the Janus kinase 2 (JAK2) gene. NS-018 is a highly selective and potent inhibitor of JAK2 developed by scientists from Nippon Shinyaku,” the press release stated.
NS-018 was previously investigated in a phase 1 clinical trial. The results showed that around half of the patients dosed had a greater than or equal to 50% reduction in palpable spleen size. Patients also had improvements in myelofibrosis-related symptoms, and roughly a third of patients had improvements in bone marrow fibrosis grade.
The manufacturer previously announced the enrollment of the first patient in an open-label, multicenter, randomized, controlled phase 2b study, where NS-018 will be compared against the best available therapy in patients with MF.
The study will investigate changes in spleen volume and total symptom score between the 2 treatment groups from baseline up to 24 weeks after treatment initiation. The number and types of treatment-emergent adverse events will also be recorded during the trial.
An estimated 120 adult participants will be enrolled in the study. Patients must have diagnoses of either primary MF, post-polycythemia vera MF, or post-essential thrombocythemia MF with severe thrombocytopenia (platelet count <50,000/μL). In the experimental group receiving NS-018, participants will self-administer 300 mg oral doses twice daily.
The study is estimated to have a primary completion date of March 24, 2024, with a study completion date of April 24, 2024.
NS-018, an investigational treatment for myelofibrosis, receives Orphan Drug Designation from the European Commission. News release. NS Pharma; August 8, 2023.
Verstovsek S, Talpaz M, Ritchie E, et al. A phase I, open-label, dose-escalation, multicenter study of the JAK2 inhibitor NS-018 in patients with myelofibrosis. Leukemia. Published online August 1, 2016. doi:10.1038/leu.2016.215
NS Pharma announces first patient enrolled in phase 2 study to assess efficacy and safety of NS-018 compared to best available therapy (BAT) in patients with myelofibrosis. News release. NS Pharma; February 17, 2023.