Sumitomo Pharma America announced that the European Medicines Agency (EMA) granted Orphan Drug Designation to nuvisertib (TP-3654), an oral investigational highly selective inhibitor of PIM1 kinase, for the treatment of patients with myelofibrosis (MF).
Orphan Drug Designation is granted by the EMA to investigational therapies addressing rare diseases or conditions that affect not more than 5 in 10,000 people in the European Union, and for which there is no satisfactory method of diagnosis, prevention or treatment of the condition. This designation comes on the heels of the recent U.S. Food and Drug Administration (FDA) Fast Track Designation granted to nuvisertib, and following the oral presentation of updated preliminary Phase 1/2 data at the European Hematology Association (EHA) 2025 Congress in Milan, Italy.
"Following the recent FDA Fast Track Designation, being granted Orphan Drug Designation from European regulators emphasizes the potential of nuvisertib as a future option for patients living with myelofibrosis," said Tsutomu Nakagawa, Ph.D, President and Chief Executive Officer of SMPA. "As we pursue our mission of addressing unique and unmet patient needs, we look forward to working with the EMA to advance the clinical development of nuvisertib."
MF, a rare type of blood cancer, is characterized by the buildup of fibrous tissues in the bone marrow, which is caused by dysregulation in the Janus-associated kinase (JAK) signaling pathway. The clinical manifestations of MF include an enlarged spleen, debilitating symptoms and reduction in hemoglobin and/or platelets. MF affects 1 in 500,000 people worldwide.1
"Patients living with incurable blood cancers like myelofibrosis continue to face limited treatment options, particularly in the relapsed or refractory setting," said Professor Francesco Passamonti, M.D., Head of Department of Oncology and Hematology-Oncology, University of Milan, Italy. "The orphan drug designation for nuvisertib underscores its potential and the urgent need for new therapeutic approaches to a difficult to treat disease."
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