Sumitomo Pharma America, Inc., US subsidiary of Japan-based pharmaceutical company Sumitomo Pharma Co., Ltd. (TYO:4506) announced on Wednesday that the European Medicines Agency (EMA) has 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).

This designation follows the recent US Food and Drug Administration (FDA) Fast Track Designation granted to nuvisertib, and the oral presentation of updated preliminary Phase 1/2 data at the European Hematology Association (EHA) 2025 Congress in Milan, Italy.

MF, a rare type of blood cancer, is characterised by the buildup of fibrous tissues in the bone marrow, which is caused by dysregulation in the Janus-associated kinase (JAK) signalling pathway.

Sumitomo says that Nuvisertib (TP-3654) is an oral investigational selective inhibitor of PIM1 kinase, which has shown potential antitumor and antifibrotic activity through multiple pathways, including induction of apoptosis in preclinical models. The safety and efficacy of nuvisertib is currently being clinically evaluated in a Phase 1/2 study in patients with intermediate and high-risk myelofibrosis (NCT04176198).

The FDA granted Orphan Drug Designation to nuvisertib for the indication of myelofibrosis in May 2022, followed by the Japan Ministry of Health, Labour and Welfare (MHLW) granting Orphan Drug Designation to nuvisertib for the treatment of myelofibrosis in November 2024.