Sumitomo Pharma America Announces that the European Medicines Agency Has Granted Orphan Drug Designation to Nuvisertib (TP-3654) for the Treatment of Myelofibrosis

– Nuvisertib (TP-3654), an oral investigational highly selective PIM-1 kinase inhibitor, is being evaluated in patients with relapsed or refractory myelofibrosis (MF) –

MARLBOROUGH, Mass., July 30, 2025 /PRNewswire/ -- Sumitomo Pharma America, Inc. (SMPA) today 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."

About Nuvisertib (TP-3654)
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.^2,3 Nuvisertib was observed to inhibit proliferation and increase apoptosis in murine and human hematopoietic cells expressing the clinically relevant JAK2 V617F mutation.³ Nuvisertib alone and in combination with ruxolitinib showed white blood cell and neutrophil count normalization, and also reduced spleen size and bone marrow fibrosis in JAK2 V617F and MPLW515L murine models of myelofibrosis.² 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. The Japan Ministry of Health, Labour and Welfare (MHLW) granted Orphan Drug Designation to nuvisertib for the treatment of myelofibrosis in November 2024. The FDA granted Fast Track Designation to nuvisertib for the indication of myelofibrosis in June 2025.

About Sumitomo Pharma
Sumitomo Pharma Co., Ltd., is a global pharmaceutical company based in Japan with key operations in the U.S. (Sumitomo Pharma America, Inc.), Canada (Sumitomo Pharma Canada, Inc.), and Europe (Sumitomo Pharma Switzerland GmbH) focused on addressing patient needs in oncology, urology, women's health, rare diseases, psychiatry & neurology, and cell & gene therapies. With several marketed products in the U.S., Canada, and Europe, and a diverse pipeline of early- to late-stage investigational assets, we aim to accelerate discovery, research, and development to bring novel therapies to patients sooner. For more information on SMPA, visit our website https://www.us.sumitomo-pharma.com or follow us on LinkedIn.

The Sumitomo corporate symbol mark is a trademark of Sumitomo Pharma Co., Ltd., used under license. SUMITOMO PHARMA is a trademark of Sumitomo Pharma Co., Ltd., used under license. SUMITOMO is a registered trademark of Sumitomo Chemical Co., Ltd., used under license. Sumitomo Pharma America, Inc. is a U.S. subsidiary of Sumitomo Pharma Co., Ltd.

©2025 Sumitomo Pharma America, Inc. All rights reserved.

References

¹ U.S. National Library of Medicine. (n.d.). Primary myelofibrosis: Medlineplus Genetics. MedlinePlus. https://medlineplus.gov/genetics/condition/primary-myelofibrosis/
² Dutta A., Nath D, Yang Y, et al. Genetic ablation of Pim1 or pharmacologic inhibition with TP-3654 ameliorates myelofibrosis in murine models. Leukemia. 2022; 36 (3): 746-759. doi: 10.1038/s41375-021-01464-2.
³ Foulks JM, Carpenter KJ, Luo B, et al. A small-molecule inhibitor of PIM kinases as a potential treatment for urothelial carcinomas. Neoplasia. 2014;16(5):403-412.

SOURCE Sumitomo Pharma America