Nuvisertib Granted Orphan Drug Designation in the European Union for Myelofibrosis

Nuvisertib (TP-3654) has received Orphan Drug Designation from the European Medicines Agency (EMA) for the treatment of myelofibrosis, according to a press release from Sumitomo Pharma America.¹

The designation is given to investigational drugs for rare diseases affecting fewer than 5 in 10,000 people in the European Union, which have no satisfactory diagnosis, prevention, or treatment.¹

Nuvisertib, an oral, investigational, highly selective PIM1 kinase inhibitor, previously received FDA Fast Track Designation in June 2025.¹

“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, PhD, president and chief executive officer of Sumitomo Pharma America, in the press release.¹

The safety and efficacy of nuvisertib is currently being investigated in a global phase 1/2 study. According to preclinical data presented at the European Hematology Association 2025 Congress, nuvisertib monotherapy was well tolerated and demonstrated clinical activity such as spleen volume reduction of 25% or greater, symptom improvement, bone marrow fibrosis reduction, and hemoglobin and platelet responses in patients with relapsed or refractory myelofibrosis.²

Read more: Oral PIM1 Kinase Inhibitor Receives FDA Fast Track Designation for Myelofibrosis