Sumitomo Pharma's Nuvisertib Gains FDA Fast Track for Myelofibrosis Treatment

Sumitomo Pharma America, Inc. (SMPA) has announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to nuvisertib (TP-3654), a promising drug candidate for treating patients with intermediate or high-risk myelofibrosis (MF). This designation is reserved for drugs that show potential in addressing serious or life-threatening conditions, highlighting nuvisertib's potential to fulfill unmet medical needs.

Nuvisertib is an investigational oral drug that selectively inhibits PIM1 kinase. Recent data from the European Hematology Association (EHA) 2025 Congress indicate that nuvisertib offers clinical benefits, including symptom improvement and spleen size reduction, through its modulation of cytokines. These promising preliminary results underline the drug's potential effectiveness in treating MF.

Myelofibrosis is a rare and severe blood cancer characterized by the fibrous buildup in bone marrow, resulting from disruptions in the Janus-associated kinase (JAK) signaling pathway. Patients with MF experience symptoms such as an enlarged spleen, fatigue, and reduced levels of hemoglobin and platelets. This disease affects approximately one in 500,000 people globally, posing significant treatment challenges due to its complexity and severity.

Tsutomu Nakagawa, Ph.D., President and CEO of SMPA, expressed optimism about the progress of nuvisertib, noting that the FDA's Fast Track Designation strengthens confidence in its potential as an alternative treatment for MF patients. This step reflects SMPA's commitment to developing therapies that address serious medical needs, particularly for conditions with limited treatment options. The company is dedicated to collaborating with the FDA to expedite the clinical development of nuvisertib.

The ongoing Phase 1/2 study of nuvisertib, targeting patients with relapsed or refractory MF, has revealed that the drug is generally well tolerated. Preliminary data indicate significant clinical activity, such as a reduction in spleen volume (SVR25) in over 22% of patients and a decrease in total symptom score (TSS50) in 44.4%. Improvements have also been observed in bone marrow fibrosis, hemoglobin levels, and platelet counts in a substantial percentage of patients. Additionally, nuvisertib has shown to affect cytokine levels, reducing pro-inflammatory and increasing anti-inflammatory cytokines, which correlates significantly with symptom improvements.

Jatin Shah, MD, Chief Medical Officer for Oncology, noted the promising clinical activity of nuvisertib, highlighting its potential to inhibit PIM1 selectively and to decelerate the progression of myelofibrosis. The Fast Track Designation by the FDA underscores the potential for nuvisertib to offer substantial benefits to those affected by this challenging condition, emphasizing the need for new therapeutic strategies that provide safe and lasting responses with minimal adverse effects.

Nuvisertib (TP-3654) is currently being evaluated in clinical trials for its efficacy and safety in treating intermediate and high-risk myelofibrosis. The drug has been granted Orphan Drug Designation by both the U.S. FDA and the Japan Ministry of Health, Labour and Welfare (MHLW), marking its significance in addressing the needs of patients with this rare disease.

Sumitomo Pharma Co., Ltd., a global pharmaceutical company, is committed to developing innovative treatments across various medical fields. With its operations extending to the U.S., Canada, and Europe, the company aims to accelerate research and bring novel therapies to the market, addressing the needs of patients worldwide.