FDA grants orphan drug designation to TP-3654 for myelofibrosis
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The FDA granted orphan drug designation to TP-3654 for the treatment of myelofibrosis.
TP-3654 (Sumitomo Pharma Oncology) is an investigational oral inhibitor of PIM kinases.
“PIM kinases have potential antitumor and antifibrotic effects through multiple pathways, including induction of apoptosis,” Jatin J. Shah, MD, chief medical officer for Sumitomo Pharma Oncology, said in a company-issued press release. “PIM kinase expression correlates with increased cell survival and reduced apoptosis in tumors, supporting the potential of PIM kinases as novel therapeutic targets. PIM-1 expression is significantly elevated in myelofibrosis hematopoietic cells and, therefore, a potential therapeutic target for myelofibrosis.”
A multicenter, open-label phase 1/phase 2 is underway to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of TP-3654 for patients with intermediate- or high-risk primary or secondary myelofibrosis. The trial is open at sites in the United States and Japan.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.
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