On December 20, 2022 NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Toru Nakai), reported that the U.S. FDA has granted Orphan Drug Designation to NS-018 (ilginatinib), an investigational treatment for myelofibrosis (MF), a rare and incurable blood cancer (Press release, NS Pharma, DEC 20, 2022, View Source [SID1234625461]).

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The FDA issues Orphan Drug Designations to support the development and evaluation of new treatments to prevent, diagnose, or treat a rare disease or condition.

MF is caused by buildup of excessive scar tissue in the bone marrow, which impairs the body’s ability to produce blood cells.1 In addition to impaired blood cell production, MF often leads to enlargement of the spleen (splenomegaly) which can lead to feelings of abdominal pain and pressure.1 Other common symptoms include fatigue, bone pain, fever, and weight loss.1 MF can be diagnosed at any age but is most common in men and women 65 years or older.1 The median survival of patients with MF is approximately six years.1

Several gene mutations are associated with MF, and the most common mutation is to the Janus kinase 2 (JAK2) gene.2 NS-018 is a highly selective and potent inhibitor of JAK2 developed by scientists from Nippon Shinyaku.