On May 16, 2023 Tyligand Bioscience, a clinical stage biotech company focused on the development of innovative drugs to treat drug resistant tumors, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to TSN084, the company’s multi-kinase inhibitor for the treatment of Acute Myeloid Lymphoma (AML) (Press release, Tyligand Bioscience, MAY 16, 2023, View Source [SID1234644986]).
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The orphan drug designation process ("ODD") begins with an application to the US Food and Drug Administration ("FDA") Orphan Drug Designation office, and is accompanied by a comprehensive package that includes the basic science, analysis of addressable number of individuals with Acute Myeloid Lymphoma (AML) and description of the patients projected to be helped by a therapy.
Dr. Tony Zhang, cofounder and CEO of Tyligand Bioscience stated, "Orphan drug designation represents a major milestone for Tyligand in pursuit of accelerated marketing approval for TSN084, our front runner of innovative molecules designed to treat drug resistant malignancies."
The company developed TSN084 leveraging a novel Type II kinase binding mode that targets CDK8/19, FLT3, AXL & cMet etc., for the treatment of both solid tumors and hematological malignancies. Drugs intended to treat orphan diseases (rare diseases that affect less than 200,000 people in the US) are eligible to apply for ODD, which provides multiple benefits to the sponsor during development and after approval.
About Acute myeloid Leukemia :
Acute myeloid leukemia (AML) is the most common type of acute leukemia in adults. The average age at diagnosis is around 68 years—though it can also occur in adolescents and children. According to the National Cancer Institute, AML accounts for 1% of all cancer cases in the U.S., and an estimated 20,050 people in the U.S. were newly diagnosed with it in 2022. Despite currently available treatments for AML, the five-year overall survival rate for patients remains less than 30%. Novel therapies are needed to improve patient survival and quality of life.