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Treadwell Receives FDA Orphan Designation for Ocifisertib, a Novel PLK4 Inhibitor, Targeting Acute Myeloid Leukemia

Wednesday, February 21, 2024

Treadwell Therapeutics, a company specializing in developing innovative cancer treatments, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to ocifisertib (CFI-400945), an experimental PLK4 inhibitor designed to treat acute myeloid leukemia (AML). Ocifisertib is currently undergoing evaluation in a Phase 1b/2 trial for adults with relapsed/refractory AML following standard therapy.

Orphan drug designation is given to drugs targeting rare diseases affecting fewer than 200,000 people in the U.S. at the time of designation. This status provides various incentives and benefits under the FDA's Orphan Drug Act, including market exclusivity for seven years after marketing authorization.

Treadwell also announced the appointment of Brenda Marczi as Senior Vice President and Head of Regulatory Affairs. With over 30 years of regulatory experience in the pharmaceutical and biotechnology industries, Brenda previously held leadership roles at Tracon Pharmaceuticals, Inc. She holds degrees in Pharmaceutical Sciences, Pharmacy, and Business Administration from reputable institutions.

Treadwell, expressed the company's dedication to addressing the needs of AML patients, particularly those with limited treatment options. He emphasized the significance of ocifisertib's orphan drug designation and its potential to offer hope to patients with relapsed and/or refractory AML. Dr. Sidhu also welcomed Brenda's appointment, highlighting her expertise in regulatory affairs, which will be instrumental as Treadwell advances ocifisertib into further studies.



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