On February 2, 2022 Theseus Pharmaceuticals, Inc. (Theseus) (NASDAQ: THRX), a clinical-stage biopharmaceutical company focused on improving the lives of cancer patients through the discovery, development and commercialization of transformative targeted therapies, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to the company’s lead candidate THE-630, a small-molecule oral pan-variant KIT inhibitor, for the treatment of patients with advanced gastrointestinal stromal tumors (GIST) (Press release, Theseus Pharmaceuticals, FEB 2, 2022, View Source [SID1234607650]).
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"We are pleased to have received ODD for THE-630 so quickly after the initiation of the first-in-human trial. This designation from the FDA emphasizes the need to bring better therapeutic options to patients with advanced GIST," said David Kerstein, M.D., Chief Medical Officer at Theseus. "For patients living with GIST, progression through standard lines of therapy is often associated with the emergence of resistance mutations in KIT. We look forward to evaluating the clinical potential of THE-630 to inhibit these resistance mutations in patients who’ve exhausted available therapies and when used in earlier lines of therapy."
The FDA’s Office of Orphan Products Development grants ODD status to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. ODD provides benefits to drug developers designed to support the development of drugs and biologics for small patient populations with unmet medical needs. These benefits include assistance in the drug development process, tax credits for qualified clinical costs, exemptions from certain FDA fees and seven years of marketing exclusivity.
About GIST
GIST is the most common sarcoma of the gastrointestinal tract with an estimated 4,000 to 6,000 new cases diagnosed in the United States each year. Approximately eighty percent of GIST cases are driven by mutations that activate the kinase activity of the receptor tyrosine kinase KIT, and up to ninety percent of all cases of progression are driven by secondary resistance mutations in KIT.