Theseus Pharmaceuticals announced the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for THE-630 to treat patients with advanced gastrointestinal stromal tumors (GIST). GIST is the most common sarcoma of the gastrointestinal tract, and a large percentage of GIST cases are driven by mutations of the receptor tyrosine kinase KIT. The new drug, THE-630, works as a small-molecule oral pan-variant KIT inhibitor and has been developed for patients who have developed mutations that have rendered previous therapies ineffective. The FDA grants ODD status to drugs and biologics intended for the diagnosis, treatment, or prevention of rare diseases or conditions that affect less than 200,000 people in the US. Theseus Pharmaceuticals is a clinical-stage biopharmaceutical company that develops targeted therapies for multiple cancer types.

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