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The Food and Drug Administration has granted Orphan Drug designation to LOXO-101 for the treatment of patients with soft tissue sarcoma. LOXO-101 is a potent, oral, selective inhibitor of tropomyosin receptor kinase (TRK) signaling molecules.

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The designation-designed to encourage development of drugs in the diagnosis, prevention, or treatment of a medical condition affecting fewer than 200,000 people in the United States-grants a product market exclusivity for a seven-year period if the sponsor complies with certain FDA specifications, as well as tax credits and prescription drug user fee waivers. The designation does not, though, shorten the duration of the regulatory review and approval process.


The TRK pathways-TRKA, TRKB, and TRKC-have been implicated in several tumor types, and LOXO-101 is currently the only TRK inhibitor in clinical development, according to a news release from the drug's manufacturer, Loxo Oncology, Inc.


LOXO-101 is currently being evaluated in a Phase I dose-escalation trial for patients with advanced solid tumors.