Stamford-based Loxo Oncology Inc., a publicly traded biopharmaceutical company focused on discovering, developing and commercializing targeted cancer therapies, said the federal Food and Drug Administration has granted “orphan drug” status to its new drug, LOXO-101.
The designation is part of a program promoting the development of medicines for underserved patient populations or rare disorders. The bar for such designation is the malady affect fewer than 200,000 people in the U.S.
The FDA’s Office of Orphan Drug Products oversaw the designation. The drug is now being evaluated in tests on patients with certain advanced ”” “solid” ”” tumors.
LOXO-101 is for patients with soft-tissue sarcoma, which afflicts the body’s connective or supportive tissues, including cartilage, fat, muscle, fibrous tissue and blood vessels.
Orphan drug designation provides Loxo with benefits including market exclusivity upon regulatory approval, if received; exemption of FDA application fees and tax credits for qualified clinical trials, the company reported.
In a brief technical rundown of the treatment, the company said, “LOXO-101 is a potent, oral, selective inhibitor of tropomyosin receptor kinase signaling molecules. The TRK family ”” TRKA, TRKB, and TRKC ”” has been implicated in diverse tumor types such as lung cancer, head and neck cancer, melanoma, colorectal cancer, sarcoma and breast cancer. LOXO-101 was built specifically to inhibit TRK and is currently the only selective TRK inhibitor in clinical development. LOXO-101 is currently being evaluated in a Phase 1 dose escalation trial for patients with advanced solid tumors.”
