The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Kymera Therapeutics
lead drug candidate KT-333 for the treatment of patients with Peripheral T-cell Lymphoma (PTCL).The company said that KT-333 is a first-in-class degrader of the transcriptional regulator STAT3, its activation has been shown to be a key modulator of disease in PTCL, and there are currently no approved therapies for PTCL that target this pathway.
KT-333 is currently being investigated in an ongoing Phase 1 clinical trial in adult patients with relapsed/refractory liquid and solid tumors, including aggressive lymphomas.
Peripheral T-cell Lymphoma (PTCL), a subtype of non-Hodgkin's lymphoma, is a heterogenous group of tumors that arise from mature white blood cells (T-cells) in the lymphoid tissues in areas such as the lymph nodes, lungs, gastrointestinal tract and skin.
Nello Mainolfi, PhD, co-founder, president and CEO, said: "The Orphan Drug Designation highlights the potential of this first-in-class heterobifunctional degrader to transform the treatment of PTCL by targeting STAT3, a protein that has historically been undruggable."
FDA grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States. It also provides benefits of seven-year period of market exclusivity if the drug is approved, tax credits for qualified clinical trials and an exemption from FDA application fees.
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