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FDA grants orphan drug designation to entrectinib for NTRK fusion–positive solid tumors
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The FDA granted orphan drug designation to entrectinib for the treatment of patients with NTRK fusion–positive solid tumors, according to the drug’s manufacturer.
NTRK fusions are molecular alterations that are observed in a variety of solid tumors diagnosed in adults and pediatric patients.
Entrectinib (RXDX-101, Ignyta) is a novel, orally available, central nervous system–active tyrosine kinase inhibitor designed to target tumors that harbor activating alterations to NTRK1/2/3, ROS1 or ALK.
The ongoing global, multicenter, open-label phase 2 STARTRK-2 trial is designed to evaluate entrectinib by utilizing a basket design, in which samples from patients with a variety of tumor types are screened for relevant molecular targets.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.
Earlier this year, the FDA granted breakthrough therapy designation to entrectinib for the treatment of NTRK fusion–positive locally advanced or metastatic solid tumors. That designation applied to use of the agent for adult and pediatric patients who either progressed following prior therapies, or for whom no acceptable standards exist.