FDA grants orphan drug designation to several agents for solid tumors

Issue: August 25, 2017

The FDA granted orphan drug designation to five agents under investigation for the treatment of solid tumors.

These include:

tucatinib (ONT-380, Cascadian Therapeutics) — an investigational, orally bioavailable, potent tyrosine kinase inhibitor that is highly selective for HER-2 without significant inhibition of EGFR — for patients with breast cancer whose disease metastasized to the brain;
IT-139 (Intezyne Technologies), which downregulates the stress induction of GRP78 in cancer cells, for pancreatic cancer;
tazemetostat (Epizyme), a first-in-class EZH2 inhibitor, for the treatment of soft tissue sarcoma;
entrectinib (RXDX-101, Ignyta) — a novel, orally available, central nervous system-active TKI designed to target tumors that harbor activating alterations to NTRK1/2/3, ROS1 or ALK — for the treatment of patients with NTRK fusion-positive solid tumors; and
ALM201 (Almac Discovery) — a therapeutic peptide developed to imitate properties of the FKBPL protein, a naturally secreted protein that has effects on multiple tumor biology processes, including cancer stem cells and angiogenesis — for ovarian cancer.

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.