On July 10, 2017 Ignyta, Inc. (Nasdaq: RXDX), a biotechnology company focused on precision medicine in oncology, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to entrectinib for "treatment of NTRK fusion-positive solid tumors (Press release, Ignyta, JUL 10, 2017, View Source [SID1234519777])." NTRK fusions are molecular alterations that occur in a broad variety of adult and pediatric solid tumor types. Entrectinib is the company’s investigational, orally available, CNS-active tyrosine kinase inhibitor targeting tumors that harbor NTRK1/2/3, ROS1, or ALK gene fusions. Schedule your 30 min Free 1stOncology Demo! About Orphan Drug Designation
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Under the FDA’s Orphan Drug Designation program, orphan drug designation is granted by the FDA to novel drugs or biologics that treat rare diseases or conditions affecting fewer than 200,000 patients in the U.S. The designation allows the drug developer to be eligible for a seven-year period of U.S. marketing exclusivity upon approval of the drug, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical trial design assistance, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees.
About Entrectinib
Entrectinib is a novel, orally available, CNS-active tyrosine kinase inhibitor targeting tumors that harbor activating alterations to NTRK1/2/3 (encoding TRKA/TRKB/TRKC), ROS1 or ALK. Entrectinib is the only TRK inhibitor with clinically demonstrated activity against primary and metastatic CNS disease, and has not shown undesirable off-target activity. This product candidate is in a Phase 2 clinical trial called STARTRK-2, which is the second of the "Studies of Tumor Alterations Responsive to Targeting Receptor Kinases." The trial is a global, multicenter, open label, potentially registration-enabling Phase 2 clinical trial of entrectinib that utilizes a basket design with screening of patient tumor samples for the relevant targets. Such a basket design takes full advantage of entrectinib’s demonstrated preliminary clinical activity across a range of different tumor types and molecular targets.