Ignyta Says FDA Grants Orphan Drug Designation For Entrectinib For Neuroblastoma

(RTTNews) - Oncology biotechnology company Ignyta, Inc. (RXDX) Monday said the U.S. Food and Drug Administration has granted both orphan drug designation and rare pediatric disease designation for its lead product candidate entrectinib for the treatment of neuroblastoma.

As per FDA's Orphan Drug Designation program, orphan drug designation is granted 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.

Entrectinib is designed as a targeted therapeutic candidate to treat patients with cancers that harbor activating alterations to TrkA, TrkB, TrkC, ROS1 or ALK. Ignyta said it has filed applications with the FDA for orphan drug designation for entrectinib in multiple adult indications, in addition to neuroblastoma.