Ignyta’s Neuroblastoma Treatment Receives Orphan Drug Designation

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Entrectinib has received FDA’s orphan drug and rare pediatric disease designations for the treatment of neuroblastoma.


Ignyta, an oncology biotechnology company, announced on Dec. 29, 2014 that its new drug, entrectinib, was granted orphan drug designation and rare pediatric designation for the treatment of neuroblastoma. FDA grants Orphan Drug Designation to novel drugs or biologics that treat rare diseases or conditions affecting fewer than 200,000 patients in the US, and grants seven-year marketing exclusivity upon approval.

"Although Ignyta is intrinsically motivated to continue to aggressively pursue our clinical development program for entrectinib in solid tumors for the benefit of adult and pediatric cancer patients everywhere, we are pleased that the incentives provided by these designations-including the potential for seven years of marketing exclusivity and the potential to obtain a valuable Pediatric Disease Priority Review Voucher from the FDA-can potentially provide additional avenues for creating value for our stockholders,” said Jonathan Lim, MD, chairman and CEO of Ignyta, in a press release.


Entrectinib is a novel, orally available, selective tyrosine kinase inhibitor of the Trk family of tyrosine kinase receptors (TrkA, TrkB and TrkC), ROS1 and ALK proteins, designed to treat patients with cancers that harbor activating alterations to these receptors and protein. The novel drug is currently in Phase I/II clinical trials.