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Neuroblastoma: Ignyta’s Orphan Drug And Rare Pediatric Disease Designations

January 5, 2015

Ignyta Inc. is a San Diego, California-based oncology biotechnology company, that is pursuing an “integrated therapeutic (Rx) and companion diagnostic (Dx) strategy” for cancer treatments. The company’s mission is to pair each of the company’s product candidates with biomarker-based companion diagnostics, that will identify specific patient populations that are likely to benefit from the company’s targeted drugs – personalized medicine.

Ignyta announces December 29th that the company’s lead product candidate, Entrectinib, is granted both the FDA’s Orphan Drug Designation (ODD) and the Rare Pediatric Disease Designation, for the treatment of Neuroblastoma.

Neuroblastoma is a rare extracranial solid tumor cancer in childhood with 650 – 800 cases diagnosed each year in North America. 90% of neuroblastoma patients are less than 5 years of age at diagnosis, and the cancer is responsible for 12% of all cancer deaths in children less than 15 years of age. 60% of children diagnosed with neuroblastoma after 1 year of age have high-risk disease with less than half of these patients cured.

Entrectinib is a novel, orally available, selective tyrosine kinase inhibitor of tyrosine kinase receptors (TrkA, TrkB, and TrkC), ROS1 and ALK proteins. It is designed to treat cancers that harbor activating alterations to TrkA, TrkB, TrkC, ROS1, or ALK. Entrectinib has been observed to efficiently cross the blood brain barrier in animal species. The drug is now in 2 Phase I/II clinical trials:


•   ALKA-372-001.

FDA ODD Database Record For Ignyta Inc.

Generic Name: N-[5-(3,5-difluorobenzyl)-1H-indazol-3-yl]-4-(4-methylpiperazin-1yl)-2-(tetrahydro-2H-pyran-4-ylamino)benzamide
Trade Name: n/a
Date Designated: 12-22-2014
Orphan Designation: Neuroblastoma.
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: Ignyta, Inc. 11095 Flintkote Avenue Suite D San Diego, CA 92121



Ignyta’s Pipeline.

Please Note: “CSIRO ScienceImage 7630 Test Tubes” from CSIRO [CC BY 3.0] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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