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Neuroblastoma: United Therapeutics’ Orphan Drug Approval And Rare Pediatric Priority Review Disease Voucher

March 15, 2015



This Blog Post discusses United Therapeutics’ orphan drug Unituxin (Dinutuximab) Injection:

•   FDA approval

•   FDA issuance of Rare Pediatric Priority Review Voucher (PPRV).

I – FDA Approval of Orphan Drug

United Therapeutics Corporation, a Silver Spring, Maryland-based biotechnology company focusing on developing products for chronic and life-threatening conditions, announces March 10th that the FDA approves of the company’s orphan drug Unituxin. The approval is for Unituxin Injection, in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), interleukin-2 (IL-2), and 13-cis-retinoic acid (RA), for pediatric patients with high-risk Neuroblastoma who achieve at least a partial response to prior first-line multiagent, multimodality therapy. Unituxin is approved with a Boxed Warning that the drug irritates nerve cells along with other serious side effects. The Boxed Warning requires United Therapeutics to conduct certain post-marketing studies.

According to an online article in Drug Discovery & Development, Unituxin is only the 3rd drug receiving initial FDA approval for a pediatric cancer in more than 20 years. Unituxin receives FDA Orphan Drug Designation (ODD) in December 2010.

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.

II – FDA Issuance of Rare Pediatric Priority Review Voucher (PPRV)

Also, United Therapeutics announces on March 10th, that the FDA issues a Rare Pediatric Priority Review Voucher (PPRV) for Neuroblastoma. The PPRV can be used to obtain priority review for a New Drug Application (NDA) or Biologics License Application (BLA) submitted later. United Therapeutics can also sell (without limitation) or transfer the PPRV in the future. The PPRV is created in 2012 by the Food and Drug Administration Safety and Innovation Act (FDASIA) with the purpose of encouraging biotechnology companies to develop therapeutics for rare pediatric diseases. This is the 2nd PPRV the FDA grants since its inception.

According to an online article from the Regulatory Affairs Professionals Society (RAPS):

“Drugmaker United Therapeutics has become just the 2nd company in US history to obtain a new type of voucher which allows a company to potentially get its drug approved by the US Food and Drug Administration (FDA) in 40% less time than it normally takes … FDA had only awarded one rare pediatric disease voucher – in  February 2014 to the pharmaceutical company BioMarin for the approval of its rare disease drug Vimizim. BioMarin later sold the voucher for a then-record-setting price of $67.5 million.”

BioMarin Pharmaceuticals sold its PPRV to Regeneron for $67.5 million in July 2014.

FDA ODD Database Record For United Therapeutics

Generic Name: Dinutuximab
Trade Name: n/a
Date Designated: 12-20-2010
Orphan Designation: Neuroblastoma
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: United Therapeutics Corporation

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

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

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