Rare Disease Report

FDA Awards $19 Million to 15 Rare Disease Clinical Programs

SEPTEMBER 30, 2014
James Radke

Today, the U.S. Food and Drug Administration (FDA) announced it has awarded 15 grants worth more than $19 million to boost the development of medical device, drug, and biological products for patients with rare diseases.

These grants are for clinical studies that could either result in, or substantially contribute to, approval of the products by the FDA.

Gayatri R. Rao, M.D., director of the FDA’s Office of Orphan Product Development said:

“The FDA is in a unique position to help those who suffer from rare diseases by offering several important incentives to promote the development of products for rare diseases, one of which is this grants program.”

“The grants awarded this year support much-needed research in difficult-to-treat diseases that have little, or no, available treatment options.”

Since its inception, the FDA’s Orphan Products Grants Program has awarded over $330 million towards more than 530 new clinical studies that have been helpful in bringing 50+ products to market approval.

The 2014 grant recipients are:

  • Denise Adams, Cincinnati Children's Hospital Medical Center (Cincinnati, Ohio), Phase 2 Study of Vincristine vs. Sirolimus for the Treatment of High Risk Kaposiform Hemangioendothelioma—$1.6 million over four years
  • Mitesh Borad, Mayo Clinic Arizona (Scottsdale, Ariz.), Phase 1 Study of VSV-hIFN-B for the Treatment of Hepatocellular Carcinoma— approximately $600,000 over three years
  • Andrew Brenner, University of Texas Health Center San Antonio (San Antonio, Texas), Phase 2 Study of TH-302 for the Treatment of Glioblastoma— approximately $1.6 million over four years
  • Kelly Dooley, The Johns Hopkins University (Baltimore, Md.), Phase 2 Study of PA-824 for the Treatment of Pulmonary Tuberculosis— $1.6 million over four years
  • Donald Durden, University of California San Diego (San Diego, Calif.), Phase 2 Study of Poly-ICLC for the Treatment of Pediatric Low Grade Gliomas— $1.6 million over four years
  • Alfred Lane, Stanford University (Stanford, Calif.), Phase 2 Study of Sildenafil for the Treatment of Lymphatic Malformations— approximately $1.6 million over four years
  • Dung Le, The Johns Hopkins University (Baltimore, Md.), Phase 2 Study of Folfirinox followed by Ipilimumab/GVAX for the Treatment of Pancreatic Cancer— $1.6 million over four years
  • Phillip Low, Mayo Clinic Rochester (Rochester, Minn.), Phase 1 Study of Intrathecal Autologous Mesenchymal Stem Cell Therapy for the Treatment of Multiple System Atrophy—$600,000 over three years
  • Guido Magni, River Vision Development Corporation (New York, N.Y.), Phase 2 Study of RV001 for the Treatment of Thyroid Eye Disease—$1.2 million over three years
  • Michael Portman, Seattle Children's Hospital (Seattle, Wash.), Phase 3 Study of Triiodothyronine Supplementation for the Treatment of Young Infants After Cardiopulmonary Bypass— approximately $1.6 million over four years
  • Jana Portnow, City of Hope Beckman Research Institute (Duarte, Calif.), Phase 1 Study of Neural Stem Cells & 5-FC/Leucovorin for the Treatment of Recurrent High Grade Gliomas—$600,000 over three years
  • Scott Rollins, Selexys Pharmaceuticals Corporation (Oklahoma City, Okla.), Phase 2 Study of SelG1 for the Treatment of Sickle Cell Disease— $1.6 million over four years
  • Beena Sood, Wayne State University (Detroit, Mich.), Phase 1/2 Study of Aerosolized Survanta for the Treatment of Neonatal Respiratory Distress Syndrome— approximately $1.4 million over four years
  • Warren Stern, Aesrx, LLC (Newton, Mass.), Phase 2 Study of Aes103 (5-HMF) for the Treatment of Stable Sickle Cell Disease—approximately $1.6 million over four years
  • Pamela Zeitlin, The Johns Hopkins University (Baltimore, Md.), Phase 2 Study of Digitoxin for the Treatment of Cystic Fibrosis— approximately $290,000 for one year
 To learn more about the program, click here.

Stay informed on the latest rare disease news and developments by signing up for our newsletter.
Copyright © RareDR 2013-2019 Rare Disease Communications. All Rights Reserved.