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FDA Grants $23 Million for Rare Disease Research

OCTOBER 17, 2016
Andrew Black
A large amount of money is being granted towards rare disease research development. The FDA is handing out $23 million in a siloed fund to research groups for early and later-stage rare disease research.
 
The funding will be distributed throughout 4 years to 21 different companies and institutions. 43% of this year’s awards fund studies that enroll pediatric patients as young as newborns. 
 
The FDA’s reasoning behind the funding is to help encourage clinical development of orphan drugs, biologics, medical devices, or medical foods for rare disease treatments.
 
“The grants awarded this year will support much-needed research in 21 different rare diseases, many of which have little, or no, available treatment options” The FDAS stated.

The Winners

  • Chemigen, LLC (Zionsville, Indiana), Yansheng Du, Phase 1 Study of CC100 for the Treatment of Amyotrophic Lateral Sclerosis — about $243,000 for one year
  • Chemocentryx, Inc. (Mountain View, California), Petrus Bekker, Phase 2 Study of CCX168 for the Treatment of Anti-Neutrophil Cytoplasmic Auto-Antibodies Associated Vasculitis — $500,000 for one year
  • Columbia University Health Sciences (New York, New York), Elizabeth Shane, Phase 2B Study of Denosumab to Prevent Bone Loss in Idiopathic Osteoporosis in Premenopausal Women Treated with Terripatide — about $1.6 million over four years
  • DNATRIX, Inc. (Houston, Texas), Frank Tufaro, Phase 2 Study of DNX-2401 for the Treatment of Glioblastoma — $2 million over four years
  • Elorac, Inc. (Vernon Hills, Illinois), Scott Phillips, Phase 3 Study of Naloxone Lotion for the Treatment of Pruritus in Mycosis Fungoides — about $2 million over four years
  • Johns Hopkins University (Baltimore, Maryland), Pamela Zeitlin, Phase 1/2 Study of Glycerol Phenylbutyrate for the Treatment of Cystic Fibrosis — $750,000 over three years
  • Oncoceutics, Inc. (Hummelstown, Pennsylvania), Wolfgang Oster, Phase 1/2 Study of ONC201 for the Treatment of Multiple Myeloma — about $1.7 million over four years
  • Oregon Health and Science University (Portland, Oregon), Kevin Winthrop, Phase 2 Study of Clofazimine for the Treatment of Pulmonary Mycobacterium Avium Disease — about $1.8 million over four years
  • Santhera Pharmaceuticals (Liestal, Switzerland), Thomas Meier, Phase 1 Study of Omigapil for the Treatment of Congenital Muscular Dystrophy — $246,000 for one year
  • Scioderm, Inc. (Durham, North Carolina), Jay Barth, Phase 3 Study of SD101 for the Treatment of Epidermolysis Bullosa — $500,000 for one year
  • Seattle Children's Research Institute (Seattle, Washington), Leslie Kean, Phase 2 Study of Abatacept Combined with Calcineurin Inhibition and Methotrexate for Prophylaxis of Graft Vs Host Disease — $99,630 for one year
  • Sloan-Kettering Institute Cancer Research (New York, New York), Katharine Hsu, Phase 1 Study of Humanized 3F8 MoAb and NK cells for the Treatment of Neuroblastoma — about $750,000 over three years
  • Taimed Biologics USA Corp (Irvine, California), Stanley Lewis, Phase 3 Study of Ibalizumab for the Treatment of Patients with Multidrug Resistant HIV — $500,000 for one year
  • University of Alabama (Birmingham, Alabama), Gregory Friedman, Phase 1 Study of HSV G207 & Radiation for the Treatment of Pediatric Brain Tumors — about $750,000 over three years
  • University of California, San Diego (La Jolla, California), Donald Durden, Phase 1 Study of PI-3 Kinase/BRD4 Inhibitor SF1126 for the Treatment of Hepatocellular Carcinoma — $750,000 over three years
  • University of Florida (Gainesville, Florida), Peter Stacpoole, Phase 3 Study of Dichloroacetate for the Treatment of Pyruvate Dehyrugenase Complex Deficiency — about $2 million over four years
  • University of Michigan (Ann Arbor, Michigan), Kathleen Stringer, Phase 2 Study of Inhaled Activase for the Treatment of Acute Plastic Bronchitis — $2 million over four years
  • University of North Carolina Chapel Hill (Chapel Hill, North Carolina), Matthew Laughon, Phase 2 Study of Furosemide for the Prevention of Bronchopulmonary Dysplasia in Premature Infants — about $1.4 million over four years
  • Vanderbilt University Medical Center (Nashville, Tennessee), Cyndya Shibao, Phase 2 Study of Atomoxetine for the Treatment of Multiple System Atrophy — about $1.6 million over four years
  • Wilson Wolf Manufacturing Corporation (New Brighton, Minnesota), Sunitha Kakarla, Phase 1 Study of Viralym-A for the Treatment of Adenovirus Disease — about $750,000 over three years


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