FDA funds $23M in grants to spur rare disease drug research

The Food and Drug Administration awarded $23 million in clinical trial research grants to research teams to encourage the development of treatments for patients with rare diseases.

The 21 grants are awarded through the Orphan Products Clinical Trials Grants Program to boost the development of drugs, biologics and medical devices used to treat rare diseases. The grants fund clinical trials used to examine the safety and effectiveness of these products, with the hope positive results will lead to FDA approval, according to the report.

The FDA received 68 grant applications this year. Here are the winners.

  • Chemigen (Zionsville, Ind.), Yansheng Du, Phase 1 study of CC100 for the treatment of amyotrophic lateral sclerosis — about $243,000 for one year
  • Chemocentryx (Mountain View, Calif.), 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 City), 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 (Houston, Texas), Frank Tufaro, Phase 2 study of DNX-2401 for the treatment of glioblastoma — $2 million over four years

  • Elorac (Vernon Hills, Ill.), 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) Pamela Zeitlin, Phase 1/2 study of glycerol phenylbutyrate for the treatment of cystic fibrosis — $750,000 over three years

  • Oncoceutics, Inc. (Hummelstown, Penn.), 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, Ore.), 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 xongenital muscular dystrophy — $246,000 for one year

  • Scioderm (Durham, N.C.), Jay Barth, Phase 3 study of SD101 for the treatment of epidermolysis bullosa — $500,000 for one year

  • Seattle Children's Research Institute, 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 City), 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 (Irvine, Calif.), 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), Gregory Friedman, Phase 1 study of HSV G207 and radiation for the treatment of pediatric brain tumors — about $750,000 over three years

  • University of California, San Diego (La Jolla), 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), 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), 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, 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, Tenn.), 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, Minn.), Sunitha Kakarla, Phase 1 study of viralym-A for the treatment of adenovirus disease — about $750,000 over three years

Medical Devices:

  • Case Western Reserve University (Cleveland), Kevin Kilgore, Phase 2 study of a networked neuroprosthesis for grasp, reach and trunk function in cervical spinal cord injury — about $2 million over four years

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