FDA awards 21 grants to stimulate product development for rare diseases

The U.S. Food and Drug Administration announced that it has awarded 21 new clinical trial research grants totaling more than $23 million over the next four years to boost the development of products for patients with rare diseases. These new grants were awarded to principal investigators from academia and industry with research spanning domestic and international clinical sites.

“We are proud of our 30-year track record of fostering and encouraging the development of safe and effective therapies for rare diseases through our clinical trials grant program,” said Gayatri R. Rao, M.D., J.D., director of FDA’s Office of Orphan Product Development, within the Office of Special Medical Programs. “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 FDA awards the grants through the Orphan Products Clinical Trials Grants Program to encourage clinical development of drugs, biologics, medical devices, or medical foods for use in rare diseases. The grants are intended for clinical studies evaluating the safety and effectiveness of products that could either result in, or substantially contribute to, the FDA approval of products.

Since its creation in 1983, the Orphan Products Clinical Trials Grants Program has provided more than $370 million to fund more than 590 new clinical studies and supported the marketing approval of more than 55 products. Five of the studies funded by this grants program supported product approvals in 2015 alone, including much needed treatments for neuroblastoma, lymphangioleiomyomatosis, hypoparathyroidism, and hypophosphatasia.

Consistent with the tenor set by Vice President Joe Biden’s National Cancer Moonshot Initiative to accelerate cancer research, 24 percent of the new grant awards fund studies enrolling patients with cancer; 40 percent of these studies target devastating forms of brain cancer, one of which recruits children with recurrent or progressive malignant brain tumors.

Forty-three percent of this year’s awards fund studies that enroll pediatric patients as young as newborns. Of these, two focus on research in transplantation and related issues.

In addition, one funded project is a medical device trial to develop a fully implantable neuroprosthesis for grasp, reach, and trunk function in individuals with spinal cord injury with the potential to enable these patients to use their hand, arm, and trunk more independently.

A total of 68 grant applications were received for this fiscal year, with a funding rate of 31 percent (21/68). The grant recipients for fiscal year 2016 include:

Drugs/Biologics:

• 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

Medical Devices:

• Case Western Reserve University (Cleveland, Ohio), 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.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.