FDA Helps Fund 21 Early Phase Trials for Rare Disease Treatments

The US Food and Drug Administration (FDA) on Monday announced it is awarding $23 million in research grants for 21 new clinical trials over the next four years to boost the development of rare disease treatments.

The grants, awarded to researchers from academia and industry at domestic and international clinical sites, are part of FDA’s Orphan Products Clinical Trials Grants Program, created in 1983, which has provided more than $370 million (including $19 million last year) to fund more than 590 new 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, including treatments for neuroblastoma, lymphangioleiomyomatosis, hypoparathyroidism and hypophosphatasia.

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.

Conflict of Interest?

This round and previous funding for products the agency later reviews could raise some eyebrows in terms of conflicts of interest, particularly as FDA is paying for the development and then gets to decide whether to approve the products it helped to fund.

However, it’s unclear how many of the FDA reviewers for these 55 products knew during the review process that the agency had funded part of the treatments’ development. 

FDA spokeswoman Sandy Walsh told Focus: "The FDA’s Office of Orphan Products Development’s (OOPD) mission includes implementing the Orphan Drug Act, in part, through its Clinical Trial Grant Program. OOPD works to pursue thorough and fair evaluation of medical products for rare disease patients. To avoid real or perceived conflicts of interest in the grant program, OOPD requires that all orphan clinical trial grant applications are reviewed and scored by external ad hoc experts who are external to FDA. FDA’s product reviewers who evaluate the subsequent marketing applications are not part of the grant application scoring, funding decisions, nor have a part in the orphan grant management process. 

"OOPD does not provide a listing of FDA-funded grants to FDA reviewers; however, we note that FDA funded studies are considered public information and these studies must comply with posting requirements for ClincialTrials.Gov," she added.

Awards

FDA says it received 68 grant applications for this fiscal year, with a funding rate of 31% (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

Editor's note: Article updated on 10/19 with comment from FDA.