FDA awards research grants to boost product development for patients with rare diseases

Oct 11 2015

The U.S. Food and Drug Administration today announced it has awarded 18 new research grants totaling more than $19 million to boost the development of products for patients with rare diseases, which affect the lives of nearly 30 million Americans. These new grants were awarded to principal investigators in ten states, with research spanning clinical sites domestically and internationally.

"The FDA is in a unique position to help those who suffer from rare diseases by offering important incentives to promote the development of products, one of which is our grants program," said Gayatri R. Rao, M.D., J.D., director of the FDA's Office of Orphan Product Development. "The grants awarded this year support much-needed research in 17 different rare diseases, many of which have little, or no, available treatment options."

The FDA awards the grants through the Orphan Products 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 Grants Program has provided more than $350 million to fund more than 570 new clinical studies and supported the marketing approval of more than 50 products.

Ten of the 18 awards fund studies that enroll pediatric patients as young as newborns. Two are funding studies related to sickle cell disease, specifically focusing on the extreme pain that patients, many of whom are children, suffer from and which serves as a leading cause of emergency room visits and hospitalizations in these patients. One of these studies evaluates the use of a medical device to treat children with acute kidney injury.

A total of 92 grant applications were received for this fiscal year. Twenty-five ad hoc panels comprising over 80 independent clinicians, scientists and researchers with expertise in these rare disease-related fields reviewed the grant applications and recommended the most promising research projects for funding.

The grant recipients for fiscal year 2015 include:

Drugs/Biologics:

• Albert Einstein College of Medicine (Bronx, New York), Deepa Manwani, Phase 2 Study of Gamunex (Intravenous Gammaglobulin) for the Treatment of Sickle Cell Acute Pain -; about $1.6 million over four years
• Baylor College of Medicine (Houston, Texas), Andrew Sikora, Phase 2 Study of ADXS11-001 Vaccine for the Treatment of HPV-Related Oropharyngeal Cancer -;about $1.2 million over three years
• Beckman Research Institute of the City of Hope (Duarte, California), Behnam Badie, Phase 1 Study of Cellular Immunotherapy Using Optimized IL13Ra2 Specific CAR T Cells for the Treatment of Malignant Glioma -;$600,000 over three years
• Columbia University (New York, New York), Suzanne Lentzsch, Phase 1A/B Study of 11-1F4 mAb for the Treatment of AL Amyloidosis -;$600,000 over three years
• Edimer Pharmaceuticals Inc. (Cambridge, Massachusetts), Neil Kirby, Phase 2 Study of EDI200 for the Treatment of X-Linked Hypohidrotic Ectodermal Dysplasia -;$1.6 million over four years
• Emory University (Atlanta, Georgia), Claudia Morris, Phase 2 Study of L-Arginine Therapy for the Treatment of Pediatric Sickle Cell Disease Pain-;$1.6 million over four years
• Indiana University-Purdue University at Indianapolis (Indianapolis, Indiana), Kent Robertson, Phase 2 Study of Imatinib for the Treatment of Airway Tumors in Children with Neurofibromatosis Type 1 -; $1.6 million over four years
• Indiana University-Purdue University at Indianapolis (Indianapolis, Indiana), Sharon Moe, Phase 1 Study of Low Dose Pioglitazone for the Treatment of Autosomal Dominant Polycystic Kidney Disease -; about $600,000 over three years
• New York University School of Medicine (New York, New York), Horacio Kaufmann, Phase 2 Study of Carbidopa for the Treatment of Familial Dysautonomia -; about $1.1 million over three years
• Northshore University Healthsystem (Evanston, Illinois), Eli Ehrenpreis, Phase 1 Study of Naltrexone for the Treatment of Mesenteric Panniculitis -; about $220,000 over three years
• Rhythm Metabolic Inc. (Boston, Massachusetts), Keith Gottesdiener, Phase 2 Study of the Melanocortin 4 Receptor Agonist RM-493 for the Treatment of Prader Willi Syndrome -; about $1 million over three years
• Scioderm Inc. (Durham, North Carolina), Robert Ryan, Phase 2 Study of SD-101 for the Treatment of Epidermolysis Bullosa -; $400,000 for one year
• Sloan-Kettering Institute Cancer Research (New York, New York), Mrinal Gounder, Phase 3 Study of Sorafenib for the Treatment of Desmoid Tumors or Aggressive Fibromatosis -; about $900,000 over four years
• Transderm Inc. (Santa Cruz, California), Roger Kaspar, Phase 1 Study of Sirolimus for the Treatment of Pachyonychia Congenita -; about $400,000 over two years
• University of California San Diego (La Jolla, California), Santosh Kesari, Phase 2 Study of Vascular-Targeted Prodrug (G-202) for the Treatment of Recurrent Glioblastoma-; about $1.6 million over four years
• University of Kansas Medical Center (Kansas City, Kansas), Mazen Dimachkie, Phase 2 Study of Arimoclomol for the Treatment of Sporadic Inclusion Body Myositis-; about $1.6 million over four years
• University of Michigan (Ann Arbor, Michigan), Meghan Arnold, Phase 3 Study of Standard vs Reduced IV Fat for the Prevention of Parenteral Nutrition-Associated Cholestasis (PNAC) -; about $1.6 million over four years

Medical Devices:

• Innovative Biotherapies Inc. (Ann Arbor, Michigan), H. David Humes, Phase 2 Study of Selective Cytopheretic Device for the Treatment of Pediatric Patients with Acute Kidney Injury -; about $1.6 million over four years