FDA Awards $19 Million for 18 New Rare Disease Research Grants

The U.S. Food and Drug Administration (FDA) has awarded 18 new research grants totaling more than $19 million to enhance the development of orphan drugs for rare disease patients.

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.

Since many rare conditions are also pediatric conditions, it is no surprise that 10 of the 18 awards fund studies that enroll pediatric patients as young as newborns. Some of the rare conditions under investigation in the grants include sickle cell anemia, amyloidosis, autosomal dominant polycystic kidney disease, prader willi syndrome, as well as numerous rare cancers,

THE GRANT RECIPIENTS FOR FISCAL YEAR 2015 ARE:

• Albert Einstein College of Medicine (Bronx, New York), Deepa Manwani (principle investigator)
  • Phase 2 Study of Gamunex (Intravenous Gammaglobulin) for the Treatment of Sickle Cell Acute Pain
  • About $1.6 million over 4 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 3 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 3 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 3 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 4 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 4 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 4 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 3 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 3 years
• Northshore University Healthsystem (Evanston, Illinois), Eli Ehrenpreis
  • Phase 1 Study of Naltrexone for the Treatment of Mesenteric Panniculitis
  • About $220,000 over 3 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 3 years
• Scioderm Inc. (Durham, North Carolina), Robert Ryan
  • Phase 2 Study of SD-101 for the Treatment of Epidermolysis Bullosa
  • $400,000 for 1 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 4 years
• Transderm Inc. (Santa Cruz, California), Roger Kaspar
  • Phase 1 Study of Sirolimus for the Treatment of Pachyonychia Congenita
  • About $400,000 over 2 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 4 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 4 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 4 years
• Innovative Biotherapies Inc. (Ann Arbor, Michigan), H. David Humes (medical device awardee)
  • Phase 2 Study of Selective Cytopheretic Device for the Treatment of Pediatric Patients with Acute Kidney Injury
  • About $1.6 million over 4 years

---

Source

FDA awards 18 grants to stimulate product development for rare diseases [news release]. Washington, DC: US Food and Drug Administration; September 21, 2015.http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm463539.htm