Orphan Products Grants Program from Food and Drug Administration

2016

  • Phase 1 Study of CC100 for the Treatment of Amyotrophic Lateral Sclerosis (website)
  • Phase 1 Study of HSV G207 & Radiation for the treatment of Pediatric Brain Tumors (website)
  • Phase 1 Study of Humanized 3F8 MoAb and NK cells for the Treatment of Neuroblastoma (website)
  • Phase 1 Study of Viralym-A for the Treatment of Adenovirus Disease (website)
  • Phase 1/2 Study of Glycerol Phenylbutyrate for the Treatment of Cystic Fibrosis (website)
  • Phase 1/2 Study of ONC201 for the Treatment of Multiple Myeloma (website)
  • Phase 2 Study of CCX168 for Treatment of Anti-Neutrophil Cytoplasmic Auto-antibodies associated Vasculitis (website)
  • Phase 2 Study of Clofazimine for the Treatment of Pulmonary Mycobacterium Avium Disease (website)
  • Phase 2 Study of DNX-2401 for the Treatment of Glioblastoma (GBM) (website)
  • Phase 2 Study of Furosemide for the Prevention of Bronchopulmonary Dysplasia in Premature Infants (website)
  • Phase 2 Study of Inhaled Activase for the Treatment of Acute Plastic Bronchitis (website)
  • Phase 2B Study of Denosumab to Prevent Bone Loss in Idiopathic Osteoporosis in Premenopausal Women Treated w Teriparatide (website)
  • Phase 3 Study of Dichloroacetate (DCA) for the Treatment of Pyruvate Dehyrugenase Complex Deficiency (website)
  • Phase 3 Study of Naloxone Lotion for the Treatment of Pruritus in Mycosis Fungoides (website)
  • Phase I Study of Omigapil for the Treatment of Congenital Muscular Dystrophy (CMD) (website)

2015

  • Phase 1 Study of Cellular Immunotherapy Using Optimized IL13Ra2 Specific CAR T cells for the Tx of Malignant Glioma (website)
  • Phase 1 Study of Low Dose Pioglitazone for the Treatment of Autosomal Dominant Polycystic Kidney Disease (website)
  • Phase 1 Study of Naltrexone for the Treatment of Mesenteric Panniculitis (website)
  • Phase 1 Study of Sirolimus for the Treatment of Pachyonychia Congenita (website)
  • Phase 1A/B Study of 11-1F4 mAb for the Treatment of AL Amyloidosis (website)
  • Phase 2 Study of ADXS11-001 Vaccine for the Treatment of HPV-Related Oropharyngeal Cancer (website)
  • Phase 2 Study of Arimoclomol for the Treatment of Sporadic Inclusion Body Myositis (website)
  • Phase 2 Study of Carbidopa for the Treatment of Familial Dysautonomia (website)
  • Phase 2 Study of EDI200 for the Treatment of X-Linked Hypohidrotic Ectodermal Dysplasia (website)
  • Phase 2 Study of Gamunex (Intravenous Gammaglobulin) for the Treatment of Sickle Cell Acute Pain (website)
  • Phase 2 Study of Imatinib for the Treatment of Airway Tumors in Children with Neurofibromatosis Type 1 (website)
  • Phase 2 Study of L-Arginine Therapy for the Treatment of Pediatric Sickle Cell Disease Pain (website)
  • Phase 2 Study of SD-101 for the Treatment of Epidermolysis Bullosa (website)
  • Phase 2 Study of Selective Cytopheretic Device for the Treatment of Pediatric Patients w/ Acute Kidney Injury (website)
  • Phase 2 Study of the Melanocortin 4 Receptor Agonist RM-493 for the Treatment of Prader Willi Syndrome (website)
  • Phase 2 Study of Vascular-Targeted Prodrug (G-202) for the Treatment of Recurrent Glioblastoma (website)
  • Phase 3 Study of Sorafenib for the Treatment of Desmoid Tumors or Aggressive Fibromatosis (website)
  • Phase 3 Study of Standard vs Reduced IV Fat for the Prevention of Parenteral Nutrition-Associated Cholestasis (PNAC) (website)

2014

  • Phase 1 Study of Intrathecal Autologous Mesenchymal Stem Cell Therapy for the Treatment of Multiple System Atrophy (website)
  • Phase 1 Study of Neural Stem Cells & 5-FC/Leucovorin for the Treatment of Recurrent High Grade Gliomas (website)
  • Phase 1 Study of VSV-hIFN-B for the Treatment of Hepatocellular Carcinoma
  • Phase 1/2 Study of Aerosolized Survanta for the Treatment of Neonatal Respiratory Distress Syndrome
  • Phase 2 Study of Aes103 (5-HMF) for the Treatment of Stable Sickle Cell Disease
  • Phase 2 Study of PA-824 for the Treatment of Pulmonary Tuberculosis (website)
  • Phase 2 Study of Poly-ICLC in the Treatment of Pediatric Low Grade Gliomas (website)
  • Phase 2 Study of RV001 for the Treatment of Thyroid Eye Disease
  • Phase 2 Study of SelG1 for the Treatment of Sickle Cell Disease
  • Phase 2 Study of Sildenafil for the Treatment of Lymphatic Malformations (website)
  • Phase 2 Study of TH-302 for the Treatment of Glioblastoma
  • Phase 2 Study of Vincristine Vs. Sirolimus for the Treatment of High Risk Kaposiform Hemangioendothelioma (website)
  • Phase 3 Study of Triiodothyronine Supplementation for the Treatment of Young Infants After Cardiopulmonary Bypass (website)

2013

  • Phase 1 Study of ALK001 for the Treatement of Stargardt Disease (website)
  • Phase 1 Study of IL-2 for the Treatment of Wiskott-Aldrich Syndrome (website)
  • Phase 1 Study of Immunotherapy with NK Cells anti-GD2 for Neuroblastoma (website)
  • Phase 1 Study of Quercetin for the Treatment of Fanconi Anemia (website)
  • Phase 1/2 Study of Clenbuterol for the Treatment of Pompe Disease (website)
  • Phase 1/2 Study of Taurine for the Treatment of Cystathionine Beta-Synthase Deficient Homocystinuria (website)
  • Phase 2 A Study of Exendin for the Treatment of Congenital Hyperinsulinism (website)
  • Phase 2 Study of Abatacept combined w Calcineurin Inhibition and Methotrexate for Prophylaxis of Graft Vs Host Disease (website)
  • Phase 2 Study of Esophageal String Test in Monitoring Inflammation in Patients with Eosinophilic Esophagitis (website)
  • Phase 2 Study of Folfirinox followed by Ipilimumab/GVAX for the Treatment of Pancreatic Cancer (website)
  • Phase 2 Study of Hemin for Treatment and Prevention of Porphyria Attacks (website)
  • Phase 2 Study of Montelukast for the Treatment of Sickle Cell Anemia (website)
  • Phase 2 Study of rhCC10 to Prevent Neonatal Bronchopulmonary Dysplasia (website)
  • Phase 2B Study of Memantine for the Treatment of Amyotrophic Lateral Sclerosis (website)

2012

  • Phase 1 Study of ABC294640 for the Treatment of Pancreatic Cancer (website)
  • Phase 1 Study of Resiquimod Gel Therapy for the Treatment of Cutaneous T-Cell Lymphoma (website)
  • Phase 1/2 of AT-101 With Lenalidomide for the Treatment of Chronic Lymphocytic Leukemia (website)
  • Phase 2 Study of Levetiracetam in the Treatment of Neonatal Seizures (website)
  • Phase 2 Study of Orlistat + SLx-4090 for Treatment of Type I Hyperlipoproteinemia (website)
  • Phase 2 Study of Rasagiline for the Treatment of Amyotrophic Lateral Sclerosis (website)
  • Phase 2 Study of TOL101 Induction in the Prevention of Kidney Transplantation Rejection (website)

2011

  • Phase 2 Study of 4-aminopyridine for Treatment of Episodic Ataxia Type 2 (website)
  • Phase 2 Study of EGEN-001 for the Treatment of Chemotherapy-Resistant Recurrent Ovarian Cancer (website)
  • Phase 2 Study of ENB-0040 for Hypophosphatasia (website)
  • Phase 2 study of Mexiletine in Tx of Myotonic Dystrophy Type 1 (website)
  • Phase 2 Study of Vitamin D for Prevention of Respiratory Complications from Sickle Cell Disease (website)

2010

  • Phase 1 Study of HSV1716 in Patients with Non-CNS Solid Tumors (website)
  • Phase 1/2 Trial of rAAV2-CB-hRPE65 for Leber Congenital Amaurosis (website)
  • Phase 2 of Nexagon for the Treatment of Persistent Corneal Epithelial Defects (website)
  • Phase 2 Studies of High TC Susceptometer to monitor Transfusional Iron Overload (website)
  • Phase 2 Study of [18F] FLT for PET Imaging of Brain Tumors in Children (website)
  • Phase 2 Study of Growth Hormone for Treatment of Albright Hereditary Osteodystrophy (website)
  • Phase 2 Study of Inhibition of Aurora Kinase A as Treatment for Stage III or IV Melanoma (website)
  • Phase 2 Study of Irinotecan with and without Flavopiridol Gastric Adenocarcinoma (website)
  • Phase 2 Study of PTC299 in Glioblastoma Multiforme (website)
  • Phase 2 Study of rAAV1-CB-hAAT for Treatment of Alpha-1 Antitrypsin Deficiency (website)
  • Phase 2 Study of Radioactive Iodide Therapy for Pediatric Graves Disease (website)
  • Phase 3 Study of Anacoral (Coralmyn) Antivenom for Emergency Treatment of Coral Snake Envenomation (website)
  • Phase 3 Study of PTC124 in Cystic Fibrosis (website)
  • Phase 3 Trial of orBec (oral BDP) in Patients with Gastrointestinal Graft Versus Host Disease (GI GVHD) (website)
  • Phase II trial of Levodopa in Angelman Syndrome (website)