FDA Awards 21 Grants for Orphan Drugs

Issue: November 2016

The FDA has awarded 21 clinical research grants for orphan drugs, encompassing more than $23 million during the course of the next 4 years, to develop products for patients with rare diseases, according to a press release.

Of the 68 grant applications, the FDA accepted the following 21 grants:

phase 3 study of naloxone lotion (Elorac Inc.) for the treatment of pruritus in mycosis fungoides ($2 million for 4 years);
phase 3 study of dichloroacetate (University of Florida) for the treatment of pyruvate dehyrugenase complex deficiency ($2 million for 4 years);
phase 3 study of SD101 (Scioderm Inc.) for the treatment of epidermolysis bullosa ($500,000 for 1 year);
phase 3 study of ibalizumab (Taimed Biologics USA Corp.) for the treatment of multidrug-resistant HIV ($500,000 for 1 year);
phase 2b study of denosumab (Columbia University Health Sciences) to prevent bone loss in idiopathic osteoporosis in premenopausal women treated with terripatide ($1.6 million for 4 years);
phase 2 study of networked neuroprosthesis (Case Western Reserve University) for grasp, reach and trunk function in cervical spinal cord injury ($2 million for 4 years);
phase 2 study of DNX-2401 (DNATRIX Inc.) for the treatment of glioblastoma ($2 million for 4 years);
phase 2 study of inhaled activase (University of Michigan) for the treatment of acute plastic bronchitis ($2 million during the span of 4 years);
phase 2 study of clofazimine (Oregon Health and Science University) for the treatment of pulmonary mycobacterium avium disease ($1.8 million for 4 years);
phase 2 study of atomoxetine (Vanderbilt University Medical Center) for the treatment of multiple system atrophy ($1.6 million for 4 years);
phase 2 study of furosemide (University of North Carolina, Chapel Hill) for the prevention of bronchopulmonary dysplasia in premature infants ($1.4 million for 4 years);
phase 2 study of CCX168 (Chemocentryx Inc.) for the treatment of anti-neutrophil cytoplasmic auto-antibodies associated vasculitis ($500,000 for 1 year);
phase 2 study of abatacept combined with calcineurin inhibition and methotrexate (Seattle Children’s Research Institute) for prophylaxis of graft-versus-host disease ($99,630 for 1 year);
phase 1/2 study of ONC201 (Oncoceutics Inc.) for the treatment of multiple myeloma ($1.7 million for 4 years);
phase 1/2 study of glycerol phenylbutyrate (Johns Hopkins University) for the treatment of cystic fibrosis ($750,000 for 3 years);
phase 1 study of PI-3 kinase/BRD4 inhibitor SF1126 (University of California, San Diego) for the treatment of hepatocellular carcinoma ($750,000 for 3 years);
phase 1 study of humanized 3F8 MoAb and natural killer cells (Sloan-Kettering Institute Cancer Research) for the treatment of neuroblastoma ($750,000 for 3 years);
phase 1 study of HSV G207 combined with radiation (University of Alabama) for the treatment of pediatric brain tumors ($750,000 for 3 years);
phase 1 study of viralym-A (Wilson Wolf Manufacturing Corp.) for the treatment of adenovirus disease ($750,000 for 3 years);
phase 1 study of omigapil (Santhera Pharmaceuticals) for the treatment of congenital muscular dystrophy ($246,000 for 1 year); and
phase 1 study of CC100 (Chemigen LLC) for the treatment of amyotrophic lateral sclerosis ($243,000 for 1 year).

Gayatri R. Rao

“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,” Gayatri R. Rao, MD, JD, director of the Office of Orphan Product Development at the FDA, said in the release. “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.”

Reference:

www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm525468.htm