FDA supports rare disease research with $19m fund

The FDA has awarded $19 million in funding to help accelerate the development of medical device, drug, and biological products for patients with rare diseases.

The US regulator has awarded grants to 15 different projects, with at least a quarter of the funding going to studies focused on paediatrics.The grants cover a range of conditions, including a range of cancers, TB, sickle cell disease and cystic fibrosis.

The FDA awards grants for clinical studies on safety and/or effectiveness of products that could either result in, or substantially contribute to, approval of the products.

“The FDA is in a unique position to help those who suffer from rare diseases by offering several important incentives to promote the development of products for rare diseases, one of which is this grants programme,” said Gayatri R. Rao, M.D., director of the FDA’s Office of Orphan Product Development. “The grants awarded this year support much-needed research in difficult-to-treat diseases that have little, or no, available treatment options.”

The grants have been administered through the FDA’s Orphan Products Grants Programme. This was created by the Orphan Drug Act, passed in 1983, to promote the development of products for rare diseases. Since its inception, the programme has given more than $330 million to fund more than 530 new clinical studies on developing treatments for rare diseases and has been used to bring more than 50 products to marketing approval.

A panel of independent experts with experience in the disease-related fields reviewed the grant applications and made recommendations to the FDA.

The 2014 grant recipients

Denise Adams, Cincinnati Children’s Hospital Medical Center (Cincinnati, Ohio), Phase 2 Study of Vincristine vs. Sirolimus for the Treatment of High Risk Kaposiform Hemangioendothelioma—$1.6 million over four years

Mitesh Borad, Mayo Clinic Arizona (Scottsdale, Ariz.), Phase 1 Study of VSV-hIFN-B for the Treatment of Hepatocellular Carcinoma— approximately $600,000 over three years

Andrew Brenner, University of Texas Health Center San Antonio (San Antonio, Texas), Phase 2 Study of TH-302 for the Treatment of Glioblastoma— approximately $1.6 million over four years

Kelly Dooley, The Johns Hopkins University (Baltimore, Md.), Phase 2 Study of PA-824 for the Treatment of Pulmonary Tuberculosis— $1.6 million over four years

Donald Durden, University of California San Diego (San Diego, Calif.), Phase 2 Study of Poly-ICLC for the Treatment of Pediatric Low Grade Gliomas— $1.6 million over four years

Alfred Lane, Stanford University (Stanford, Calif.), Phase 2 Study of Sildenafil for the Treatment of Lymphatic Malformations— approximately $1.6 million over four years

Dung Le, The Johns Hopkins University (Baltimore, Md.), Phase 2 Study of Folfirinox followed by Ipilimumab/GVAX for the Treatment of Pancreatic Cancer— $1.6 million over four years

Phillip Low, Mayo Clinic Rochester (Rochester, Minn.), Phase 1 Study of Intrathecal Autologous Mesenchymal Stem Cell Therapy for the Treatment of Multiple System Atrophy—$600,000 over three years

Guido Magni, River Vision Development Corporation (New York, N.Y.), Phase 2 Study of RV001 for the Treatment of Thyroid Eye Disease—$1.2 million over three years

Michael Portman, Seattle Children’s Hospital (Seattle, Wash.), Phase 3 Study of Triiodothyronine Supplementation for the Treatment of Young Infants After Cardiopulmonary Bypass— approximately $1.6 million over four years

Jana Portnow, City of Hope Beckman Research Institute (Duarte, Calif.), Phase 1 Study of Neural Stem Cells & 5-FC/Leucovorin for the Treatment of Recurrent High Grade Gliomas—$600,000 over three years

Scott Rollins, Selexys Pharmaceuticals Corporation (Oklahoma City, Okla.), Phase 2 Study of SelG1 for the Treatment of Sickle Cell Disease— $1.6 million over four years

Beena Sood, Wayne State University (Detroit, Mich.), Phase 1/2 Study of Aerosolized Survanta for the Treatment of Neonatal Respiratory Distress Syndrome— approximately $1.4 million over four years

Warren Stern, Aesrx, LLC (Newton, Mass.), Phase 2 Study of Aes103 (5-HMF) for the Treatment of Stable Sickle Cell Disease—approximately $1.6 million over four years

Pamela Zeitlin, The Johns Hopkins University (Baltimore, Md.), Phase 2 Study of Digitoxin for the Treatment of Cystic Fibrosis— approximately $290,000 for one year

For the grants programme therapies, a disease or condition is considered rare if it affects less than 200,000 persons in the United States. There are about 7,000 rare diseases and conditions, according to the National Institutes of Health. In total, nearly 30 million Americans suffer from at least one rare disease.

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