FDA funds $14m worth of rare disease R&D grants

The US Food and Drug Administration has awarded US $14 million worth of grants to boost the development of products for patients with rare diseases.

In the US, a disease is considered rare if it affects less than 200,000 people. In 1983, the Orphan Drug Act was passed to stimulate the development of rare disease drugs. To continue promoting this development, the Orphan Products Grants Program was created. This program enabled a panel of outside experts with experience in the disease-related fields to review applications for the grants and chose the following recipients:

• Leonide Saad, Alkeus Pharmaceuticals, Inc., phase 1 Study of ALK001 for the Treatment of Stargardt Disease — about $167,000 for one year

• Parinda Mehta, Children's Hospital Medical Center Cincinnati, phase 1 Study of Quercetin for the Treatment of Fanconi Anemia — $600,000 over three years

• Diva De Leon, Children's Hospital of Philadelphia, phase 2A Study of Exendin for the Treatment of Congenital Hyperinsulinism — about $759,000 over three years

• Soma Jyonouchi, Children's Hospital of Philadelphia, phase 1 Study of IL-2 for the Treatment of Wiskott-Aldrich Syndrome — $600,000 over three years

• Dwight Koeberl, Duke University, phase 1 / 2 Study of Clenbuterol for the Treatment of Pompe Disease — $400,000 over two years

• Leslie Kean, Emory University, phase 2 Study of Abatacept combined with Calcineurin Inhibition and Methotrexate for Prophylaxis of Graft versus Host Disease — about $1.6 million over four years

• Todd Levine, PNA Center for Neurological Research, phase 2B Study of Memantine for the Treatment of Amyotrophic Lateral Sclerosis — about $990,000 over three years

• Joseph Reynolds, Spineform LLC, phase 2 Study of the HemiBridge System for the Treatment of Idiopathic Scoliosis — $1.6 million over four years

• Jonathan Davis, Tufts Medical Center, phase 2 Study of rhCC10 to Prevent Neonatal Bronchopulmonary Dysplasia — about $1.6 million over four years

• Glenn Furuta, University of Colorado Denver, phase 2 Study of Esophageal String Test in Diagnosing Eosinophilic Esophagitis — about $400,000 over two years

• Johan Van Hove, University of Colorado Denver, phase 1 / 2 Study of Taurine for the Treatment of Cystathionine Beta-Synthase Deficient Homocystinuria — about $400,000 over two years

• Laurence Cooper, University Of Texas MD Anderson Cancer Center, phase 1 Study of Umbilical Cord Blood Derived CD19 Specific T cell Therapy in the Treatment of Advanced B Cell Malignancies — $600,000 over three years

• Karl Anderson, University of Texas Medical Branch Galveston, phase 2 Study of Hemin for the Treatment and Prevention of Porphyria Attacks — about $1.5 million over four years

• Christopher Goss, University of Washington, phase 2 Study of Intravenous Gallium Nitrate for the Treatment of Cystic Fibrosis — about $1.6 million over four years

• Michael Debaun, Vanderbilt University, phase 2 Study of Montelukast for the Treatment of Sickle Cell Anemia — about $1.59 million over four years

"The FDA is committed to fostering and encouraging the development of products for rare diseases, most of which have no available or adequate treatments. The grants awarded this year support studies in very vulnerable, difficult-to-treat populations who have no available options."

Gayatri R. Rao, M.D., J.D., director of the FDA's Office of Orphan Product Development.

Since the inception of the Orphan Products Grants Program, more than $300 million has been awarded to fund over 530 new clinical studies developing treatments for rare diseases and has been used to bring 50 products to marketing approval.