FDA Announces $14 Million in Grants for Trials in Support of Rare Disease Therapies

| 21 October 2013 |  By 

The US Food and Drug Administration (FDA) is often seen as a reactive entity-reacting, that is, to the information contained within applications in order to render an approval decision, or to postmarketing adverse event data to determine if a product remains safe for use.

But as it illustrated in an announcement made on 21 October 2013, it can also be proactive when it needs to be.

FDA said it will be issuing 21 grants through its Orphan Products Grants Program with the intent of stimulating the development of treatments for rare diseases and conditions. In the US, the term "rare disease" is defined under the 1984 Orphan Drug Act (ODA), and refers to any disease for which there are fewer than 200,000 patients.

FDA said there are approximately 6,800 diseases currently known to science, cumulatively affecting nearly one in 10 Americans.

The grants are intended to turbo-charge early-stage research, some of which can struggle to find funding given the miniscule size of the eventual market or the presence of other low-cost competitors. Of the 21 grants listed by FDA as receiving funding, three are in phase 1 and 11 are in Phase II. None have yet reached Phase III, which are traditionally some of the most expensive to conduct. Typically government agencies will license research that successfully reaches Phase III studies to industry for further development.

FDA explained that it selected the diseases it did because they currently have "no available options" and represent "very vulnerable, difficult-to-treat populations."

The $14 million in funding ranges from a one-year, $167,000 contract to as much as $1.59 million spread out over four years. The agency's Orphan Products Grants Program has dispensed more than $300 million to more than 530 clinical studies and brought 50 products to market since its inception, FDA said.

The studies being funded this year are as follows:

  • Phase 2 Study of Intravenous Gallium Nitrate for the Treatment of Cystic Fibrosis-about $1.6 million over four years
  • Phase 2 Study of Montelukast for the Treatment of Sickle Cell Anemia-about $1.59 million over four years
  • Phase 2B Study of Memantine for the Treatment of Amyotrophic Lateral Sclerosis-about $990,000 over three years
  • 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
  • Phase 2 Study of the HemiBridge System for the Treatment of Idiopathic Scoliosis-$1.6 million over four years
  • Phase 2 Study of rhCC10 to Prevent Neonatal Bronchopulmonary Dysplasia-about $1.6 million over four years
  • Phase 2 Study of Esophageal String Test in Diagnosing Eosinophilic Esophagitis-about $400,000 over two years
  • Phase 2 Study of Hemin for the Treatment and Prevention of Porphyria Attacks-about $1.5 million over four years
  • Phase 2A Study of Exendin for the Treatment of Congenital Hyperinsulinism-about $759,000 over three years
  • Phase 1/2 Study of Clenbuterol for the Treatment of Pompe Disease-$400,000 over two years
  • Phase 1/2 Study of Taurine for the Treatment of Cystathionine Beta-Synthase Deficient Homocystinuria-about $400,000 over two years
  • Phase 1 Study of ALK001 for the Treatment of Stargardt Disease-about $167,000 for one year 
  • Phase 1 Study of Quercetin for the Treatment of Fanconi Anemia-$600,000 over three years
  • Phase 1 Study of IL-2 for the Treatment of Wiskott-Aldrich Syndrome-$600,000 over three years
  • 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

FDA Funding Announcement


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