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Today, the United States Food and Drug Administration announced that it has awarded 11 new research grants for clinical trials, equivalent to more than $ 25 million in funding over the next four years. The FDA’s congressionally-funded Orphan Products Grants program awards these grants to clinical researchers to support the development of medical products for patients with rare diseases.

“Supporting the development and evaluation of new treatments for rare diseases is a critical part of the FDA’s mission,” said Janet Woodcock, Acting Commissioner of the FDA. needs of those suffering from a rare disease.

Grants awarded support clinical studies of products that address unmet needs in rare diseases or conditions or that provide very significant improvements in treatment or diagnosis.

Many of these studies involve children, as young as newborns, including one evaluating the treatment of a rare inherited skin disease known as recessive dystrophic epidermolysis bullosa, or RDEB, a condition that can lead to severe painful blisters and sores that are often disfiguring and fatal. Another study aims to assess early treatment before seizures appear in infants with tuberous sclerosis, which is an inherited disease that can affect various organs and lead to long-term brain development problems. This grant also includes an innovative demonstration project that will use a collaborative approach to evaluate a tool with the potential to improve data accuracy for clinical trials taking place in more than one location.

Some of the new awards fund clinical studies of products for use in brain cancer. More specifically, we will evaluate a new peptide vaccine to treat pediatric brain cancers. The vaccine is designed to be directed specifically at tumor areas of the brain and has the potential to have a significant impact on how these rare and fatal tumors are treated.

“The Orphan Product Development Office strives to identify, review and ultimately fill the gaps that exist within the rare disease drug development community by funding the necessary clinical studies.” and groundbreaking in determining the safety and effectiveness of potential treatment options, ”said Sandra Retzky, DO, JD, MPH, Director of OOPD. “These grants demonstrate the FDA’s commitment to supporting the development of new treatments for patients living with rare diseases.

Here is a complete list of grants in alphabetical order:

  • Armgo Pharma, INC. (Ardsley, New York); Eugene Marcantonio; Phase 2 study of S48168 (ARM210) for the treatment of catecholaminergic polymorphic ventricular tachycardia type 1 (CPVT1); $ 1 million over two years
  • Boston Children’s Hospital (Boston, Massachusetts); Mark Puder; RELiZORB Phase 3 Study for the Treatment of Short Bowel Syndrome; $ 2.7 million over four years
  • Castle Creek Biosciences, LLC (Exton, Pa); Mary Spellman; Phase 3 study of FCX-007 (genetically modified autologous human dermal fibroblasts) for the treatment of recessive dystrophic epidermolysis bullosa; $ 1.8 million over four years
  • Cincinnati Children’s Hospital Medical Center (Cincinnati, Ohio); Darcy Krueger; Phase 2b study of sirolimus for the prevention of epilepsy in patients with tuberous sclerosis Bourneville; $ 5 million over four years
  • Cincinnati Children’s Hospital Medical Center; Michael Jordan; Phase 2 study of abatacept for the treatment of common variable immune deficiency associated with interstitial lung disease (ABCVILD); $ 3.1 million over four years
  • Duke University (Durham, North Carolina); Eric Thompson; Phase 2 study of a peptide vaccine targeting CMV antigen for the treatment of newly diagnosed high-grade pediatric glioma, diffuse intrinsic pontine glioma and recurrent medulloblastoma; $ 1.8 million over four years
  • Massachusetts General Hospital (Boston, Massachusetts); Amy Dickey; Phase 2 study of oral cimetidine for the treatment of protoporphyria; $ 1.6 million over four years
  • Mayo Rochester Clinic (Rochester, Minnesota); Sani Kizilbash; Phase 1 study of WSD0922-FU for the treatment of high-grade astrocytoma; $ 1 million over three years
  • Mayo Rochester Clinic (Rochester, Minnesota); singer Wolfgang; Phase 2 study of autologous mesenchymal stem cells administered intrathecally for the treatment of multisystem atrophy; $ 3.2 million over four years
  • Reveragen Biopharma, Inc .; Eric Hoffman; Phase 2a study of vamorolone for the treatment of Becker’s muscular dystrophy; $ 1.2 million over two years
  • University of Florida (Gainesville, Florida); Peter Stacpoole; Phase 2A trial of dichloroacetate for the treatment of glioblastoma multiforme; $ 2.5 million over four years

As challenges and rising costs have continued this year for clinical trials due to the COVID-19 pandemic, the FDA remains committed to supporting rare disease research by providing existing recipients with additional funding. These resources allow ongoing studies to take the necessary steps to allow their research to continue and to ensure the safety of study participants, maintain compliance with good clinical practice, and minimize risk to the patient. integrity of tests.

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The FDA, an agency of the U.S. Department of Health and Human Services, protects public health by ensuring the safety, efficacy, and safety of drugs, vaccines, and other biologicals for human and veterinary use, as well as medical devices. . The agency is also responsible for the safety and security of our country’s food supply, cosmetics, dietary supplements, products that emit electronic radiation, and the regulation of tobacco products.