FDA Awards 11 Grants to Clinical Trials to Develop New Medical Products for Rare Disease Treatments

FDA News Release

• For Immediate Release: October 14, 2021

Today, the U.S. Food and Drug Administration announced it has awarded 11 new clinical trial research grants, equaling more than $25 million of funding over the next four years. The FDA’s Congressionally-funded Orphan Products Grants Program awards these grants to clinical investigators 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 Acting FDA Commissioner Janet Woodcock, M.D. “These grants are the latest examples of the FDA’s ongoing commitment to help meet the future and current health needs of those who suffer from a rare disease.”

The grant awards support clinical studies of products that address unmet needs in rare diseases or conditions or provide highly 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 painful blisters and wounds that are often disfiguring and fatal. Another study seeks to evaluate early treatment before the onset of seizures in infants with tuberous sclerosis complex, which is an inherited disease that can affect a variety of organs and can result in long term brain development issues. This grant also includes an innovative demonstration project that will utilize a collaborative approach to evaluate a tool with the potential to improve data accuracy for clinical trials taking place at more than one location.

Some of the new awards fund clinical studies of products for use in brain cancers. Specifically, one will evaluate a novel peptide vaccine to treat pediatric brain cancers. The vaccine is designed to be directed specifically to areas of tumor in the brain and has the potential to significantly impact the way these rare and deadly tumors are treated.

“The Office of Orphan Products Development works to identify, examine and ultimately fill the gaps that exist within the rare disease drug development community by funding necessary and revolutionary clinical studies to determine the safety and efficacy of potential treatment options,” said Sandra Retzky, D.O., J.D., MPH, director of OOPD. “These grants demonstrate the FDA’s commitment to supporting the development of new treatments for patients living with rare diseases.”

Below is a complete list of the 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; Phase 3 study of RELiZORB for the treatment of short bowel syndrome; $2.7 million over four years
• Castle Creek Biosciences, LLC (Exton, Pennsylvania); 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 complex; $5 million over four years
• Cincinnati Children’s Hospital Medical Center; Michael Jordan; Phase 2 study of abatacept for the treatment of common variable immunodeficiency with interstitial lung disease (ABCVILD); $3.1 million over four years
• Duke University (Durham, North Carolina); Eric Thompson; Phase 2 study of peptide vaccine targeting CMV antigen for the treatment of newly diagnosed pediatric high-grade glioma and 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 protoporphyrias; $1.6 million over four years
• Mayo Clinic Rochester (Rochester, Minnesota); Sani Kizilbash; Phase 1 study of WSD0922-FU for the treatment of high-grade astrocytoma; $1 million over three years
• Mayo Clinic Rochester (Rochester, Minnesota); Wolfgang Singer; Phase 2 study of intrathecally administered autologous mesenchymal stem cells for the treatment of multiple system atrophy; $3.2 million over four years
• Reveragen Biopharma, Inc.; Eric Hoffman; Phase 2a study of vamorolone for the treatment of becker 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 increased costs continued this year for clinical trials due to the COVID-19 pandemic, the FDA remains committed to supporting rare disease research by providing existing grantees with additional funding. These resources allow ongoing studies to implement necessary steps to allow their research to continue and assure the safety of study participants, maintain compliance with good clinical practice and minimize risks to trial integrity.