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

The U.S. Food and Drug Administration has awarded 11 new clinical trial research grants totaling more than $25 million under its Orphan Products Grants Program, to support the development of medical products for patients with rare diseases.

Photo: Janet Woodcock, acting FDA Commissioner

“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. “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, director of OOPD.

The grants are awarded to:

Eugene Marcantonio of Armgo Pharma for a phase 2 study of S48168 (ARM210) for the treatment of catecholaminergic polymorphic ventricular tachycardia type 1 (CPVT1); $1 million over two years

Mark Puder of Boston Children’s Hospital for a phase 3 study of RELiZORB for the treatment of short bowel syndrome; $2.7 million over four years

Mary Spellman of Castle Creek Biosciences for a 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

Darcy Krueger of Cincinnati Children’s Hospital Medical Center for a phase 2b study of sirolimus for the prevention of epilepsy in patients with tuberous sclerosis complex; $5 million over four years

Michael Jordan of Cincinnati Children’s Hospital Medical Center for a phase 2 study of abatacept for the treatment of common variable immunodeficiency with interstitial lung disease (ABCVILD); $3.1 million over four years

Eric Thompson of Duke University for a 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

Amy Dickey of Massachusetts General Hospital for a phase 2 study of oral cimetidine for the treatment of protoporphyrias; $1.6 million over four years

Sani Kizilbash of the Mayo Clinic Rochester for a phase 1 study of WSD0922-FU for the treatment of high-grade astrocytoma; $1 million over three years

Wolfgang Singer of the Mayo Clinic Rochester for a phase 2 study of intrathecally administered autologous mesenchymal stem cells for the treatment of multiple system atrophy; $3.2 million over four years

Eric Hoffman of Reveragen Biopharma for a phase 2a study of vamorolone for the treatment of becker muscular dystrophy; $1.2 million over two years

Peter Stacpoole of the University of Florida for a 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 said it 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.

Author: Rare Daily Staff