FDA Awards $25M in Grants to Clinical Trials Studying Rare Diseases

FDA recently awarded 11 new clinical trial research grants, totaling $25 million over the next four years to support the development of medical products for patients with rare diseases. 

The FDA’s Congressionally-funded Orphan Products Grants Program (OOPD) awards the funding to clinical investigators to support studies that address unmet needs of various diseases and conditions or provide improvements in treatment in diagnosis.

The grant also includes a demonstration project that will utilize a collaborative approach to evaluate a tool that can improve data accuracy for trials taking place at multiple locations. 

“Supporting the development and evaluation of new treatments for rare diseases is a critical part of the FDA’s mission, “Acting FDA Commissioner Janet Woodcock, MD, said in the announcement. 

“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,” Woodcock continued.

A majority of the funded studies include newborns and children. One trial is currently evaluating the treatment of a rare inherited skin disease known as recessive dystrophic epidermolysis bullosa (RDEB). This condition can be fatal.

Another study evaluates early treatment before the onset of seizures in infants with tuberous sclerosis complex. An FDA spokesperson explained that this inherited disease can affect multiple organs and result in long-term brain development issues. 

The awards also include clinical studies of products for use in brain cancers, such as one study that will evaluate a novel peptide vaccine to treat pediatric brain cancers.

The vaccine is designed to target areas of tumor in the brain and has the potential to substantially impact the treatment of these rare and deadly tumors. 

Other studies that received funding include a Phase 1 study of a drug to treat high-grade astrocytoma, a Phase 2 study of stem cells to treat multiple system atrophy, and a Phase 2a study of vamorolone to treat Becker muscular dystrophy. 

“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, 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,” Retzky continued.