USA- The FDA has funded clinical trials for the development of rare disease treatments with 11 grants totaling approximately US$25 million over the next four years.

The FDA’s Congressionally funded Orphan Products Grants Program provides grants to clinical investigators to aid in the development of medical products for patients suffering from rare diseases.

The grants fund clinical trials of products that address unmet needs in rare diseases or conditions or provide highly significant advances in treatment or diagnosis.

Many of these studies involve children as young as newborns, including one examining the treatment of recessive dystrophic epidermolysis bullosa, or RDEB, a rare inherited skin disease.

Another study aims to assess early treatment before the onset of seizures in infants with tuberous sclerosis complex, an inherited disease that can affect a variety of organs and result in long-term brain development issues.

The grant also includes an innovative demonstration project that will use a collaborative approach to evaluate a tool that has the potential to improve data accuracy for clinical trials that take place in multiple locations.

“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.

Some of the new awards will go toward clinical trials of products for use in brain cancer. One will specifically evaluate a novel peptide vaccine to treat pediatric brain cancers that is designed to be directed specifically to tumor areas in the brain.

The FDA believes it has the potential to significantly impact the treatment of these rare and deadly tumors.

The Office of Orphan Products Development (OOPD) 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,” commented Sandra Retzky, director of OOPD.

“These grants demonstrate the FDA’s commitment to supporting the development of new treatments for patients living with rare diseases.”

Some of the studies funded include:

Armgo Pharma’s Phase II study of S48168 (ARM210) for the treatment of catecholaminergic polymorphic ventricular tachycardia type 1 (CPVT1),

Boston Children’s Hospital’s Phase III study of RELiZORB for the treatment of short bowel syndrome,

Cincinnati Children’s Hospital Medical Center’s Phase IIb study of sirolimus for the prevention of epilepsy in patients with tuberous sclerosis complex,

Mayo Clinic Rochester’s Phase II study of intrathecally administered autologous mesenchymal stem cells for the treatment of multiple system atrophy.

The FDA has remained committed to supporting rare disease research by providing additional funding to existing grantees and these resources will enable ongoing studies to take the necessary steps to ensure the safety of study participants, maintain compliance with good clinical practice, and minimize risks to trial integrity.

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