FDA Boosts Rare Disease Research with $15 Million in Grants – BioSpace

Posted: Published on October 15th, 2019

This post was added by Alex Diaz-Granados

The U.S. Food and Drug Administration (FDA) awarded $15 million in grants to fund 12 new clinical trials over the next four years to foster the development of rare disease treatments.

The grants were awarded through the FDAs Orphan Products Clinical Trials Grants Program that was provided by Congress to specifically encourage the development of treatments for rare diseases. The grants are intended to substantially contribute to the marketing approval of products to treat rare diseases or provide essential data needed for the development of such products. The FDA selected the 12 recipients out of 89 different applications that were evaluated by more than 100 rare disease experts, the agency said.

FDA Principal Deputy Commissioner Amy Abernethy said the regulatory agency has provided much-needed financial support to clinical trials for rare disease treatments for more than three decades. Over that time, the more than $400 million provided by these grants has led to the approval of more than 60 different drugs for rare diseases, she said.

We are encouraged by the amount of interest we continue to have in the grants program and are committed to working with researchers and industry to facilitate and support the study and development of treatments for patients with rare diseases, Abernathy said in a statement.

The 12 grant awards are primarily focused on discovering treatments for rare diseases affecting children, with about two-thirds looking at rare cancers, including a type of brain cancer. More than three-fourths of the trials will enroll children, including infants, the FDA said. Some of these diseases include Duchenne Muscular Dystrophy, sickle cell disease and Fanconi Anemia, a rare inherited condition that can result in bone marrow failure and has a high risk for squamous cell cancers. Another of the funded studies is evaluating a novel drug delivery system that delivers chemotherapy on a sustained basis directly to the eye to treat retinoblastoma, a rare cancer in the eye most commonly affecting young children.

Janet Maynard, director of the FDAs Office of Orphan Products Development, said the majority of rare diseases do not have approved therapies and this grant program is aimed at the hope of developing treatments for these diseases.

By helping to spark research, we hope to speed the development of products for rare diseases, and ultimately, make needed treatments available to those patients who need them most, Maynard said.

The awarded grants are:

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FDA Boosts Rare Disease Research with $15 Million in Grants - BioSpace

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