FDA Awards 12 Grants Towards Treatment of Rare Diseases


The grants were awarded through the Orphan Products Clinical Trials Grants Program, which is funded by Congress.

The FDA has awarded 12 new clinical trial research grants to enhance the development of medical products for patients with rare diseases.

The grants were awarded through Orphan Products Clinical Trials Grants Program, which is funded by Congress. This program encourages clinical development of drugs, biologics, medical devices, and medical foods for the treatment of rare diseases.

The FDA received 89 clinical trial grant applications that were reviewed and evaluated by more than 100 rare disease experts.

Each grant is intended to contribute to marketing approval of products to treat rare diseases or provide essential data needed for development of such products. They are focused on supporting product development to meet the needs of patients impacted by a variety of rare diseases. Some of the diseases that will be targeted include Duchenne muscular dystrophy, sickle cell disease, and Fanconi anemia.

Past studies that were funded by this program have made it to product approval, such as: fish oil triglycerides, an =FDA-approved lipid emulsion made from fish oil, and the first treatment for heart disease caused by transthyretin mediated amyloidosis.


FDA awards 12 grants to fund new clinical trials to advance the development of medical products for the treatment of rare diseases [news release]. Silver Spring, MD; FDA Newsroom: October 8, 2019. https://www.fda.gov/news-events/press-announcements/fda-awards-12-grants-fund-new-clinical-trials-advance-development-medical-products-treatment-rare-0. Accessed October 8, 2019.

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