FDA hands out $23M for rare disease development

The FDA says it has granted $23 million in a siloed fund for industry and academic groups to help bolster early- and later-stage research into a host of rare diseases.

The U.S. regulator, which awards the grants through its Orphan Products Clinical Trials Grants Program--a program that has been around for more than three decades--says its investment is designed to “encourage clinical development of drugs, biologics, medical devices, or medical foods for use in rare diseases.”

Its latest cash injection, which will be used over the next four years, could help development across 21 rare disease areas.


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In total, 68 companies and groups applied for a grant, with 21 getting part of the cash pot. This includes biotechs Chemigen, which has been given around quarter of a million dollars toward its ALS Phase I, as well as $1.7 million over four years for Oncoceutics’ blood cancer treatments, and Scioderm, which was given half a million dollars for a late-stage study of rare skin disorder epidermolysis bullosa.

About one-quarter of the new grant awards fund studies enrolling patients with a form of cancer, while 40% are for forms of brain cancer. Meanwhile, just under half (43%) of this year’s awards fund studies that enroll pediatric patients as young as newborns.

“We are proud of our 30-year track record of fostering and encouraging the development of safe and effective therapies for rare diseases through our clinical trials grant program,” said Gayatri Rao, director of the FDA’s Office of Orphan Product Development. “The grants awarded this year will support much-needed research in 21 different rare diseases, many of which have little, or no, available treatment options.” The FDA’s program has handed out around $370 million since its inception in 1983. 

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