FDA Distributes $23M Grant for Rare Disease R&D Initiatives

A variety of rare disease research programs received a financial boost on Monday.

The Food and Drug Administration (FDA) announced it would distribute a grant worth more than $23 million through the Orphan Products Clinical Trials Grants program. The funding will be allocated to 21 finalists, a mix of academic institutions and private companies, over a four year period to help with clinical development for their respective rare disease research initiatives.

“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, within the Office of Special Medical Programs in a statement.

“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,” continued Dr. Rao.

Some of the beneficiaries include biotech firms Chemigen and Scioderm, reported FiercePharma. The former will use about a quarter of a million dollars to launch a Phase 1 study of its Amytrophic Lateral Sclerosis candidate whereas the former plans on using an estimated half a million dollars to advance a late-stage study of its rare skin disorder therapy targeting epidermolysis bullosa.

Other finalists like Johns Hopkins University will be granted $750,000 over three years for research on a cystic fibrosis treatment. The University of Alabama at Birmingham will receive a similar amount for a phase 1 study focusing on an early-stage combination therapy targeting pediatric brain tumors.

This FDA program has provided over $370 million in funding for more than 590 new clinical studies since it was formed in 1983.

You can see who else was on this final list here.