FDA program awards $14M for rare disease research

In Clinical Development, Partnering & Investment, Regulation & Government by Tim Peplow

fda rare disease research (flickr NIAID)

The FDA has announced that its Orphan Products Grants Program has awarded a new round of grants to support research into the treatment of rare diseases.

The 15 grants, which total $14 million, will fund the research of treatments for diseases such as Stargardt disease, a rare form of macular degeneration, and Pompe disease, a lysosomal storage disorder.

“The FDA is committed to fostering and encouraging the development of products for rare diseases, most of which have no available or adequate treatments,” said Gayatri R. Rao, M.D., J.D., director of the FDA’s Office of Orphan Product Development. “The grants awarded this year support studies in very vulnerable, difficult-to-treat populations who have no available options.”

So far, the program has awarded over $300 million to fund more than 530 clinical studies. 50 such products have been brought to marketing approval.

Read the press release >

Next: 5 lysosomal storage diseases with orphan drugs in development

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