FDA awards $19m in rare disease grants

The US FDA is to encourage research in rare diseases through 15 grants totalling more than $19m.

The regulator said the grants will support clinical studies involving medical devices, drugs and biological products that relate to diseases that affect fewer than 200,000 people in the US. At least a quarter of the funding will go to studies focused solely on diseases in children and young people.

“The FDA is in a unique position to help those who suffer from rare diseases by offering several important incentives to promote the development of products for rare diseases, one of which is this grants program,” said Dr Gayatri Rao, director of the FDA's Office of Orphan Product Development.

“The grants awarded this year support much-needed research in difficult-to-treat diseases that have little, or no, available treatment options.”

Eight studies will each received the largest grants of $1.6m. These include a study by Cincinnati Children's Hospital Medical Center to investigate a new treatment for kaposiform hemangioendothelioma, a rare tumour in the cell of a blood vessel that resembles a birthmark.

Other studies to receive the top grants include one by the University of Texas Health Center San Antonio to research a bran tumour treatment and another by Johns Hopkins University involving a new drug for pulmonary tuberculosis.

One study – to investigate neonatal respiratory distress syndrome – received $1.4m; one involving thyroid eye disease received $1.2m; and three more received less than $1m.

The awards are part of the FDA's Orphan Products Grants Programme created by the Orphan Drug Act, which was brought in to promote the development of products for rare diseases.

Since its inception, the programme has given more than $330m to fund more than 530 new clinical studies to develop new treatments for rare diseases. Of these more than 50 products have been brought to marketing approval.

Last year the FDA awarded $14m across 15 orphan disease projects.

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