Please login to the form below

Not currently logged in
Email:
Password:

FDA awards $19m in rare disease grants

US regulator supports 15 orphan drug projects
FDA headquarters White Oak

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.

Article by
Thomas Meek

1st October 2014

From: Research, Regulatory

Share

Tags

Featured jobs

Subscribe to our email news alerts

PMHub

Add my company
Porterhouse Medical Group

The Porterhouse Medical Group provides market research, insights, and scientific and medical communication services to the pharmaceutical industry across the...

Latest intelligence

The relevance of patient perspectives to value
Exploring the evolution of patient involvement in health technology assessment, and the role of the patient voice in market access....
How is the NHS Long-term Plan being put into action?
Steve How, Paul Midgley and Oli Hudson, of Wilmington Healthcare, explore some of the changes that have occurred since the plan was published...
real world studies
The evolution of real-world studies
How industry and regulators are using real-world data...

Infographics