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

PMEA Awards 2020

COVID-19 Updates and Daily News

Featured jobs

PMHub

Add my company
Streaming Well

Streaming Well is a healthcare-focused, award-winning video production company which operates in the US and Europe. We create engaging visual...

Latest intelligence

EU health fund
An EU emergency health fund to limit the impact of future crises
Leading finance experts propose an Emergency Health Financing Facility to limit the impact of future crises on the EU...
Mistrust in medical research: a patient perspective
The recent development of several COVID-19 vaccines has placed medical research firmly in the spotlight, highlighting public confusion and misinformation about clinical trials. Patient advocate, Trishna Bharadia reveals what the...
Real-world evidence: breaking boundaries in rare disease
Generating data for drug launches is a challenging process. In rare diseases, with small patient populations and high unmet need, evidence generation is even more complex. Consultant Sarah Poole and...

Infographics