FDA awards 21 grants focusing on the treatment of rare diseases

The U.S. Food and Drug Administration (FDA) has awarded 21 clinical trail grants, totaling more than $23 million, for the advancement of products for patients with rare diseases. These grants were given to lead academic investigators and industries with both domestic and international research clinics.

The grants, distributed through the Orphan Products Clinical Trials Grants Program, are directly funding research into the development of drugs, biologics, medical devices and medical foods for the treatment of rare diseases. The FDA hopes to provide more approved products to patients. Just last year, five of the studies that received similar grants received approval for products to treat neuroblastoma, lymphangioleiomyomatosis, hypoparathyroidism and hypophosphatasia.

“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 R. Rao, MD, JD, director of the FDA’s Office of Orphan Product Development, within the Office of Special Medical Programs. “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.”

The statistics of studies that received grants include:

• 24 percent of grants toward studies on patients with cancer.
• 40 percent went to studies focusing on deadly brain tumors, one of which tests children with recurrent malignant brain tumors.
• 43 percent funded studies enrolling pediatric patients as young as newborns, two of which focus on transplantation.
• One study on the testing of medical devices for a fully implantable neuroprosthesis to grasp, reach and improve trunk function in patients with spinal cord injury.