SILVER SPRING, Md. , Oct. 1, 2024 /PRNewswire/ -- Today, the U.
S. Food and Drug Administration is providing an at-a-glance summary of news from around the agency: Today, the FDA announced seven new clinical trial grants awarded in fiscal year (FY) 2024 under the Orphan Products Grants Program for clinical trials to address the significant and often unmet needs of people affected by rare diseases. In total, clinical researchers will receive approximately $17.
2 million over the next four years. Newly funded projects include: a treatment for Cushing's syndrome—a disorder caused by pituitary tumors; two treatments for blood disorders; two cancer treatments; a treatment for lymphatic malformations; and a gene therapy for an inherited eye disease that causes blindness. The FDA also awarded three new natural history study grants under the Orphan Products Grants Program, providing approximately $4.
7 million over four years to advance the understanding of how specific rare diseases progress over time. This year's funded projects include those studying: intestinal failure in children; an inherited polycystic kidney disease; and congenital central hypoventilation syndrome—a rare neurological disorder that affects normal breathing, especially during sleep. "There is little knowledge on the progression of many rare diseases, which makes medical product development challenging.
These new natural history studies will help address knowledge gaps in support of future clinical trials," sai.