FDA Roundup: October 1, 2024

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,” said Sandra Retzky, D.O., J.D., MPH, director of the FDA’s Office of Orphan Products Development. “In addition, the new clinical trial awards will support innovative and efficient trial designs that can be used to advance treatments through product development and as models for future drug development in rare diseases.”

• Today, the FDA announced over $5.4 million in funding to support clinical research under the Rare Neurodegenerative Disease (RNDD) Grants Program in FY 2024. The FDA received 15 applications and was able to fund one new grant for $1.3 million annually over four years—approximately $5 million in total—to support development of retinal imaging biomarkers for individuals affected with CADASIL (cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy). FDA also provided an additional $4.1 million in FY 2024 to nine ongoing studies to support ALS and other rare neurodegenerative diseases including: Niemann-Pick disease, type C; myotonic dystrophy, type 1; and familial dysautonomia. The $5.4 million FY 2024 budget for the RNDD grant program equally funded studies to support development of treatments for ALS and other rare neurodegenerative diseases.  

  “Neurodegenerative diseases occur when nerve cells in the brain or peripheral nervous system lose function over time and ultimately die. In general, they are incurable, debilitating, and progressive conditions,” said Sandra Retzky, D.O., J.D., MPH, director of the FDA’s Office of Orphan Products Development. “The FDA remains committed to supporting medical product development for all rare neurodegenerative diseases.” 

  The FDA has issued a new funding opportunity for research in rare neurodegenerative diseases for FY 2025 to continue to advance these goals.

• On Friday, the FDA authorized for marketing Pi-Cardia Ltd.’s ShortCut, a device intended to prepare previously placed bioprosthetic aortic valves that have failed in certain patients who are at risk of coronary obstruction. A bioprosthetic aortic valve may fail over time due to stenosis (narrowing of the valve that causes the heart to work harder to pump blood), regurgitation (when the valve does not close completely and blood leaks backwards), or a combination. Valve-in-valve procedures are an alternative to repeat surgery and avoids open heart surgery. While valve-in-valve replacement is an effective treatment for patients with failed bioprosthetic aortic valves, the transcatheter aortic valve may increase risk of coronary obstruction, a rare complication in which blood flow to the heart is blocked. The ShortCut received a Breakthrough Device designation as it is a first-of-a-kind valve leaflet splitting device that may reduce the risk of coronary artery obstruction.
• On Friday, the FDA updated At-Home OTC COVID-19 Diagnostic Tests to share that U.S. households are eligible to order four free COVID-19 tests at www.COVIDTests.gov. The COVID-19 tests available through this program are intended to detect a number of current COVID-19 variants and can be ordered through the end of the year. Many COVID-19 tests have extended expiration dates, so you may be able to use your COVID-19 tests after the expiration date that is printed on the box. To find out if your test has an extended expiration date, check the list of extended expiration dates.

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