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," 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.
Additional Resources:
Media Contact: FDA Office of Media Affairs,
301-796-4540
Consumer Inquiries: 888-INFO-FDA
The FDA, an agency within the U.S. Department
of Health and Human Services, protects the public health by
assuring the safety, effectiveness, and security of human and
veterinary drugs, vaccines and other biological products for human
use, and medical devices. The agency also is responsible for the
safety and security of our nation's food supply, cosmetics, dietary
supplements, radiation-emitting electronic products, and for
regulating tobacco products.
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SOURCE U.S. Food and Drug Administration