The U.S. Food and Drug Administration, the National Institutes of Health, 10 pharmaceutical companies and five non-profit organizations have partnered to accelerate development of gene therapies for the 30 million Americans who suffer from a rare disease. While there are approximately 7,000 rare diseases, only two heritable diseases currently have FDA-approved gene therapies.

Today, the U.S. Food and Drug Administration took action to expand the use of a booster dose for COVID-19 vaccines in eligible populations. The agency is amending the emergency use authorizations (EUA) for COVID-19 vaccines to allow for the use of a single booster dose as follows:

Today, the U.S. Food and Drug Administration announced it has awarded 11 new clinical trial research grants, equaling more than $25 million of funding over the next four years. The FDA's Congressionally-funded Orphan Products Grants Program awards these grants to clinical investigators to support the development of medical products for patients with rare diseases.

Today, the U.S. Food and Drug Administration approved Rethymic for the treatment of pediatric patients with congenital athymia, a rare immune disorder. Rethymic is the first thymus tissue product approved in the U.S.

"Today's action marks the first FDA approval of a therapy to treat this very rare and devastating disease in children," said Peter Marks, M.D., Ph.D., director of the FDA's Center for Biologics Evaluation and Research.

Today, the U.S. Food and Drug Administration authorized marketing of software to assist medical professionals who examine body tissues (pathologists) in the detection of areas that are suspicious for cancer as an adjunct (supplement) to the review of digitally-scanned slide images from prostate biopsies (tissue removed from the body).

Today, the U.S. Food and Drug Administration announced the reorganization of the agency's information technology (IT), data management and cybersecurity functions into the new Office of Digital Transformation (ODT). The office has been realigned to report directly to the FDA commissioner, elevating the office and its functions to agency-level.

Based on current evidence, there is no urgent need for the administration of booster doses of vaccines to fully vaccinated individuals in the general population, according to a technical report issued by the European Centre for Disease Prevention and Control (ECDC). The report also notes that additional doses should already be considered for people with severely weakened immune systems as part of their primary vaccination.