FDA Biologics

Today, the FDA issued a draft guidance for sponsors seeking approval for targeted individualized therapies by generating substantial evidence of effectiveness and safety when randomized controlled trials are not feasible due to small patient populations. fda.gov/news-events/pr…

To further expedite product development, the FDA announced a more flexible approach to the agency’s oversight of chemistry, manufacturing and control requirements for cell and gene therapies. fda.gov/news-events/pr…

Today, the FDA approved the first cell-based gene therapy for the treatment of Wiskott-Aldrich Syndrome (WAS). fda.gov/news-events/pr…

FDA approved the first hematopoietic stem cell transplant (HSCT) therapy to treat patients with severe aplastic anemia (SAA). Learn more: fda.gov/news-events/pr…

Today, the FDA approved the first Chimeric Antigen Receptor (CAR) T-cell therapy in the U.S. for treatment of adults with marginal zone lymphoma (MZL) who have failed treatment with or relapsed after two or more prior lines of therapy. fda.gov/news-events/pr…

Today, the FDA approved a nerve scaffold for the treatment of sensory nerve discontinuity in adults and pediatric patients aged one month and older. The treatment has also been approved for larger sensory nerve, motor, and mixed nerve discontinuities. fda.gov/news-events/pr…

Step into the forefront of novel endpoint development for rare disease drug development! CBER invites you to submit a proposal to the rare disease endpoint advancement (RDEA) Pilot Program, a collaboration with @FDA_Drug_Info. Deadline is 12/30/25! bit.ly/48UBUx4

Today, the FDA approved a gene therapy for the treatment of spinal muscular atrophy in adult and pediatric patients 2 years of age and older with confirmed mutation in the survival motor neuron 1 gene. fda.gov/news-events/pr…

Join CBER’s Office of Vaccines Research and Review (OVRR) on October 23, 2025, for up-to-date information about the diagnosis of allergic contact dermatitis using patch tests. Register: fda.gov/AllergicContac…

On October 23, join FDA’s Public Workshop to gain perspective from health care providers, academia, and industry on approaches used for the review and approval of new patch test allergens for the diagnosis of allergic contact dermatitis. Register: fda.gov/news-events/fd…

Previously, Vonvendi was approved only for on-demand treatment of bleeding episodes and perioperative use in adults and preventative use only in adults with Type 3 VWD, the most serious type.

Today, FDA approved expanded use of Vonvendi for routine preventative use in adults (age 18 years+) with all types of von Willebrand disease and on-demand and treatment of bleeding episodes and perioperative use in children with VWD. fda.gov/news-events/pr…

Step into the forefront of novel endpoint development for rare disease drug development! CBER invites you to submit a proposal to the Rare Disease Endpoint Advancement (RDEA) Pilot Program, a collaboration with @FDA_Drug_Info. Deadline is 9/30/25! bit.ly/48UBUx4

Today, we approved a first-of-its-kind non-replicating adenoviral vector-based immunotherapy for the treatment of adult patients with recurrent respiratory papillomatosis. fda.gov/news-events/pr…

CBER invites you to submit a proposal to the Rare Disease Endpoint Advancement (RDEA) Pilot Program, a collaborative initiative with @FDA_Drug_Info. Join us in advancing development of #RareDiseaseTherapies. Submit your proposal now! bit.ly/48UBUx4

Listen in as CBER Director Dr. Vinay Prasad dives deeper into his recent decision memo related to COVID-19 vaccine approvals. youtu.be/jtudwT6zZwc

FDA provides recommendations to blood establishments that collect blood and blood components, including Source Plasma, for testing blood & blood components for #HepatitisB surface antigen to reduce the risk of transfusion-transmitted Hepatitis B virus. fda.gov/regulatory-inf…

FDA CBER Director Dr. Vinay Prasad shares key take home points explaining the rationale behind recent mRNA COVID-19 vaccine safety label updates. Watch the full discussion here. youtu.be/68QzzWGc4mI

FDA updated COVID-19 vaccine safety labeling based on two key data points. Watch CBER Director Dr. Vinay Prasad discuss these findings. Full video available on the FDA’s YouTube channel. youtu.be/68QzzWGc4mI

The FDA will not negotiate on safety. We will pursue safety signals with the best available scientific methods and ensure that our marketing authorizations are made only to populations where we have substantial confidence the benefits outweigh the risks.