Today, the FDA approved the first cell-based gene therapy for the treatment of Wiskott-Aldrich Syndrome (WAS). https://t.co/9h9BRtqgwr

FDA approved the first hematopoietic stem cell transplant (HSCT) therapy to treat patients with severe aplastic anemia (SAA). Learn more: https://t.co/xc9y2LXbMt

🚀Episode #3 of The Upside is live! Pancreatic cancer remains one of the hardest, deadliest cancers to treat. But $ACTU’s lead drug has shown meaningful improvements in survival – and could be the start of a true “pipeline in a molecule.” With major regulatory + clinical

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. https://t.co/d7Aw5vBkNs

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. https://t.co/PEae8YxTQ3

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! https://t.co/AGflP3kz6C

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. https://t.co/CFy5AHrgpu

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: https://t.co/mFGpb7sjN5

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: https://t.co/2l5xoJV3CA

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. https://t.co/95Zxg90ren

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! https://t.co/AGflP3k1h4

Today, we approved a first-of-its-kind non-replicating adenoviral vector-based immunotherapy for the treatment of adult patients with recurrent respiratory papillomatosis. https://t.co/ThwUZ3Swsc

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! https://t.co/AGflP3k1h4

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

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. https://t.co/3Ugwo8hmGw

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. https://t.co/hKblvAJeRO

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. https://t.co/hKblvAJeRO

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.

Today, FDA provided important updates on the risk of myocarditis after receiving an mRNA COVID-19 vaccine, noting the risk is higher in young men.

When we can minimize the burden on the healthcare system, while at the same time ensuring that benefits outweigh risks, we will! The FDA has taken a big step in removing barriers impeding the delivery of cures by removing the REMS requirement for BCMA- and CD19-directed