#FDA approves a treatment for relapsed or refractory multiple myeloma under the FDA Commissioner’s National Priority Review Voucher (CNPV) pilot program.
https://www.fda.gov/news-events/press-announcements/fda-grants-third-approval-under-national-priority-voucher-program
#DYK the FDA launched a new public list of infant formulas available in the U.S.? https://www.fda.gov/food/infant-formula-homepage/infant-formulas-marketed-us
Access key product information in one place to help inform infant nutrition decisions.
Supporting President Trump's Great American Recovery initiative, FDA is seeking public input on new standards for in-home opioid disposal products to help combat the opioid crisis. Comments due April 6, 2026. Learn more: https://www.fda.gov/news-events/press-announcements/fda-address-unused-opioids-american-homes
Caring for a baby comes with enough questions. Finding reliable infant formula information shouldn’t be one of them.
Our updated webpages now make infant formula information easier to find. https://www.fda.gov/food/resources-you-food/infant-formula-homepage
FDA has warned 30 telehealth companies about misleading drug ads.
Consumers should not be fooled into thinking that unapproved, compounded GLP-1 drugs—marketed by telehealth companies—are generic versions or the same as FDA-approved products.
This #WomensHistoryMonth learn how our office protects and promotes the health of women through policy, science, education and research.
https://www.fda.gov/consumers/womens-health/about-owh
#WomensHealth
FDA issued 30 warning letters to telehealth companies for false and misleading claims about compounded GLP-1 products. This marks the second round of enforcement action since the agency's September crackdown on misleading pharmaceutical ads. https://www.fda.gov/news-events/press-announcements/fda-warns-30-telehealth-companies-against-illegal-marketing-compounded-glp-1s
🎬Missed FDA's virtual #RareDiseaseDay? The full recording is now available on the FDA’s YouTube page! https://www.youtube.com/live/B6bGjpQVqEs
Explore our discussions on key rare disease topics and FDA's continued commitment to this community. http://www.fda.gov/RareDiseaseDay
Infant formula safety and nutrition matter.
That’s why @HHSGov and FDA launched Operation Stork Speed, strengthening standards, increasing testing, expanding research, and improving transparency for families. https://www.fda.gov/food/infant-formula-homepage/operation-stork-speed
LAST CALL: 📢 The FDA is looking for patients and caregivers to share their experience living w/ Primary Sclerosing Cholangitis (PSC) during a virtual Patient Listening Session on April 30, 2026. To be considered for the session, fill out this survey by tomorrow, March 3, 2026. https://qualtricsxmjqffz4ktl.qualtrics.com/jfe/form/SV_3xbePqkVXSOlVQy
⏰Final reminder! Living with PSC?
Take a few minutes to share your experiences through this brief survey before tomorrow's deadline! https://qualtricsxmjqffz4ktl.qualtrics.com/jfe/form/SV_3xbePqkVXSOlVQy
This week, we've explored the landscape of rare diseases—from the 7,000+ conditions affecting 30 million Americans to the innovative programs bringing hope to patients.
The bottom line? Progress happens through partnership. While much work remains, we're committed to supporting the rare disease community and advancing treatments that transform lives.
Thank you for joining us this Rare Disease Week. Learn more about FDA's ongoing rare disease programs: https://www.fda.gov/patients/rare-diseases-fda
#RareDiseaseWeek
Searching for reliable infant formula information?
FDA’s new public list features formulas available in the U.S. that have been notified and reviewed for safety and nutrition, so you can make informed choices for your baby. https://www.fda.gov/food/infant-formula-homepage/infant-formulas-marketed-us
“I am excited about FDA Rare Disease Day and the opportunity to be part of a celebration of our community's progress, collaboration, and advocacy to bring new and improved treatments to patients and families living rare. I feel lucky to be part of such a strong, supportive community where patients like me can use our voices to drive change.”
Carter Hemion
Young adult rare disease patient advocate
Patients and caregivers are essential partners in FDA's rare disease work. Through Patient Listening Sessions with the National Organization for Rare Disorders (NORD), you can share your experiences directly with FDA staff. Your insights help us understand what matters most in treatment development and approval.
Have questions about FDA processes or treatments for your condition? Contact our Public Engagement Staff. http://www.fda.gov/PatientsAskFDA
#RareDiseaseWeek
What is Bayesian statistics? It will help our quest to see more rare disease treatments.
The FDA's "Gold Standard" depends on protecting sensitive information. From trade secrets to personal health data, safeguarding confidential information isn't just policy—it's essential to innovation and public trust. Our Deputy Commissioner for Operations and Chief Operating Officer Barclay Butler, Ph.D., explains why this balance matters. https://www.fda.gov/news-events/fda-voices/why-protecting-confidential-information-key-fdas-gold-standard
Food allergies can be a burden for children and their families.
Today, the FDA issued approval for a lung cancer drug as part of the new Commissioner's National Priority Voucher (CNPV) pilot program. https://www.fda.gov/news-events/press-announcements/fda-grants-second-approval-under-national-priority-voucher-pilot-program
Today, FDA published 98 new and revised product-specific guidances (PSGs) to accelerate generic drug development and increase competition for medications treating conditions such as breast cancer, reducing the risk of major adverse cardiovascular events and reducing excess body weight, including 68 for products that currently have no approved generics. https://www.accessdata.fda.gov/scripts/cder/psg/index.cfm
“From caring for children with rare diseases to supporting product development at FDA, my career has always been inspired by the resilience of rare disease families. Rare Disease Week is a powerful time of year because it brings together patients, families, advocates, sponsors and regulators around a shared purpose with common goals. At OOPD, I’m proud to support the rare disease community by helping advance treatments that can offer meaningful hope and potentially change lives.”
Lewis Fermaglich, MD
Medical Officer, Office of Orphan Products Development (OOPD), FDA
Have you had a liver transplant and are living w/ Primary Sclerosing Cholangitis (PSC)? The FDA is hosting a virtual Patient Listening Session for patients and caregivers to share their experience w/ PSC. Fill out this survey by March 3, 2026: https://qualtricsxmjqffz4ktl.qualtrics.com/jfe/form/SV_3xbePqkVXSOlVQy
#PSC #IBD
The FDA revamped its infant formula webpages to be more consumer-friendly, making it easier for parents and caregivers to find clear, trusted info on safety, nutrition, and recalls. https://www.fda.gov/food/hfp-constituent-updates/fda-augments-and-improves-infant-formula-resources-enhance-transparency-safety-and-access-american
How is industry incentivized to develop treatments for diseases affecting small populations? The Orphan Drug Act provides powerful incentives: tax credits for clinical testing, waiver of nearly $3 million in user fees, and potential 7-year market exclusivity after approval.
These incentives have helped bring hundreds of orphan drugs to patients who previously had no options. FDA also awards research grants to advance rare disease product development. Innovation requires investment—and patients deserve solutions.
#RareDiseaseWeek
“Rare Disease Day is an important education and engagement opportunity for FDA and the rare disease patient community. On this day we, together, continue the important work of advancing effective treatments for rare diseases, while we honor the 30 million patients and their families who struggle with rare disease, with particular focus on the many who are still in need of medical products for their disease.”
Amy Comstock Rick
Director, FDA Rare Disease Innovation Hub
We’re implementing bold reforms for rare disease patients.
A disease is considered rare if it affects fewer than 200K people in the U.S. Many are life-threatening, and developing treatments is uniquely challenging due to small patient populations and limited understanding of disease progression. That's why FDA partners with patients, caregivers, researchers, and manufacturers to support breakthrough treatments.
#RareDiseaseWeek
The FDA, in collaboration with @NASDANews, @afdonews, and state partners, are establishing the new Produce Regulatory Program Standards to strengthen our integrated food safety system and advance produce safety nationwide. https://www.fda.gov/food/hfp-constituent-updates/fda-establishes-regulatory-program-standards-strengthen-produce-safety-oversight-activities-states
“Rare Disease Week is important to me because it allows the community to unite together and remind one another that we are not alone. I am excited to be given the opportunity to be a part of FDA’s Rare Disease Day event because it only further shows that we as a community are seen and heard.”
Liam McCarthy
Young adult rare disease patient advocate
FDA should move with urgency to address rare diseases.
Did you know over 10,000 rare diseases affect more than 30 million Americans? That's roughly 1 in 10 people.
Since the Orphan Drug Act passed in 1983, FDA has approved hundreds of treatments for rare diseases—but our work is far from over.
Learn more about how we're supporting patients and advancing rare disease research: https://www.fda.gov/patients/rare-diseases-fda
#RareDiseaseDay #FDARareDiseaseDay #FDA120Years
Collectively, 30 million Americans have a rare disease. The problem isn't rare — it's a call to action.
We learn a lot by listening to people who are proximate.
Honored to host FDA‘s own Jim Traficant for a discussion on rare diseases.
His personal story is captivating and inspiring — we’re grateful to have him.
🚨 LIVE: FDA's #RareDiseaseDay virtual event! Topics include FDA’s Rare Disease Innovation Hub, clinical trials, AI, real-world evidence & patient engagement.
Join now: https://www.youtube.com/live/B6bGjpQVqEs
#FDA #RareDisease
This could not be further from the truth. This administration has addressed a roadblock that prohibited companies using natural colors from highlighting that fact on the labeling.
Under the old way, companies that used only natural color were forced to call them “artificial” when making labeling claims, creating consumer confusion and disincentivizing use. Fixing this issue will help companies continue to transition to using natural color alternatives as part of an industry wide phase out. Follow FDA’s dye removal tracker at https://www.fda.gov/food/color-additives-information-consumers/tracking-food-industry-pledges-remove-petroleum-based-food-dyes
As shown on FDA’s list of chemicals under review https://www.fda.gov/food/food-chemical-safety/list-select-chemicals-food-supply-under-fda-review FDA is actively reviewing the safety of titanium dioxide. We have already received numerous commitments from industry leaders to abandon the use of TiO2.
The FDA has a moral duty to move quickly to get meaningful treatments to patients.
Are you living with Primary Sclerosing Cholangitis (PSC) or caring for a loved one with PSC? Share your experience with FDA staff during a virtual Patient Listening Session on April 30, 2026. To be considered for the session, fill out this survey by March 3, 2026: https://qualtricsxmjqffz4ktl.qualtrics.com/jfe/form/SV_3xbePqkVXSOlVQy
Hospital contracting and IRB reviews need to move much faster.
Join us for the FDA Grand Rounds Feb. 19. Dr. Ronit Mazor, a principal investigator in the FDA’s Gene Transfer Immunogenicity Branch in CBER, will discuss gene therapy technology and examine the immunological challenges facing AAV gene therapy.
http://www.FDA.gov/GrandRounds