FDA

FDA, working with international regulatory partners through the International Coalition of Medicines Regulatory Authorities (ICMRA), has completed a collaborative assessment of a new testing approach for biological products that could reduce reliance on horseshoe crab blood while continuing to ensure product quality. https://lnkd.in/gxdia3pX This assessment is part of the ICMRA Collaborative Assessment Pilot, which brings participating international regulators together to review proposed manufacturing changes at the same time, coordinate their questions to applicants, and make their own independent decisions within a standard 120-day review timeline. The pilot was established to improve consistency in regulatory expectations, reduce duplicative submissions and assessments, and facilitate more timely patient access to medicines. FDA participated in this assessment with international partners - Australia’s Therapeutic Goods Administration, Health Canada, Japan’s Pharmaceuticals and Medical Devices Agency, and Swissmedic, with the European Medicines Agency serving as the leading authority for this submission under the pilot. The review concluded with the approval of the PACMP in June 2026. All participating authorities reached aligned decisions within days of each other, which is a significant achievement in international regulatory coordination. https://lnkd.in/gQBuBfig FDA will continue working with international partners to support modern testing methods and approaches that advance pharmaceutical quality while protecting public health. Work is underway to build on the pilot and develop a more sustainable, globally coordinated regulatory approach.

Today, the FDA approved Mimrylo (rusfertide) for the treatment of erythrocytosis in adults with polycythemia vera. https://lnkd.in/ezGRW3kZ Polycythemia vera is a chronic blood cancer caused by a genetic mutation that drives the bone marrow to produce too many red blood cells. The excess red blood cells — a condition called erythrocytosis — thicken the blood, raising the risk of dangerous blood clots, stroke, and heart attack. Managing polycythemia vera typically means keeping the proportion of red blood cells in the blood (hematocrit) below 45%, often through regular phlebotomies — a procedure in which blood is drawn and removed — alongside medications. Despite these efforts, many patients continue to need frequent blood draws, representing a significant and ongoing burden. Mimrylo offers a new approach. It is the first hepcidin mimetic approved for polycythemia vera, meaning it works by mimicking hepcidin — the body's natural hormone that regulates iron levels. Because iron is essential for producing red blood cells, Mimrylo restricts that iron supply, reducing the overproduction of red blood cells rather than managing the consequences after the fact. In the VERIFY clinical trial, 76.9% of participants receiving Mimrylo required zero phlebotomies over 32 weeks compared with 32.9% receiving placebo. For patients living with polycythemia vera, today's approval is a meaningful step forward in managing their disease.

FDA approved Stelara to treat moderately-to-severely active pediatric ulcerative colitis (UC), marking a new class of therapy available to pediatric patients, and a meaningful step forward for children and families living with inflammatory bowel disease. https://lnkd.in/eZRYbVhc Stelara is now the first approved therapeutic monoclonal antibody that does not target human tumor necrosis factor alpha (TNFα) for the two main forms of pediatric inflammatory bowel disease — UC and Crohn's disease. This new pediatric therapy delivers on the Trump Administration’s Make America Healthy Again Initiative - the bold executive action to confront the childhood chronic disease crisis head on.

FDA will hold a public meeting on September 17 to discuss proposed recommendations for the reauthorization of the Generic Drug User Fee Amendments for fiscal years 2028 through 2032. https://lnkd.in/eCNgzYyq The public is encouraged to share their input on proposed recommendations. Submit your comments by October 17! https://lnkd.in/eY4Y5UWh

The FDA authorized 3 new JUUL e-cigarette products including the JUUL2 device, a tobacco-flavored pod, and a menthol-flavored pod. https://lnkd.in/eybrY7Gt The FDA found that a substantial proportion of adults who smoke combustible cigarettes and used the JUUL2 products stopped smoking entirely. Those who completely switched were exposed to lower levels of harmful chemicals than they would have been had they continued smoking cigarettes.

Join us on September 29, for the FDA’s Center for Biologics Evaluation and Research’s roundtable focused on strengthening the supply chain for blood manufacturing. The FDA and key stakeholders will discuss supply chain challenges, root causes, potential solutions, and strategies to build greater resilience and help ensure a safe and adequate blood supply in the U.S. 📅 September 29, 2026 🕘 9:00 a.m.–4 p.m. ET The registration link and draft agenda are available at: https://lnkd.in/evX-9D64 #BloodSupply #PublicHealth #SupplyChain

FDA's Enforcement Report is sometimes misinterpreted as breaking news or a new recall action, when it's not. Today's report includes the classification for the Taylor Farms de Mexico iceberg lettuce recall related to a Cyclospora outbreak. This is a routine step in the recall process. No new action has been taken, and the original July 17, 2026, recall notice has not changed. The FDA Enforcement Report is FDA’s official public database of recalls that the agency monitors across FDA regulated products, including foods, drugs, medical devices, biologics, veterinary products, and others. An Enforcement Report entry is not necessarily a new recall announcement or new FDA action. A company may have initiated and publicly announced a recall days or weeks earlier. FDA subsequently completes its health hazard evaluation, assigns a Class I, II, or III classification, and updates/posts the recall in the Enforcement Report. The updated posting dated 8/27/26 should not be interpreted as an expansion or change to the firm's earlier voluntary public warning. https://lnkd.in/eCV7CBs8

The FDA has approved Lisraya (brepocitinib), the first oral drug indicated to treat dermatomyositis in adults — a meaningful milestone for patients living with this rare and debilitating disease. https://lnkd.in/ev476C5v Dermatomyositis is a rare autoimmune disease that causes chronic inflammation, progressive muscle weakness, and distinctive skin rashes. For decades, patients have had to rely primarily on therapies originally developed for other conditions. Lisraya, a once-daily oral JAK/TYK2 inhibitor developed by Priovant Therapeutics Inc., was evaluated in a phase 3 randomized, placebo-controlled study of 241 adults. Participants treated with Lisraya 30 mg demonstrated greater clinical improvement at Week 52, including improvement in physical function and skin disease activity, and reduced corticosteroid use. The FDA granted Lisraya Priority Review, reflecting the significant unmet need this approval addresses. This approval highlights how rigorous science and a steadfast commitment to the rare disease community translates into meaningful progress for patients.

Today, the FDA issued an Emergency Use Authorization for a generic drug to prevent New World screwworm (NWS) infestations in certain cattle when administered within 24 hours of birth, at castration, or when a wound appears, providing cattle producers with an additional line of defense against NWS. Learn more: https://lnkd.in/eeeYK7Hh

The FDA is seeking public comment on Draft Guidance for Industry (GFI) #256B, “Compounding Animal Drugs from Bulk Drug Substances: Compounding under CGMP in Federally-Registered Facilities.” https://lnkd.in/eB8j5gt6 The draft guidance clarifies FDA’s enforcement discretion policy for animal drugs compounded from bulk drug substances, including its application to federally registered drug manufacturers and outsourcing facilities. Comments are accepted beginning Aug. 28 and should be submitted by Nov. 27. Visit Regulations.gov and search docket FDA-2018-D-4533 to learn more and submit comments.