This Week at FDA: New international partnerships, UniQure and Moderna reversals, and more
Welcome to another installment of This Week at FDA, your weekly source for updates – big and small – on FDA, drug and medical device regulation, and what we’re reading from around the web. This week, FDA announced new partnerships with two international regulators and reversed two notable decisions made under former top officials.
FDA and the African Medicines Agency (AMA) recently signed a Statement of Cooperation to develop a work plan to exchange information, strengthen regulatory cooperation, and coordinate with stakeholders on medical product regulations.
The UK's Medicines and Healthcare products Regulatory Agency (MHRA) also announced a new partnership with FDA to have dedicated and reciprocal liaison officers within each other's agencies. The agencies said that liaison officers will enable faster, more effective coordination when addressing emerging regulatory challenges.
Earlier this month, Reuters reported that Acting FDA Commissioner Kyle Diamantas met with representatives of rare disease groups in a bid to fix relationships after they soured under former FDA Commissioner Marty Makary when FDA declined to approve several rare disease treatments. This week, the FDA posted a blog post about that meeting, emphasizing its "renewed unwavering commitment to the rare disease community."
Advisors on FDA's Vaccines and Related Biological Products Advisory Committee are meeting on Thursday, 18 June, to discuss whether to recommend approval of Moderna's MFLUSIVA mRNA influenza vaccine for adults ages 50–64 and accelerated approval for adults 65 and older. As BioSpace reported, in February, Vinay Prasad, former director of the Center for Biologics Research and Evaluation, refused to file the application, but the agency's briefing documents published earlier this week suggest a more favorable view of the submission.
STAT News reported that FDA has withdrawn its objection to UniQure's Huntington’s disease drug AMT-130, clearing the path for the company to file an accelerated approval application in the third quarter of 2026. Makary and Prasad, both of whom are no longer with the agency, had previously objected to the drug, stating the data were insufficient for a marketing application.
The Oncology Center of Excellence has published its annual report on its efforts to promote patient-centered regulatory decision-making. The agency noted that it approved 17 new cancer treatments in 2025, 12 cancer drugs and biologics for children, and collaborated with international regulatory agencies to accelerate reviews.
FDA has renewed its Science Board, a committee of 15 external experts, to advise it for another two years until 26 June 2028. The committee is intended to be composed of panelists with scientific expertise across a broad range, including food safety and nutrition, clinical research, veterinary medicine, pharmacology, toxicology, medical devices, and more.
Bloodlines used in dialysis have been removed from the FDA's medical device shortage list, while the agency also extended the estimated duration of shortages for endoscopic vessel harvesting systems and oxygenator devices intended for extracorporeal circulation through the first quarter of 2027.
FDA also warned healthcare providers about ongoing interruptions in the supply of stereotactic breast biopsy needles, which it says may require making adjustments when caring for patients who need breast biopsies, and recommended healthcare providers consider conservation strategies. The agency said it expects the needle shortage to continue at least through the first quarter of 2027.
FDA published more than two dozen warning letters this week, primarily to telehealth companies for illegally marketing compounded glucagon-like peptide-1 (GLP-1) receptor agonist drugs without authorization, and several drug and device companies for violating current good manufacturing practices requirements. The agency particularly highlighted a warning letter to Happiest Baby, the maker of SNOO Smart Sleeper, for marketing the products without authorization, and said some refurbished products have been found to be unsanitary.
Drugs & Biologics
Amphastar Pharmaceuticals' nasal spray Rextovy (naloxone hydrochloride) has been approved as the first over-the-counter intranasal emergency treatment for opioid overdose. Consumers can now directly buy the product without a prescription in what the Trump administration says is a significant step to address the opioid crisis.
Norwich Pharmaceuticals' generic version of Genentech's Xofluza (baloxavir marboxil) was approved by FDA as the first single-dose treatment for acute uncomplicated influenza and prophylaxis in patients 5 years and older. The agency said the decision will expand access to the flu treatment.
FDA announced it is hosting a two-day hybrid workshop in partnership with the University of Maryland Center of Excellence in Regulatory Science and Innovation to discuss developing antihypertensive therapies for pediatric patients. More specifically, stakeholders will have the opportunity to discuss similarities and differences in disease pathophysiology and drug response across age groups, the extent to which efficacy data can be extrapolated from adults to children, and more.
FDA also reminded stakeholders that it is hosting a hybrid meeting on 25 August Patient-Focused Drug Development (PFDD) for nonhealing chronic wounds. Patients, patient representatives, and care partners will share their experiences with nonhealing chronic wounds during the meeting, and attendees will learn how the condition affects patient health, current treatments, and clinical trial considerations.
The iPLEDGE Risk Evaluation and Mitigation Strategy (REMS), intended to mitigate the risk of embryo-fetal toxicity, has been delayed by FDA until 15 November. The REMS was set to go into effect on 8 August, but the agency said it wants to allow more time to test the iPLEDGE REMS system updates, minimize technical issues for iPLEDGE REMS platform users, and ensure patients continue to have access to isotretinoin treatment.
FDA has published a technical specifications document entitled "Submitting Clinical Trial Datasets to Evaluate the Impact of Immunogenicity on the Pharmacokinetics of a Drug," intended to help support the regulatory evaluation of immunogenicity data. It describes the content and format specifications for Analysis Dataset for Immunogenicity Specimen (ADIS), Analysis Dataset for Pharmacokinetic Concentrations (ADPC), and Subject Level Analysis Dataset (ADSL).
GlaxoSmithKline's drug Utebzi (tebipenem pivoxil) has been approved by FDA to treat complicated urinary tract infections (cUTI), including pyelonephritis, caused by several susceptible microorganisms in adults with limited or no alternative oral treatment options. The agency noted the drug received priority review, fast track, and Qualified Infectious Disease Product Designation.
FDA published meeting minutes from its Biosimilar User Fee Act (BsUFA) reauthorization meeting on 12 May with industry representatives, including the Association for Accessible Medicines, the Biotechnology Innovation Organization (BIO), the Biosimilars Forum, and PhRMA. The parties discussed several key issues, including a data fidelity proposal, an industry exclusivity determinations proposal, and an industry inter-center review proposal.
Medtech
As part of its Home as a Health Care Hub Initiative, FDA has announced a new challenge called “READI-Home: Reducing Readmissions through Device Innovation for the Home.” The aim of the challenge is to help medical devices that can reduce hospital readmissions get to market sooner, thereby reducing morbidity and mortality associated with chronic conditions and lowering financial and logistical burdens on the healthcare system.
Gastrointestinal blood-detection capsules were classified by the FDA as class II devices with special controls. The classification order includes mitigation measures, including biocompatibility evaluations, clinical and non-clinical performance testing, electrical, thermal, and mechanical safety testing, electromagnetic compatibility testing, and labeling changes.
Radiological machine learning-based quantitative imaging software with predetermined change control plans was also classified to class II devices with special controls. The imaging software are required to take mitigating measures, including design verification and validation, and several other special controls.
Draeger is expanding its October 2024 Class I recall for certain Atlan A350 and Atlan A350XL anesthesia workstations due to concerns that a manufacturing error may cause the piston ventilator to fail before use or mechanical ventilation to fail during use, potentially leading to hypoxia, lung recruitment, bradycardia, cardiac arrest, and death. While no injuries or deaths have been reported so far, the company said it will replace the ventilator motor assembly for suspected affected products.
