WASHINGTON, D.C. — The U.S. Food and Drug Administration (FDA) announced a series of actions last week aimed at strengthening consumer protections, advancing rare disease treatment development, and improving drug safety oversight. The measures address growing public health concerns ranging from nicotine pouch packaging to transparency in drug approvals and the safety of widely used GLP-1 medications.
FDA Urges Child-Resistant Packaging for Nicotine Pouches
Amid a sharp rise in accidental nicotine exposures, the FDA is calling on manufacturers to adopt child-resistant packaging for nicotine pouches. Between April 2022 and March 2025, U.S. poison centers reported a steady increase in exposure cases, with 72% involving children under five years old.
Nicotine pouches contain highly concentrated nicotine, which can be toxic or fatal to young children even in small amounts. Reported symptoms of accidental ingestion include confusion, vomiting, and loss of consciousness.
FDA Commissioner Dr. Marty Makary urged manufacturers to act, warning that “the fruity flavors and bright, colorful designs of nicotine pouch products could resemble candy and seem attractive to children.” The agency also released storage guidance for consumers and advised caregivers to secure nicotine products out of children’s reach.
FDA Introduces New Evidence Principles for Rare Disease Drug Development
To accelerate access to treatments for patients with limited options, the FDA unveiled the Rare Disease Evidence Principles (RDEP), a new framework designed to provide greater clarity and flexibility to drug developers targeting small patient populations.
Under the RDEP, drug approvals may rely on one well-controlled clinical study supported by robust confirmatory evidence, which can include biomarker data, non-clinical models, pharmacodynamic studies, and patient case reports.
The process applies to therapies addressing diseases affecting fewer than 1,000 U.S. patients, particularly those involving known genetic defects and limited or no existing treatments. Sponsors must apply for RDEP participation before beginning pivotal trials.
FDA officials emphasized that the initiative balances scientific rigor with practicality, ensuring rare disease treatments meet statutory safety and efficacy standards without imposing impractical evidence burdens.
FDA Expands Transparency by Publishing Complete Response Letters
In a major shift toward transparency, the FDA announced it will release complete response letters (CRLs) to the public in real time. CRLs explain why a drug application cannot be approved in its current form and outline safety or efficacy concerns identified during review.
Last week, the agency published 89 previously unpublished CRLs issued since 2024 and committed to releasing all future CRLs promptly. Sensitive commercial and personal information will be redacted.
Commissioner Makary described the move as a step toward “radical transparency,” stating that publishing CRLs will help drug developers avoid common pitfalls, speed up innovation, and restore public trust in FDA decision-making.
FDA Launches “Green List” to Combat Illegal GLP-1 Drug Imports
Responding to safety concerns over compounded versions of popular GLP-1 medications, including semaglutide and tirzepatide, the FDA introduced a “green list” import alert aimed at preventing unverified foreign active pharmaceutical ingredients (APIs) from entering the U.S. supply chain.
Only APIs from facilities inspected and approved by the FDA will be cleared for entry. Other sources face automatic detention without physical examination.
The agency’s action follows reports of improper formulations, dosing errors, and adverse events tied to compounded GLP-1 products. Officials stressed that the initiative is intended to safeguard patients and strengthen oversight of imported drug ingredients.
FDA Approves Expanded Use of Vonvendi for von Willebrand Disease
The FDA approved expanded indications for Vonvendi (von Willebrand factor, recombinant), making it the first recombinant therapy approved for pediatric patients with von Willebrand disease (VWD) in the U.S.
The updated approval now allows:
- Routine preventative use in adults with all VWD types.
- On-demand treatment and perioperative use in both adults and children.
Clinical studies demonstrated Vonvendi’s efficacy in controlling bleeding episodes and managing surgical bleeding risks across all ages. The therapy had already been approved for limited adult uses but now represents a broader treatment option for patients, particularly children.
Vonvendi received Priority Review status and Orphan Drug Designation, underscoring its importance in addressing unmet medical needs.
A Comprehensive Push for Public Health and Drug Safety
The FDA’s actions last week reflect a broader strategy to strengthen patient safety, improve transparency, and streamline drug development. From preventing accidental poisonings to increasing accountability in the approval process, the agency is signaling an assertive approach toward safeguarding public health and restoring trust in regulatory oversight.
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