FDA took several notable actions this week across drugs and medical devices. In an unusual move, a senior FDA official publicly criticized UniQure’s Huntington’s disease gene therapy data as “distorted” and “manipulated” during an agency-arranged media call—a rare occurrence that may create political complications for the Trump Administration.

State representatives met with FDA to discuss the Section 804 importation program, which allows importing cheaper prescription drugs from Canada, aligning with President Trump’s executive order on drug prices. Additionally, FDA issued 30 warning letters to telehealth companies for misleading marketing of compounded GLP-1 products.

In enforcement actions, former ExThera Medical executive Sanja Ilic agreed to plead guilty to defrauding FDA by failing to report adverse events related to blood filtration devices.

FDA approved several significant drugs: Janssen’s Tecvayli combination for multiple myeloma (the third approval under the new Commissioner’s National Priority Voucher pilot program, processed in just 55 days), the first generic version of GlaxoSmithKline’s Flovent HFA asthma inhaler, and Ascendis Pharma’s Yuviwel for achondroplasia in children.

On the medical device front, FDA held MDUFA VII negotiations with industry, published updated lists of AI-enabled and digital health devices, and recommended using established choking rescue protocols before anti-choking devices. Medline issued a Class I recall for reprocessed catheters due to contamination risks.

FDA also published international guidance documents and announced upcoming workshops on generic drug science and pediatric cell and gene therapy trials.

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This week’s FDA updates focus on regulatory reforms, product approvals, and safety recalls. Commissioner Marty Makary announced a pilot program offering bonuses to drug reviewers who complete work ahead of schedule, with initial payments beginning in August. Makary also defended CBER Director Vinay Prasad against media criticism, asserting he’s selecting the best scientists regardless of political backgrounds.

The agency published meeting minutes for ongoing user fee negotiations (GDUFA IV, PDUFA VIII, MDUFA VI) and released guidance on protecting confidential commercial information. FDA’s Division of Applied Regulatory Science reported completing 49 research projects and numerous collaborations in 2025.

**Key Drug Approvals:**
– Boehringer Ingelheim’s Hernexeos received accelerated approval for non-small cell lung cancer in just 44 days under the new Commissioner’s National Priority Voucher program
– Pfizer’s Braftovi gained approval for metastatic colorectal cancer with BRAF mutations
– Sanofi/Regeneron’s Dupixent became the first FDA-approved treatment for allergic fungal rhinosinusitis

**Medical Device Updates:**
CDRH reported improved performance metrics, with first-cycle major deficiency letters decreasing from 82% to 74%. However, several serious safety recalls were announced:
– Fresenius Kabi’s Ivenix pump (Class I recall for software anomalies)
– Boston Scientific’s Axios stents (167 injuries, 3 deaths reported)
– Olympus’s High Flow Insufflation Units (software overpressure issues)
– Abiomed’s Impella devices (purge leak risks)

FDA announced plans for 85 new drug-specific guid

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This Week at FDA highlights several significant regulatory developments. The most notable announcement came from FDA Commissioner Marty Makary and CBER Director Vinay Prasad, who revealed in the New England Journal of Medicine that the agency will now require only a single pivotal clinical trial for most drugs, departing from the decades-old standard of requiring two confirmatory trials.

The FDA reversed its initial refuse-to-file decision on Moderna’s mRNA influenza vaccine after the company proposed a revised regulatory approach involving full approval for adults 50-64 and accelerated approval for those 65 and older. Reports suggest this reversal followed President Trump’s frustration with the agency’s vaccine handling, though White House officials denied presidential involvement.

Commissioner Makary advocated for expanding over-the-counter drug availability, stating that medications should only require prescriptions if they’re unsafe, need laboratory monitoring, could be used nefariously, or are addictive. This aligns with administration efforts to lower drug prices.

In personnel changes, NIH Director Jay Bhattacharya became acting CDC director after Jim O’Neill’s dismissal—the fifth director removed this year.

Other updates include: AstraZeneca’s Calquence receiving approval for chronic lymphocytic leukemia treatment; upcoming meetings on rare disease data sharing and generic drug development; FDA recognition of updated international medical device standards; and a safety alert regarding Trividia Health’s glucose monitoring system manual error that could delay treatment for high glucose events.

The Duke-Margolis Institute and FDA will co-host a March 30 meeting on rare disease data sharing practices.

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This week’s FDA update covers a range of regulatory developments. First, the agency transitioned from the Quality System Regulation (QSR) to the more globally harmonized Quality Management System Regulation (QMSR), publishing revised guidances to support the transition. FDA also launched its PreCheck pilot program to strengthen the domestic pharmaceutical supply chain and provide greater regulatory predictability.

The report notes concerns raised by staff and former officials about the Commissioner’s National Priority Voucher program, which aims to expedite drugs considered national priorities but may lack sufficient regulatory oversight. The White House also launched a website, TrumpRx.gov, to help consumers find discounted brand-name drugs.

In other news, FDA is allowing food producers to claim “no artificial colors” if they use natural dyes, and approved new food coloring ingredients. The Health and Human Services Office of Inspector General is investigating potential conflicts of interest involving an FDA deputy chief of staff. Congress has reauthorized FDA’s rare pediatric disease priority review voucher program.

The summary also covers several drug and medical device regulatory actions. FDA rejected Corcept Therapeutics’ drug relacorilant and issued a warning about companies marketing “illegal copycat drugs.” Amgen rejected FDA’s call to withdraw its drug avacopan. FDA published new guidance on patient preference studies and updated labeling for cancer drugs. Finally, Abbott Diabetes Care issued a Class I recall for certain Freestyle Libre 3 CGM sensors due to inaccurate blood glucose readings, prompting an FDA warning letter.

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The FDA’s Center for Devices and Radiological Health (CDRH) released its 2025 annual report, highlighting a record number of 124 novel medical devices authorized last year, including the first blood test to diagnose Alzheimer’s disease. The report also noted that over 250 devices have been approved using real-world evidence.

While a partial government shutdown may affect other federal agencies, the FDA will continue normal operations as it has already been funded for the full year. However, the proposed government spending package does include measures that would impact the FDA, such as enabling the agency to mandate pediatric studies for certain cancer drug combinations and reauthorizing the Pediatric Disease Priority Review Voucher program.

There were some disputes over FDA’s actions, with ImmunityBio claiming the agency had endorsed its cancer drug Anktiva, which the FDA denied. Moderna’s CEO also stated the company would not invest in new late-stage vaccine trials due to opposition from US health officials.

In other news, the Trump administration requested a pause on a lawsuit by Louisiana seeking to restrict abortion pill access, while the launch of the administration’s TrumpRx website for discounted medications was postponed.

The FDA also took regulatory actions, including placing clinical holds on two of Regenxbio’s gene therapy investigational products, approving a multiple myeloma drug combination, and issuing warning letters to several medical product manufacturers for various quality and safety issues.

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This week, the US Department of Health and Human Services (HHS) announced the end of research using aborted human fetal tissue and the US withdrawal from the World Health Organization (WHO). HHS cited ethical concerns and mishandling of the COVID-19 pandemic as reasons for these decisions.

In an effort to strengthen the domestic pharmaceutical supply chain, the FDA is launching a PreCheck Pilot Program to streamline oversight and encourage domestic manufacturing. The FDA also published its annual report on new drug approvals, highlighting that 46 novel drugs were approved in 2025, with 33 utilizing expedited programs. Additionally, 18 biosimilars were approved, including 4 for new reference products.

The FDA is seeking public input on improving labeling and cross-contamination information related to gluten in food products. The agency also provided an initial review of the FDA ImportShield Program, which has led to faster processing, increased capacity, and reduced staff hours for regulated import products.

In the drugs and biologics sector, the FDA and the generic drugs industry discussed several issues during the Generic Drug User Fee Amendments (GDUFA III) negotiations. Additionally, Valneva has decided to withdraw its applications for its chikungunya vaccine, Ixchiq, in the US due to a suspension and clinical hold.

The Reagan-Udall Foundation for the FDA will host a public meeting on reducing reliance on animal testing in drug development. The FDA also issued early alerts for two medical device products: Integra LifeSciences’ MediHoney Wound and Burn products and Olympus’ High Flow Insufflation Units, citing packaging and software issues, respectively.

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This week’s FDA update covers a range of regulatory issues and developments. The main focus is on the Commissioner’s National Priority Voucher (CNPV) program, which has raised concerns among FDA officials and the industry. The program aims to prioritize the review of drugs deemed critical to national security, potentially shortening their review times. However, unnamed officials have expressed worries that this may be illegal, unethical, and could lead to a disregard for the agency’s scientific standards. There are also reports of delays in the review of two drugs that are part of the CNPV program due to safety concerns.

In other news, Republican lawmakers are calling for restrictions on the use of the abortion drug mifepristone, arguing that it should only be prescribed based on in-person visits. Meanwhile, the Department of Health and Human Services has appointed two OB/GYNs with histories of opposing vaccines and antidepressants to the CDC’s Advisory Committee on Immunization Practices.

The FDA has also requested the removal of information about the risk of suicidal ideation and behavior from the labeling of certain glucagon-like peptide-1 (GLP-1) receptor agonist drugs, and it has been reported that the agency quietly deleted a webpage listing unproven and bogus autism treatments.

Additionally, the FDA has published updates to its Standard Operating Policy and Procedure manuals for the administrative processing of clinical holds and IND applications, and has approved the first treatment for Menkes disease in children.

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This week, the FDA has made several updates and announced upcoming events related to digital health, drug regulation, and medical device oversight. The agency has updated two guidances to ease its oversight of general wellness devices and clinical decision support software, aiming to bolster the adoption of AI-enabled digital health products.

The FDA has scheduled public meetings on generic drug development, challenges in designing Phase 3 trials for rare diseases, and the implementation of its Quality Management System Regulation. The Centers for Disease Control and Prevention has also updated its childhood vaccination recommendations, reducing the number of vaccines broadly recommended.

In the drugs and biologics domain, the FDA has updated its policies on posting new safety information, published meeting minutes from user fee negotiations with the drug industry, and approved several new treatments, including Aqvesme for thalassemia, Yartemlea for stem cell transplant-associated thrombotic microangiopathy, and Myqorzo for obstructive hypertrophic cardiomyopathy.

For medical devices, the FDA has proposed launching a new iteration of its Total Product Lifecycle Advisory Program and developing additional IT tools to facilitate interactions with manufacturers. The agency has also emphasized international harmonization in its device user fee negotiations, focusing on areas like harmonized pre-market reviews and increasing the frequency of meetings with global regulatory authorities.

Overall, the FDA is working to streamline its oversight, support the development of innovative digital health and medical products, and enhance its collaboration with industry and international partners.

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This week at the FDA, there are several notable updates across drug, medical device, and regulatory policy. Staffers have raised concerns that the Commissioner’s National Voucher Program (CNVP) is being used as a political tool by the Trump administration, alleging that vouchers were steered to companies with ties to the administration’s drug pricing agreements.

The FDA recently awarded CNVP vouchers to Merck for two drugs – an oral PCSK9 inhibitor and an antibody-drug conjugate – citing their potential to increase treatment access and reduce healthcare costs. However, the FDA Commissioner stated the agency has no plan to add a boxed warning to COVID-19 vaccines, contrary to recent reports.

The FDA finalized several new guidances this week, including accepting real-world evidence in medical device submissions without requiring patient-level data, and providing clarity on its bioresearch monitoring program. New draft and final guidances were also issued on improving clinical trial enrollment, especially for female participants.

On the drug and biologics front, the FDA updated standard operating procedures for sponsor meetings and refuse to file determinations. Key approvals include Milestone’s nasal spray for paroxysmal supraventricular tachycardia, Daiichi Sankyo’s breast cancer drug, and Rubraca for prostate cancer. The agency also issued a priority review voucher for a Barth Syndrome treatment.

Lastly, the FDA published meeting minutes on reauthorizing the Generic Drug User Fee Amendments, with industry proposals around review timelines and the complex generics program.

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The FDA has issued a final guidance that removes the requirement for identifiable patient-level data in medical device submissions using real-world evidence (RWE). The guidance acknowledges that meaningful information can be derived from large de-identified databases, such as cancer and cystic fibrosis registries, without needing access to individual patient data.

This policy change enables the use of a wider range of real-world data sources, including cancer registries, hospital systems, insurance claims, and electronic health records. The FDA will now assess the strength of submitted RWE on an application-by-application basis, rather than requiring patient-level data in all cases.

The guidance also provides new examples of when the FDA will and will not accept investigational device exemptions (IDEs) that leverage real-world data (RWD) in submissions, as well as a demonstration of how RWE can be obtained for a device under emergency use authorization (EUA).

The final guidance complies with a Congressional mandate under the Food and Drug Omnibus Reform Act of 2022 (FDORA) and a commitment made in the Medical Device User Fee Amendments Performance Goals and Procedures for Fiscal Years 2023 through 2027 (MDUFA V) to clarify the use of RWE and RWD in regulatory decision-making. The FDA has reported that the integration of RWE into device approvals has been more extensive than for drugs and biologics, with over 250 premarket authorizations including RWE since 2016, compared to 35 drugs, biologics, or vaccines in the same timeframe.

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