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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Today’s batch of 98 PSGs (31 New and 67 Revised) contains:

• 68 PSGs for products with no approved ANDAs (including 31 complex products)
• 46 PSGs for complex products (7 new and 39 revised PSGs)
• PSGs for products such as the first-in-class PI3K inhibitor for treatment of breast cancer with PIK3CA mutations, a pan-genotypic direct-acting antiviral, the first FDA-approved treatment for geographic atrophy secondary to age-related macular degeneration, and the first tissue-agnostic approval for treatment of NTRK gene fusion-positive solid tumors
• Additional noteworthy PSGs are described below, including PSGs that were supported by GDUFA-funded research. These PSGs reference products used for opioid use disorder, reduction of risk of major adverse cardiovascular events and excess body weight, and pain associated with diabetic neuropathy among other indications/conditions

When finalized, the PSGs in today’s batch posting will describe the agency’s current thinking and recommendations on how to develop generic drug products that are therapeutically equivalent to specific reference listed drugs. FDA considers all comments to the public docket before finalizing PSGs.

Newly Updated – Upcoming PSGs 

Today, FDA also updated the “Upcoming Product-Specific Guidances for Generic Drug Product Development” web page, which includes new and revised PSGs under development and includes the planned revision categories and brief descriptions of the revisions. As stated in the GDUFA III Commitment Letter, eligible applicants may request a PSG teleconference to obtain FDA’s feedback on the potential impact of a new or revised PSG on its development program and a subsequent PSG meeting following feedback received at the PSG teleconference. See the guidance for industry Product-Specific Guidance Meetings Between FDA and ANDA Applicants Under GDUFA for more information.  The web page also provides information about the agency’s plans for issuing new or revised PSGs in the coming year for all generic drug products (complex and non-complex), along with anticipated publication dates, consistent with FDA’s GDUFA III commitments.

Noteworthy PSGs in Today’s Batch:  

• New PSG for buprenorphine subcutaneous extended-release solution indicated for the treatment of opioid use disorder (RLD: BRIXADI, NDA 210136)
• New PSG for semaglutide oral tablet indicated for the reduction of the risk of major adverse cardiovascular events and reduction of excess body weight (RLD: WEGOVY, 218316)
• Revised PSGs for four modified-release drug products to remove in vitro alcohol dose dumping studies based on new GDUFA-funded research that a therapeutic equivalence assessment can be made without these studies (RLD: THIOLA EC, NDA 211843; RLD: DRIZALMA SPRINKLE, NDA 212516; RLD: CYMBALTA, NDA 021427; RLD: TALICIA, NDA 213004)
• Revised PSGs for 30 transdermal/topical delivery systems to reduce the need to conduct a sensitization study to limited conditions, e.g., when a proposed generic product has differences in composition compared to the RLD. The change is expected to significantly reduce the number of subjects enrolled in such studies and streamline generic product development

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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 U.S. Food and Drug Administration (FDA) has proposed several changes to the Generic Drug User Fee Amendments (GDUFA) program as part of the renewal process for fiscal years 2028-2032. One key proposal is to waive annual facility fees for the first three years for companies that establish domestic manufacturing operations for finished generic drugs or active pharmaceutical ingredients (APIs).

However, the pharmaceutical industry has opposed this proposal, arguing that there are more effective ways to enhance domestic manufacturing capacity, such as expanding production at existing U.S. facilities or retrofitting unused ones. Industry representatives claim the waiver would unfairly subsidize new competitors at the expense of established U.S. manufacturers.

The FDA has also proposed raising the foreign facility fee from $15,000 to $25,000 to account for inflation and facilitate more unannounced inspections of overseas facilities. The industry opposes this increase, fearing it may lead companies to exit the U.S. generic drug market.

Additionally, the FDA has proposed prioritizing ANDA reviews for firms that conduct pivotal bioequivalence testing in the U.S., have a waiver from bioequivalence testing, or manufacture finished dosage forms domestically. The industry agrees this could encourage domestic manufacturing and testing but is unsure if the FDA can grant priority review to companies meeting only a subset of the criteria.

Finally, the FDA suggests extending ANDA goal dates by 180 days if there are data fidelity or integrity issues related to bioequivalence or bioanalytical data. The industry supports this proposal but questions how it will address the root causes of such data problems.

Overall, the FDA’s proposals aim to enhance domestic pharmaceutical manufacturing and reduce reliance on foreign production, though the industry has raised concerns about the effectiveness and potential unintended consequences

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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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FDA Commissioner Marty Makary wants the same sense of urgency applied to the agency’s Commissioner’s National Priority Voucher (CNPV) program as was seen with Operation Warp Speed’s COVID-19 vaccine development. The CNPV program aims to expedite reviews of the chemistry, manufacturing, and controls (CMC) portion of drug submissions while therapies are still in clinical trials. Makary likened this to the efficiency of Operation Warp Speed, stating the agency should challenge assumptions that drug development takes 10-12 years and that manufacturing must be done outside the US.

Makary also discussed the FDA’s PreCheck program, which will designate its first company this summer. This program is intended to streamline oversight of certain manufacturing facilities based on national priorities, provide regulatory predictability, and encourage domestic factory construction.

Regarding technological innovations, Makary highlighted the agency’s use of AI to expedite application processing and reduce the need for animal testing. He noted FDA’s ELSA AI assistant and how computational modeling can predict drug interactions, potentially shaving 6-9 months off development timelines. The FDA has also loosened animal testing requirements for monoclonal antibodies and other drugs, instead relying on real-world data and New Approach Methodologies.

Lastly, Makary said the FDA is updating guidance to ease oversight of general wellness devices and clinical decision support software, allowing the agency to “get out of the way” for consumer-grade products while maintaining oversight for medical-grade devices.

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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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