FDA

New FDA guidance signals a major shift for CAR-T development, calling for randomized trials with standard-of-care control groups and clear evidence of superiority over existing therapies, while simultaneously easing REMS requirements to reduce logistical burdens for treatment centers and patients.

Twelve former FDA leaders have publicly challenged the agency’s proposed overhaul of vaccine approvals, sparked by an internal memo linking child deaths to COVID-19 vaccination, arguing the changes threaten evidence-based standards, weaken immunobridging practices, and risk eroding public trust.

The FDA has introduced a secure, agency-wide agentic AI system to support complex regulatory workflows—enhancing reviews, surveillance, and inspections—while maintaining strict data protections and human oversight.

In this Q&A, John Kirk, principal regulatory strategist at Veristat, explains how the FDA’s Commissioner’s National Priority Voucher (CNPV) Program is accelerating operational timelines, tightening cross-functional workflows, and redefining what sponsors and CROs must deliver to secure and execute an ultra-compressed one-to-two-month review.

The company submitted the new dosage for approval based on its Phase III trial results.

Epkinly plus rituximab and lenalidomide is the first bispecific antibody combination FDA-approved for relapsed or refractory follicular lymphoma, backed by Phase III data showing substantially improved disease control over standard therapy.

Regulatory inspections of interactive response technology systems have surged 140% since 2022, signaling intensified global scrutiny of data integrity, randomization, and system validation in clinical trials.

FDA outlines its limited operations during the funding lapse, focusing on essential public health activities while halting new drug applications.

A quick look at FDA’s 2025 transparency moves, what CRLs are (and aren’t), how FAERS works and its limits, who can report, and where the agency is applying AI.

The designation supports expedited development of SAR402663, Sanofi’s one-time gene therapy designed to reduce treatment burden and slow vision loss in patients with wet age-related macular degeneration.

FDA approves COVID-19 vaccines from Moderna, Pfizer-BioNTech, and Novavax targeting the LP.8.1 sublineage of SARS-CoV-2, with eligibility limited to adults aged 65 years and older and those with underlying medical conditions that place them with a high risk for severe disease.

The FDA will now publish adverse event data from FAERS on a daily basis, marking a major step toward greater transparency in drug development.

With approval to proceed, the Phase II trial (NCT06092034) of RP-A501 for Danon disease restarts under an optimized dosing and immunomodulatory strategy.

Breakthrough Therapy Designation was based on results from the Phase II IDeate-Lung01 trial (NCT05280470), which demonstrated clinically meaningful benefits in patients with extensive-stage small cell lung cancer treated with ifinatamab deruxtecan.

Ron Lanton, partner, Lanton Law, explains how shifts in policy and government guidance could reduce public participation and complicate the design and recruitment of future vaccine trials.

In this video interview, Meri Beckwith, Co-CEO of Lindus Health, explains why sponsors pursuing breakthrough or priority review designations must maintain rigorous trial design while building organizational agility.

In this video interview, Meri Beckwith, Co-CEO of Lindus Health, discusses recent Complete Response Letters issued to Replimune and Capricor, explaining how shifts in FDA reviewers can affect single-arm trial acceptance, especially in oncology settings where control groups may be deemed unethical.

In this episode of the Applied Clinical Trials Brief, we recap our three most-viewed stories of the previous week with a look into the FDA’s heightened scrutiny of trial design, the wind-down of federal mRNA vaccine programs, and how digital innovation is reshaping the clinical research landscape.

In this video interview, Meri Beckwith, Co-CEO of Lindus Health, examines how recent developments with Complete Response Letters have shifted industry perceptions of FDA breakthrough designation—highlighting that it offers faster review timelines and investor visibility, but not a higher likelihood of final approval.

A unified technology approach improves study efficiency and data collection.

In this video interview, Luke Wilson, senior director, biotech, pharma services at Thermo Fisher Scientific, discusses how integrated systems and data transparency enable sponsors to meet evolving regulatory expectations and streamline FDA inspections through enhanced operational readiness.

Complete Response Letters recently issued by the FDA signal heightened scrutiny of trial design and reinforce the agency’s shifting regulatory expectations for sponsors and CROs.

Despite ambitions to streamline regulatory review, FDA’s Elsa platform has been prone to hallucinations, prompting internal scrutiny and questions about AI reliability and governance.

The FDA’s complete response letter cited concerns that the Phase I/II IGNYTE (NCT03767348) trial in advanced melanoma was not an adequate, well-controlled study and that its heterogeneous patient population limited interpretability, preventing approval in its current form.

Despite the setback with the Oncologic Drugs Advisory Committee vote, GSK emphasizes the unmet need in multiple myeloma and highlights global momentum behind Blenrep combination approvals.