Drugs
FDA has cited four deaths among patients treated with Newron Pharmaceuticals’ investigational schizophrenia drug evenamide as a potential cardiac safety signal, according to new details disclosed by the company concerning the agency's continuing clinical hold on its Phase 3 ENIGMA-TRS 2 study in the U.S. A
Drugs
A new Stanford Biodesign policy paper is calling for a dozen bipartisan FDA reforms aimed at accelerating clinical trials and drug reviews, including allowing some lower-risk Phase 1 trials to begin without waiting for prior FDA approval and expanding rolling review of marketing applications. The paper, Roadmap for FDA
Drugs
A review of 42,192 observations from 8,663 inspections conducted between 2008 and 2026, as shown on the FDA inspection dashboard, found that 2026 does not represent a wholesale shift in FDA inspection priorities. Rather, according to regulatory consultant Jeffrey Palmer, writing in an online post, it reflects intensified
Drugs
FDA’s decision to establish its new Office of Innovation and Clinical Trial Modernization (OICTM) and form dedicated divisions for artificial intelligence (AI) and real-world evidence (RWE) analytics (see earlier story) could provide drug developers with a clearer regulatory pathway for emerging technologies, but the agency now faces the
Drugs
FDA should strengthen its oversight and enforcement of confirmatory trials for accelerated approval drugs, with Medicare and Medicaid payments limited until the products’ clinical benefits are confirmed, according to a new proposal for expanding the agency's role in addressing drug costs. The proposal is one of several recommendations
Drugs
FDA’s new commissioner, once confirmed and sworn in, should provide greater clarity on regulatory pathways for rare disease therapies, especially the use of single-arm clinical trials, a rare disease advocate argued in a new Washington Examiner commentary. David Gusick said uncertainty over FDA’s expectations could delay development
Drugs
FDA has released the transcript of its 9/16 public meeting that outlined the Prescription Drug User Fee Act Agreement for reauthorizing the program for fiscal years 2028 through 2032. Under the agreement, the agency is proposing new mechanisms to reduce complete response letters, improve sponsor communications and address manufacturing
Drugs
HHS’ Advanced Research Projects Agency for Health has launched a funding initiative aimed at dramatically shortening clinical development of drugs and biologics through adaptive platform trials, artificial intelligence, and real-time data analysis. The “Simulation-augmented, Real-time Platform Adaptive Seamless Trials (SURPASS)” program is seeking technologies and trial approaches
Drugs
FDA says it has directed Takeda to add a Boxed Warning to the labeling for its Adzynma, a recombinant ADAMTS13 enzyme replacement therapy approved for congenital thrombotic thrombocytopenic purpura (cTTP). An agency online notice says Adzynma has been associated with the development of neutralizing antibodies that can prevent the drug
Drugs
FDA has accepted a letter of intent (LOI) for a wearable sensor-based digital outcome measure designed to assess foot drop severity and gait impairment in patients with Charcot-Marie-Tooth disease type 1A (CMT1A), marking the first digital health technology for a rare neurological condition accepted into the agency&
Drugs
FDA says efforts by CDER scientists to develop a systematic plasma proteomic biomarker framework can accelerate drug development by providing early mechanistic insights into therapeutic effects and enabling more informed decision-making in clinical trials through the selection of robust, biologically relevant biomarkers. An online post says that to evaluate
Drugs
Four stakeholders are suggesting ways FDA could improve its draft guidance on substantial evidence of effectiveness for human drug and biological products. The Center for Science in the Public Interest (CSPI) says it “opposes any shift in the regulatory standard to a one-trial default….” While the draft does not
Drugs
FDA has released the Form FDA-483 with three observations from its 8/10-8/19 inspection at the STAQ Pharma Ohio outsourcing facility in Columbus, OH. The inspection observations were: * failure of laboratory controls to include the establishment of scientifically sound and appropriate specifications, standards, sampling plans, and test
Drugs
A 3/31-4/3 FDA inspection at the kdc/one Chatsworth drug manufacturing facility in Chatsworth, CA, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A just-released 9/8 Warning Letter says the violations were: * failure to have, for each batch of drug
Drugs
An 11/3-11/14/2025 FDA inspection at Empower Clinic Services, doing business as Empower Pharmacy, a compounding pharmacy in Houston, TX, found that the pharmacy is compounding GLP-1 drugs that don’t meet legal requirements for such compounding. A 9/16 Warning Letter says the agency investigators
Drugs
Three major pharmaceutical industry associations are urging FDA to revise its proposed distributed manufacturing rule to provide greater flexibility for existing multi-site manufacturing networks, clarify inspection and registration requirements, and avoid imposing additional regulatory burdens that could undermine drug supply-chain resilience. In 9/21 comments to the agency,
Drugs
FDA has accepted for priority review an Insmed supplemental NDA seeking traditional approval for Arikayce (amikacin liposome inhalation suspension) for treating Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen. The agency has set a user fee review action target date of 1/28/2027.
Drugs
A coalition of 20 state attorneys general is urging the Trump administration to tighten or suspend an FDA import program, alleging that Chinese suppliers are exploiting it to send unapproved pharmaceutical ingredients used in compounded GLP-1 products into the U.S. In a letter led by Alabama Attorney General
Drugs
FDA has granted Eli Lilly full approval for the combination use of Inluriyo (imlunestrant) with Verzenio (abemaciclib) in adults with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative, estrogen receptor 1 (ESR1)-mutated advanced or metastatic breast cancer, as detected by an FDA-authorized test, with
Drugs
A bipartisan Senate bill intended to give patients and health care providers more information about where prescription drugs are manufactured has lost two provisions that drug safety experts considered important to making the new disclosure requirements effective, according to ProPublica. The bill, the Consumer Labeling for Enhanced API Reporting and
Drugs
FDA says it will begin accepting requests on 10/1 from drug and biologics sponsors seeking to participate in the fifth and final year of its Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) program. The CDRP program, established under the PDUFA VII commitment letter for fiscal years 2023
Drugs
FDA’s proposed drug user fee (PDUFA VIII) commitments could give drug and biologics sponsors several new opportunities to address manufacturing facility problems before they delay approvals or result in complete response letters (CRLs), according to an analysis from Arnold & Porter. The law firm said the proposed “CMC facility