Drugs
Former FDA commissioner Robert Califf says the form FDA-483 is the means by which an agency investigator notifies a firm that conditions were observed that may have violated the Federal Food, Drug, and Cosmetic Act. Writing in a Clinical Leader column, he says the FDA-483 is too often
Drugs
A 9/22-9/26/2025 FDA inspection at the Henan Lvyuan Pharmaceutical Company drug manufacturing facility in Henan, China, found significant deviations from current good manufacturing practice (CGMP) regulations for active pharmaceutical ingredients (API). A 3/26 Warning Letter says the specific violations were: * failure to properly maintain buildings
Drugs
Researchers at the University of Cincinnati have launched a new study to investigate whether widely used diabetes and weight-loss drugs could help prevent or delay Alzheimer’s disease, a condition affecting more than 6 million Americans. Backed by two-year grant from the National Institute on Aging, part of
Drugs
FDA has denied as unwarranted a 3/1/2024 petition submitted by Aimed Alliance asking that the agency issue a guidance with an interpretation and a clear position statement about drug importation activities conducted by alternative funding programs. Specifically, the petition said the proposed guidance should address: * whether third-party
Drugs
FDA has issued a safety alert warning that patients taking Amgen’s Tavneos (avacopan) for a rare autoimmune condition may face a risk of severe liver injury, including cases that have resulted in death. The agency says it has identified postmarketing reports of drug-induced liver injury (DILI) in patients
Drugs
While FDA has been taking a more aggressive approach to publicizing its criticisms of drugmakers, the shift is drawing both praise from investors and concern across the pharmaceutical industry, according to a 3/30 analysis by PharmaVoice. The agency’s evolving stance, which is part of the Trump administration’s
Drugs
FDA inspection records show that some generic drug manufacturers are routinely failing to carry out basic safety and quality checks, raising concerns about the integrity of medications reaching U.S. patients, according to a new report published March 30 by the MedShadow Foundation. The analysis, authored by Emma Yasinski, found
Drugs
FDA has accepted for priority review a Praxis Precision Medicines NDA for relutrigine, an investigational therapy targeting rare genetic epilepsies. The agency set a user fee review action target date of 9/27. Relutrigine is being developed to treat developmental and epileptic encephalopathies (DEEs) caused by mutations in the SCN2A
Drugs
FDA has approved a higher-dose regimen of Biogen’s Spinraza (nusinersen) for treating spinal muscular atrophy, giving patients and clinicians a new dosing option aimed at improving outcomes in the rare neuromuscular disorder. The decision is based on data from the Phase 2/3 DEVOTE study and expands on
Drugs
Merck says new Phase 3 data show its investigational cholesterol drug enlicitide significantly lowered “bad” cholesterol compared with commonly used non-statin pills, positioning the therapy as a potential oral alternative to injectable PCSK9 inhibitors. Results from the CORALreef AddOn trial, presented at the American College of Cardiology Annual Scientific
Drugs
FDA has issued an untitled letter to STAQ Pharma of Ohio, citing significant manufacturing, quality control, and labeling violations that could put patients at risk. The just-posted 12/5/2025 letter follows an FDA inspection conducted in May at the company’s Columbus, OH facility, which is registered as
Drugs
A new CDER guidance recap podcast explains the newly published FDA draft guidance on the use of Bayesian methodology in clinical trials of drug and biological products. On the podcast, CDER Office of Biostatistics master mathematical statistician James Travis says the guidance is important because, as the number of proposals
Drugs
At the 2/26 PDUFA 8 reauthorization meeting, FDA said it would consider an industry counterproposal to the agency’s America First fee incentive proposal. Meeting minutes indicate that industry representatives said FDA’s proposed three-time fee differential would be “extremely punitive rather than an incentive for substantial changes
Drugs
In an FY 2025 GDUFA Science and Research Report, GDUFA Science and Research Program joint directors Iilun Murphy and Michael Kopcha say extended GDUFA-funded research on bioequivalence (BE) risk assessments and Biopharmaceutics Classification System classification supported the FY 2025 publication of over 800 product-specific guidances that aligned FDA’
FDA Policy/General
University of Southern California Price School of Public Policy associate professor Genevieve Kanter says policymakers should consider conflict of interest policies to be part of a broader strategy of building public trust by being more trustworthy as an agency. “This includes providing comprehensive scientific justification for decisions, being transparent about
Drugs
The National Association of Manufacturers (NAM) is petitioning FDA to follow the statutory and regulatory procedures governing advisory committees under regulations and the Federal Advisory Committee Act “to allow for mandatory and all other stakeholders to comment on current and upcoming regulatory policies and procedures that FDA is discussing during
Drugs
A conservative-leaning advocacy group is taking aim at FDA, arguing in a new report that the agency’s drug review process has grown more costly and restrictive — potentially slowing patient access to new therapies. In a report titled “Blocking Breakthroughs: Delays and Denials at the FDA,” the Taxpayers Protection
Drugs
Kodiak Sciences says its anti-VEGF therapy Zenkuda (tarcocimab tedromer) hit the mark in a second Phase 3 trial in diabetic retinopathy, reinforcing a dataset the company says is now ready to support a BLA. In the GLOW2 study, 62.5% of patients treated with Zenkuda achieved at least a
Drugs
Health experts say a new FDA policy framework could mark a turning point for highly individualized genetic medicines, as regulators attempt to keep pace with technologies that can now design treatments for single patients. In a commentary published in JAMA Health Forum, former FDA commissioner Scott Gottlieb and regulatory expert
Drugs
Wave Life Sciences is reporting new interim Phase 1 data Thursday indicating that a 240 mg dose of its investigational obesity therapy WVE-007 produced stronger and more consistent reductions in key fat measures than a higher 400 mg dose, raising questions about dose-response dynamics in the early-stage
Drugs
Rezolute is trying to chart a path forward for its congenital hyperinsulinism program after its Phase 3 trial of ersodetug missed the primary endpoint, with FDA now asking for a deeper dive into the data before weighing next steps. Following a Type B meeting earlier this month, the agency encouraged
Drugs
Quoin Pharmaceuticals says FDA has signaled openness to a streamlined development path for its rare disease candidate QRX003, indicating that a single Phase 3 trial could be sufficient to support approval in Netherton Syndrome. The feedback came out of a recent Type C meeting, where regulators also suggested flexibility on