Biologics
FDA researchers say there have been global efforts to streamline biosimilar development of therapeutic proteins (TPs) by reducing reliance on comparative efficacy studies and moving instead toward a comparative analytic assessment plus pharmacokinetics approach. Writing in the open-source Clinical Pharmacology & Therapeutics, the researchers say the new approach is
Drugs
FDA's updated operating framework for drug facility inspections makes several procedural changes affecting inspection classifications, remote assessments and the use of foreign regulatory inspection reports, according to an analysis by Hogan Lovells. The revised Concept of Operations (ConOps 2.0) replaces the agency's 2017 framework governing
Drugs
A 4/7-4/17 FDA inspection at the USV Private Limited drug manufacturing facility in Dabhel, Daman, Dadra and Nagar Haveli and Dam, India, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. An 11/2 Warning Letter says the specific violations were: · failure to
Biologics
BIO says the window to protect America’s biotechnology lead is closing and it offers a seven-step plan to ensure the U.S. remains the global biotech leader for decades to come. In a white paper, “Bio Advance: Securing America’s Biotech Century,” the trade association says the biotechnology
Drugs
Cingulate says it has resubmitted its NDA for CTx-1301, an investigational treatment for attention-deficit/hyperactivity disorder. The filing addresses issues the agency raised in a June-issued complete response letter, which reportedly were limited to requests for additional chemistry, manufacturing and controls (CMC) information, according to the company.
Drugs
Drugs
FDA is recommending less restrictive patient eligibility criteria and more flexible dose-escalation strategies in early-phase clinical trials for solid tumors, under a new draft guidance addressing common deficiencies identified during the agency's review of INDs. The draft guidance, Early-Phase IND Protocol Recommendations for Solid Tumors,
Drugs
CDER and CBER are seeking industry input on how to overcome legal, privacy and technical barriers that prevent them from accessing patient-level data used to support product applications containing real-world evidence (RWE). In a request for information (RFI) scheduled for publication in the 11/12 Federal Register, FDA
FDA Policy/General
Former senior HHS adviser Calley Means has accused FDA commissioner nominee Heidi Overton of pressuring former commissioner Marty Makary to approve Moderna's mRNA influenza vaccine despite his scientific objections, alleging that she conditioned assistance on other policy matters on his willingness to approve the product. Means resigned 10/
Devices
FDA has issued Novocure a Not Approvable Letter for its PMA for Optune Mya for treating adult patients with brain metastases from non-small cell lung cancer following stereotactic radiation. The said the agency determined that uncertainty around the degree of clinical benefit provided by Optune Mya prevented approval, according
Independent news and analysis of FDA regulation, policy and enforcement
FDA has posted 20 Form 483 inspection records (see below), including reports involving Novo Nordisk, AnaptysBio, Protagonist Therapeutics, Ascendis Pharma and Charles River Laboratories, as the agency continues expanding public access to inspection findings through its electronic reading room. Their availability is part of the agency’s expanded transparency initiative
Democratic control of Congress following the 11/3 midterm elections could bring increased oversight of FDA and HHS, complicate the nomination of a permanent FDA commissioner and turn the 2027 user fee reauthorization into a vehicle for broader health policy legislation, according to FDA policy analyst and former HHS official
FDA is requiring manufacturers of all glucagon-like peptide-1 receptor agonist (GLP-1 RA) drugs to add warnings about a potential increased risk of non-arteritic anterior ischemic optic neuropathy (NAION), a rare eye condition that can cause sudden, permanent vision loss. NAION typically causes acute, painless vision loss
Boehringer Ingelheim says it will seek global approvals for survodutide, which it describes as the first glucagon/GLP-1 dual agonist to demonstrate a favorable cardiovascular safety profile in a Phase 3 trial alongside significant cardiometabolic benefits. The company says its SYNCHRONIZE-CVOT trial met its primary endpoint using both
FDA is seeking public input on how to evaluate the benefits and risks of medical robots capable of performing tasks autonomously or being operated remotely, as advances in artificial intelligence and connectivity expand their potential clinical applications. In a just-posted discussion paper, CDRH identifies regulatory questions involving autonomous decision-
More than 90% of public comments submitted during five FDA drug advisory committee reviews favored approval of the products under consideration, according to a new study examining public participation in the agency's regulatory decision-making process. The study, published 11/7 in Therapeutic Innovation & Regulatory Science, analyzed
FDA is investigating the safety of Novartis’ Zolgensma, an adeno-associated virus (AAV) vector-based gene therapy for spinal muscular atrophy (SMA), based on postmarketing adverse event reports of malignancies in treated patients. The adverse events include confirmed reports of “product-related insertional oncogenesis involving the GLI1 (Glioma-Associated Oncogene
Long-term clinical trial extension studies can provide important evidence about drug safety and durability of treatment effects, but their findings may be compromised by selection bias, patient attrition and the loss of randomized comparison groups, according to a 10/7 JAMA editorial. The editorial highlights a challenge for FDA
Chemistry, manufacturing and controls (CMC) concerns accounted for 58% of the topics raised in FDA information requests during reviews of seven adeno-associated virus (AAV) gene therapy applications, according to an analysis by agency scientists published 11/6 in Nature Reviews Drug Discovery. The analysis, conducted by CBER researchers, examined
As FDA moves to reduce animal testing requirements for certain drugs, researchers say emerging technologies involving human organoids, organ-on-a-chip systems and artificial intelligence could replace some traditional animal studies, although significant scientific and regulatory limitations remain. In a Perspective published 11/2 in The Innovation, researchers from
FDA officials remain confident that Congress will reauthorize the agency's four major user fee programs before they expire next September, although Democratic control of Congress and the prospect of Sen. Bernie Sanders (I-VT) chairing the Senate HELP Committee could complicate the reauthorization over the already-negotiated agreements.
Merck's investigational GLP-1/glucagon receptor dual agonist efinopegdutide (MK-6024) demonstrated higher rates of MASH resolution and liver fibrosis improvement than placebo in a Phase 2b trial involving patients with metabolic dysfunction-associated steatohepatitis (MASH) and moderate to advanced fibrosis. The results, presented at the American Association