FDA Policy/General
HHS has launched a broad, department-wide initiative (Operation TrialBlazer) aimed at speeding clinical research, reducing regulatory barriers, and attracting more clinical trials back to the U.S. A centerpiece of the initiative is a new FDA proposal to establish a pilot program designed to shorten the timeline between drug
Drugs
FDA has released a draft guidance outlining how sponsors can use quantitative systems pharmacology (QSP) models to determine starting doses for first-in-human (FIH) clinical trials, particularly for higher-risk therapies where traditional animal-based approaches may be less predictive. The document focuses on using QSP models to estimate
Drugs
FDA has issued a revised draft guidance that updates how sponsors can demonstrate "substantial evidence" of effectiveness for new drugs and biologics, reflecting advances in clinical trial design, real-world evidence, and precision medicine. The document is part of an HHS and FDA initiative (see story) to help
Drugs
FDA has released a revised draft guidance aimed at helping sponsors design and conduct clinical trials using master protocols, a trial framework increasingly used to evaluate multiple therapies, diseases, or patient populations within a single overarching study. The draft guidance, Master Protocols for Drug and Biological Product Development, replaces a
Drugs
Regenxbio has reached agreement with FDA on a path toward a potential accelerated approval of Navsunli (clemidsogene lanparvovec-sngl), the first gene therapy for treating Hunter syndrome, or mucopolysaccharidosis type II (MPS II). The company says FDA has concluded that existing clinical data for the gene therapy are sufficient to
Drugs
A new analysis published in the journal Frontiers in Pharmacology found substantial differences in how major international regulators evaluate and approve oncology drugs that first receive expedited approval from FDA, despite often reviewing largely the same clinical evidence. Researchers from Ewha Womans University in South Korea and Keio University in
FDA Policy/General
Former FDA commissioner Scott Gottlieb is urging federal health officials to restore scientific leadership at the agency's medical product centers, arguing that recent political appointments and workforce losses have weakened the agency’s effectiveness and credibility. In a commentary published in JAMA Health Forum, Gottlieb said FDA has
Drugs
Former FDA Office of New Drugs (OND)director Peter Stein sharply criticized the agency's National Priority Voucher (NPV) program at the DIA Global Annual Meeting in Philadelphia last week, arguing that its one- to two-month review timeline undermines the rigor of FDA's drug review process.
Drugs
An opinion article is calling on FDA acting commissioner Kyle Diamantas to establish a formal regulatory framework for evaluating vaccines as potential interventions to prevent dementia, arguing that the absence of clear guidance is hindering the launch of large-scale clinical trials. In an open letter published by Clinical Trial
Drugs
Merck says FDA has approved an expanded indication for its pneumococcal conjugate vaccine, Capvaxive, allowing its use in children and adolescents aged 2 to 17 years who have completed a primary pneumococcal vaccination series and are at increased risk for pneumococcal disease due to chronic medical conditions. The action makes
Biologics
FDA has given Beam Therapeutics the green light in its IND for BEAM-304, allowing the company to begin clinical testing of its experimental base-editing treatment for phenylketonuria (PKU). The clearance enables the launch of a Phase 1/2 trial evaluating BEAM-304, an in vivo gene-editing therapy
Drugs
FDA has approved the first generic version of Genentech’s Xofluza (baloxavir marboxil), clearing Norwich Pharmaceuticals' generic tablets for the treatment and prevention of influenza in patients aged five years and older. The approval covers the drug’s administration as a single oral dose for acute uncomplicated influenza. The
Biologics
The FDA Vaccines and Related Biologic Products Advisory Committee has voted to recommend approval of Moderna’s mFlusiva mRNA flu shot. The Associated Press reports the company is seeking approval for the vaccine’s use in people ages 50-64, along with authorization for use in those aged 65 and
Drugs
FDA is requesting updates to the prescribing information for testosterone replacement therapy products following what it calls a comprehensive review of clinical data and existing scientific evidence. A HHS news release says the labeling changes would: * remove the limitation of use that says the safety and effectiveness of testosterone replacement
Drugs
FDA has released an unusual internal memorandum detailing how former CDER acting director Tracy Beth Hoeg opposed approval of a Sanofi supplemental BLA for Tzield, only to have her position overturned after her departure from the agency. The sBLA was eventually approved earlier this month The just-posted memo from
Drugs
Grace Therapeutics says it plans to resubmit its NDA for GTx-104 after receiving an FDA Complete Response Letter in April, with a Type A meeting now scheduled to clarify the agency's expectations and outline a path forward for the intravenous nimodipine candidate. The letter cited outstanding manufacturing
Devices
FDA has provided additional details on its newly launched READI-Home Innovation Challenge, an initiative designed to accelerate development of medical devices intended to reduce avoidable hospital readmissions by supporting patients in home settings following discharge. The challenge, formally known as READI-Home: Reducing Readmissions through Device Innovation for the
Biologics
Sens. Jeanne Shaheen (D-NH) and Susan Collins (R-ME), co-chairs of the Senate Diabetes Caucus, are calling on FDA to improve access to deceased donor islet cell transplantation for certain individuals with Type 1 diabetes (T1D). In a letter to FDA acting commissioner Kyle Diamantas, the senators say
Devices
FDA’s release of information from the first 100 medical device inspections conducted under the new Quality Management System Regulation (QMSR) provides a real service for medical device manufacturers that treat QMSR as the inspection model it actually is. That’s the view of independent expert Marcelo Trevino writing in
Drugs
FDA has issued a guidance that is intended to assist industry in the submission of datasets from clinical studies that evaluate immunogenicity and its impact on the pharmacokinetics of a drug. It provides detailed information and specifications for these datasets. “These specifications may elicit discussions between the sponsor and reviewers
Biologics
Sen. Ron Johnson (R-WI) has launched a new investigation into federal handling of Covid-19 vaccine safety issues, seeking records from HHS, FDA and CDC related to internal discussions about adding a warning for "myocarditis with fatal outcomes" to mRNA Covid-19 vaccine labeling. In a 6/
Drugs
A petition submitted to FDA by Buchanan on behalf of an interested client asks the agency to take several actions involving Intas Pharmaceuticals and Sun Pharmaceuticals, manufacturers of generic forms of the prostate cancer drug Casodex (bicalutamide). The petition says the law firm’s client is raising potential patient safety