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
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
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
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
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
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
Drugs
FDA is asking all manufacturers of generic drugs citing Accutane (isotretinoin) as the reference-listed drug. An agency statement says the updates are necessary “because new scientific evidence exists that is not reflected in current labeling, and the approved labeling does not follow the current format that better serves healthcare
Drugs
FDA and the African Medicines Agency (AMA) have signed a Statement of Cooperation establishing a formal framework for collaboration between the U.S. regulator and Africa's continental medicines agency. The agreement is intended to strengthen regulatory convergence, information sharing, and harmonization efforts as the AMA continues to build
FDA Policy/General
Two Mintz attorneys say recent leadership changes at FDA, coupled with many executive-level vacancies, have affected the priorities started by former commissioner Marty Makary. Writing in the one-year anniversary edition of their firm’s FDA in Flux newsletter, the attorneys say the leadership changes coincided with an immediate
Drugs
An FDA review of records requested by the agency on 7/7/2025 from Sante Manufacturing in Aylmer, Ontario, Canada, identified significant violations of current good manufacturing practice (CGMP) regulations for finished drugs in the firm’s manufacturing of over-the-counter drug products. A 6/5 Warning Letter says
Drugs
North Dakota State University economics professor Raymond March says there is a clear reason why FDA struggles to provide effective risk management guidance and seems to flounder when asked to manage risk for others: it can’t. Writing for the Independent Institute in an online post, March argues that “risk
Drugs
Ochsner MD Anderson Cancer Center system medical director of precision medicine Marc Matrana is calling on FDA to require a labeling change for immunotherapies used to treat non-small cell lung cancer involving a Limitation of Use to exclude patients with ROS Proto-Oncogene 1, Receptor Tyrosine Kinase (“ROS1”) and
Drugs
FDA has issued a Warning Letter to Greek contract manufacturer Pharmathen International citing significant GMP violations at its sterile drug manufacturing facility in Sapes, Greece, including deficiencies in aseptic processing, contamination control, quality investigations, facility design, and data integrity. The just-posted 5/27 letter follows an FDA inspection conducted
Drugs
FDA says there is an implementation delay for isotretinoin iPLEDGE Risk Evaluation and Mitigation Strategy (REMS) modifications. The modifications were scheduled to go into effect 8/8, but the planned implementation date has been changed to 11/15 because more time is needed to test iPLEDGE REMS system updates, minimize
Drugs
A physician has asked FDA to impose an immediate moratorium on the use of gadolinium-based contrast agents in MRI scans, arguing that a growing body of scientific literature demonstrates the agents can cause widespread and potentially permanent health effects. In a just-posted citizen petition, Regina Sutton called on
Drugs
FDA has authorized Colorado's prescription drug importation program, marking another step forward for state-led efforts to lower drug costs through imports from Canada under Section 804 of the Federal Food, Drug, and Cosmetic Act. In a 6/15 letter, the agency said the state's revised
Drugs
Eli Lilly is reporting positive Phase 3 results for its BTK inhibitor Jaypirca (pirtobrutinib) in combination with venetoclax and rituximab, showing a significant progression-free survival benefit in patients with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL). The BRUIN CLL-322 trial met its primary