Drugs
FDA and the Clinical Trials Transformation Initiative are hosting an 8/6 hybrid public workshop on artificial intelligence (AI) in drug and biological product development. Meeting organizers say that “AI use in drug development creates new and unique challenges that require both careful management and a risk-based regulatory framework
Drugs
FDA has placed a clinical hold on Biomea Fusion’s Phase 1-2 clinical trials of its investigational covalent menin inhibitor BMF-219 in Type 2 and Type 1 diabetes (COVALENT-111 and COVALENT-112), respectively. The agency cited deficiencies based on the level of possible drug-induced hepatotoxicity observed
Drugs
The Critical Path Institute (C-Path) says revisions in an FDA draft guidance on developing drugs to treat early Alzheimer’s disease from a 2018 draft “add helpful clarity about strategies to assist sponsors developing treatments for early-stage disease.” The comment letter says this is particularly true in terms
Drugs
The U.S. Pharmacopeia (USP) inaugural drug shortage report shows that shortages are at a decade high and are lasting longer than they did in 2020. A USP statement (includes a link to the report) says that the average drug shortage lasts for over three years, compared to about two
Drugs
Amgen is announcing positive topline results from its randomized, placebo-controlled Phase 3 clinical trial (MITIGATE) evaluating the efficacy and safety of Uplizna (inebilizumab-cdon) for treating immunoglobulin G4-related disease (IgG4-RD). Currently, the therapy is approved for neuromyelitis optica spectrum disorder. The company says the trial met its
Drugs
The Biosimilars Council, a division of the Association for Accessible Medicines, says the biosimilar development paradigm in the U.S. should be updated and streamlined to reflect new scientific understandings. In a position paper, the council says an updated development paradigm “must account for scientific advances that allow for the
Drugs
FDA has published a draft guidance, Processes and Practices Applicable to Bioresearch Monitoring Inspections, addressing the types of records and information to be provided, best practices for communication between FDA and industry in advance of or during an inspection or request for records or other information, and other inspection-related
Drugs
A January FDA inspection at Jiangsu Hengrui Pharmaceuticals in Jiangsu, China, resulted in an eight-observation Form FDA-483. Agency inspectors found that the firm’s procedures designed to prevent microbiological contamination of drug products did not include adequate validation of the aseptic and sterilization process. The inspection also found
Biologics
FDA commissioner Robert Califf and two agency colleagues say FDA is interested in pragmatic clinical research as a “middle path between traditional randomized clinical trials and observational evidence to inform decisions about the use of medical products after initial market approval.” Writing in a JAMA Viewpoint, the three say pragmatic
Drugs
Bristol Myers Squibb says data from the Phase 3 CheckMate-9DW trial evaluating the dual immunotherapy combination of Opdivo (nivolumab) plus Yervoy (ipilimumab) compared to investigator’s choice (lenvatinib or sorafenib) as a first-line treatment for patients with unresectable hepatocellular carcinoma (HCC) showed that the combination had a statistically
Drugs
The Association for Accessible Medicines (AAM) says it appreciates that FDA is issuing a draft guidance on achieving and maintaining data integrity for the clinical and bioanalytical portions of bioavailability and bioequivalence studies submitted to support INDs, NDAs, ANDAs, the bioanalytical portion of clinical pharmacologic studies supporting CDER-regulated biosimilar
Drugs
Agios Pharmaceuticals plans to submit by the end of the year an NDA for mitapivat and its use in adults with transfusion-dependent alpha- or beta-thalassemia. The move is based on just-announced Phase 3 (ENERGIZE-T) study data that met the primary endpoint of transfusion reduction response. “Statistical
Drugs
FDA has placed a partial clinical hold on a Blueprint Medicines early-stage trial testing experimental cancer drug BLU-222 because of safety concerns, according to a Reuters report. Some patients faced episodes of light sensitivity and blurred vision, the company says. The 366-participant drug dosing trial was initiated
Drugs
Takeda says it is beginning a Phase 3 trial by 6/30 of its narcolepsy Type 1 (NT1) drug TAK-861 after reporting positive results from a Phase 2b trial. Based on the data, the investigational oral orexin receptor 2 (OX2R) agonist has the potential to “provide transformative efficacy in
Drugs
FDA has extended by three months its review of a Regeneron supplemental BLA for Dupixent (dupilumab) as an add-on maintenance treatment in certain adult patients with uncontrolled chronic obstructive pulmonary disease (COPD). The company said earlier this month that the agency recently requested sub-population breakdowns from the Phase
Drugs
Gilead Sciences says it will be discussing with FDA the continued marketing of its accelerated-approval Trodelvy (sacituzumab govitecan-hziy) in patients with metastatic urothelial cancer following topline results from a Phase 3 confirmatory trial that showed the therapy did not meet the primary endpoint of overall survival (OS) in
Drugs
An FDA advisory committee briefing document shows reviewer concerns about safety and efficacy issues in a Lykos Therapeutics NDA for midomafetamine (MDMA) capsules for a proposed indication of treating post-traumatic stress disorder (PTSD). The agency’s Psychopharmacologic Drugs Advisory Committee is set to convene 6/4 to discuss the
Drugs
FDA has approved Bristol Myers Squibb’s Breyanzi (lisocabtagene maraleucel; liso-cel) for treating adult patients with relapsed or refractory mantle cell lymphoma (MCL) who have received at least two prior lines of systemic therapy, including a Bruton tyrosine kinase (BTK) inhibitor. The. company says this is the fourth subtype
Drugs
Attorney James Beck (Reed, Smith) says the 5th Circuit Court of Appeals issued 5/24 a “defense-friendly” decision in Hickey v. Hospira that reversed in large part a decision by a Louisiana federal court. Beck says the appeals court applied the same implied preemption analysis to Section 355(b)
Drugs
FDA has denied a 4/2019 Novartis petition asking the agency to take specified steps regarding ANDAs that reference the company’s Entresto (sacubitril and valsartan tablets). The agency’s 5/28 denial letter says Novartis asked FDA to: * require ANDAs that reference Entresto to demonstrate active ingredient sameness based
Drugs
CDER Office of Medical Policy director Khair ElZarrad says the use of artificial intelligence (AI) in drug regulatory submissions has seen a rapid increase in recent years. Interviewed on a Q&A with FDA podcast, ElZarrad said that from 2016 to 2024, some 300 submissions were received that reference
Drugs
Federal Register notice: FDA is withdrawing approval of three NDAs from multiple applicants after they notified the agency that the products were no longer marketed and requested their withdrawal. The products are: * Bayer Healthcare’s Cipro in Dextrose 5% in plastic container (ciprofloxacin) Injectable, 200 mg/100 mL and 400