Biologics
FDA has approved Novartis’ Itvisma (onasemnogene abeparvovec-brve), a new formulation of a gene therapy for spinal muscular atrophy (SMA), allowing the treatment for adults and children two years of age and older with confirmed mutations in the SMN1 gene. Itvisma contains the same active ingredient as Zolgensma, the intravenous
FDA Policy/General
FDA has issued a draft guidance, Cross-Center Master Files: Where to Submit, with recommendations for master file holders about to which Center a master file is to be submitted (1) that is referenced in and intended to support more than one regulatory submission for which the lead Center for
FDA Policy/General
National Center for Health Research president Diana Zuckerman says FDA commissioner Marty Makary’s plan to rely on “spontaneous [and] robust” expert panels rather than advisory committees misses the point on the value of advisory committees and undermines the credibility of agency decisions. Writing in a Medpage Today opinion column,
Drugs
Novo Nordisk is reporting favorable Phase 2 data for its investigational dual-action obesity and diabetes drug amycretin, showing significant reductions in both weight and blood sugar among adults with Type 2 diabetes inadequately controlled on standard therapies. The study marks the first time amycretin has been tested in a
FDA Policy/General
A new analysis of nearly a decade of FDA advisory committee meetings reveals that a substantial portion of public speakers support drug approval — and many have financial conflicts of interest (COI), according to research published in JAMA Internal Medicine. Researchers from Mass General Brigham examined transcripts from 161 FDA human-
Drugs
FDA has released the form FDA-483 with six observations from a 6/23-7/14 inspection at the Catalent Indiana sterile drug manufacturing facility in Bloomington, IN. FDA gave an “Official Action Indicated (OAI)” classification to the inspection that could delay the approval and production timelines for drug products,
Drugs
FDA has cited PharmaTher over alleged misleading promotional claims for its generic ketamine. An agency review found the company’s Web page omitted required risk information about the drug product. It also claimed the generic ketamine was approved “for surgical pain management” or “pain and surgery management,” language that deviates
Drugs
Novo Nordisk says data from two global Phase 3 trials, evoke and evoke+, of semaglutide (Ozempic and Wegovy) have failed to slow clinical progression in adults with early-stage symptomatic Alzheimer’s disease over two years. The trials enrolled 3,808 people aged 55 to 85 with mild cognitive impairment
Drugs
U.S. House and Senate health committee leaders are voicing concern about a new FDA review pilot program because it appears designed to enable corruption and reward President Trump’s allies with faster drug approvals. In an 11/20 letter to FDA commissioner Marty Makary, House Energy and Commerce Committee
Drugs
FDA investigators have cited Molecular PharmaGroup, an outsourcing facility in New Providence, N.J., for multiple sterility and quality-control deficiencies after a two-week inspection uncovered gaps in aseptic processing, environmental monitoring, and product quality review. Inspectors from the agency’s New Jersey District Office visited the facility between
Devices
FDA has issued an early alert warning about a potentially high-risk defect in certain Fresenius Kabi Ivenix large volume pump (LVP) primary administration sets that could cause dangerous reverse fluid flow and delay critical medication delivery. The issue involves a misassembly in one lot of administration sets in which
Drugs
Johnson & Johnson has decided to discontinue its Auτonomy Phase 2b Alzheimer’s study after interim data showed its experimental antibody posdinemab failed to produce a statistically significant benefit in slowing the clinical decline of early-stage Alzheimer’s disease. The company described the trial — which it billed as a
Drugs
Former FDA principal deputy commissioner Janet Woodcock and policy experts from Duke University are calling on the agency to adopt new tools and pathways to support evidence-based drug repurposing, arguing that the current system — built around commercial drug sponsors — prevents many proven new uses of generic medicines from ever
Biologics
FDA is investigating the death of a pediatric patient with congenital thrombotic thrombocytopenic purpura (cTTP) who developed neutralizing antibodies to the enzyme ADAMTS13 after treatment with Takeda’s Adzynma, a recombinant ADAMTS13 therapy approved in 2023 for the rare, life-threatening blood disorder. In a new safety communication, the agency
Drugs
FDA investigators have cited Immacule Lifesciences Private Limited, a sterile drug contract manufacturer in Himachal Pradesh, India, for extensive lapses in aseptic manufacturing, equipment design, environmental monitoring, and contamination prevention during a 10-day inspection in September. The findings are detailed in a just-posted lengthy FDA Form 483. Investigators
FDA Policy/General
The White House recently requested the resignation of a senior aide to FDA commissioner Marty Makary, highlighting growing friction between FDA leadership and Trump Administration senior staff. The aide, Sanjula Jain-Nagpal, had been slated for a promotion to deputy chief of staff, which Makary had advocated, but the White
Devices
Intersurgical has recalled (Class 1) its i-View Video Laryngoscope, citing serious safety risks that could potentially lead to severe injury or death. The recall affects devices with model number 8008000 and serial numbers 1240555 and 1240793. The recall was prompted by reports that the devices may fail to turn
Drugs
Leading health policy and regulatory experts are raising concerns over a new FDA review program that they say could politicize drug approvals and undermine scientific standards. The agency’s National Priority Voucher program is designed to accelerate FDA review times for certain drugs to as little as one to two
Drugs
FDA has posted a Federal Register notice finalizing the withdrawal of Intercept Pharmaceuticals’ Ocaliva (obeticholic acid) from the U.S. market for treating primary biliary cholangitis (PBC), following an FDA request that cited liver injury concerns. Last December, FDA issued a drug safety communication, saying its review of postmarket clinical
Devices
FDA has issued a correction notice for NOxBOX Ltd.’s NOxBOXi Nitric Oxide Delivery System after reports of dosing fluctuations when the device is used with the Bunnell LifePulse High Frequency Jet Ventilator and possibly other conventional ventilators. The agency warned that the problem can interrupt nitric oxide therapy and
Drugs
FDA has approved Merck’s checkpoint inhibitor pembrolizumab — administered as either Keytruda or the subcutaneous formulation Keytruda Qlex — in combination with Astellas and Pfizer’s antibody drug conjugate Padcev (enfortumab vedotin-ejfv) as a perioperative treatment for adults with muscle-invasive bladder cancer (MIBC) who are not eligible for cisplatin
Drugs
FDA has issued an untitled letter to SK Life Science, raising concerns over a direct-to-consumer television advertisement for its seizure medication Xcopri (cenobamate tablets). According to the just-posted letter, CDER Office of Prescription Drug Promotion (OPDP) determined that the TV ad, titled “Road to Reduction XCOPRI DTC