Drugs
Pfizer says its Tukysa (tucatinib) achieved a statistically significant and clinically meaningful improvement in progression-free survival in the HER2CLIMB-05 trial evaluating Tukysa versus placebo, both in combination with first-line standard of care maintenance therapy (Genentech’s Herceptin (trastuzumab) plus Genentech’s Perjeta (pertuzumab) following chemotherapy-based induction
Biologics
FDA has accepted for review a Rocket Pharmaceuticals BLA resubmission for Kresladi (marnetegragene autotemcel; marne-cel), a lentiviral vector–based gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I). The agency has set a 3/28/2026 user fee review action target date. The resubmission addresses issues raised in
Drugs
FDA has approved an expanded indication for Teva and Medincell’s Uzedy (risperidone) to permit its use as monotherapy or adjunctive therapy to lithium or valproate for the maintenance treatment of adults with Bipolar I disorder. The once-monthly extended-release injectable suspension for subcutaneous use was first approved in
Drugs
Tvardi Therapeutics is reporting that its experimental drug TTI-101 failed to show benefit in a Phase 2 clinical trial for idiopathic pulmonary fibrosis, a progressive and fatal lung disease. The company said the trial did not meet its endpoints and variability in the data limited interpretation of the results.
Biologics
Regeneron Pharmaceuticals says new clinical data show its investigational gene therapy DB-OTO has restored meaningful hearing in nearly all children treated for genetic hearing loss caused by OTOF gene variants. The company plans to submit a BLA later this year after discussing its options with the agency. The data
Drugs
FDA has extended by three months its review of a Denali Therapeutics BLA for tividenofusp alfa, a potential treatment for mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome. According to the company, the review action target date has been pushed back — from 1/5/2026 to 4/5
Drugs
FDA has approved an expanded indication for Medtronic’s Endurant II/IIs Stent Graft System, allowing its use in abdominal aortic aneurysm (AAA) patients with short, challenging neck anatomies when used with the company’s Heli-FX EndoAnchor System. The approval enables treatment of patients with aortic neck lengths as
Drugs
FDA has granted BeOne Medicines a breakthrough therapy designation for sonrotoclax and its use in treating adults with relapsed or refractory mantle cell lymphoma (MCL). The designation is based on positive data from a Phase 1-2 clinical study (BGB-11417-201) evaluating sonrotoclax in patients with MCL who had
Drugs
FDA has given an “Official Action Indicated (OAI)” classification to a recent inspection of a Novo Nordisk manufacturing facility in Bloomington, IN. The July inspection revealed significant GMP deficiencies. The serious compliance designation could delay the approval and production timelines for drug products, including those from partner companies, including Regeneron
Drugs
FDA has approved an updated indication for AbbVie’s Rinvoq (upadacitinib), expanding its use in treating adults with moderately to severely active ulcerative colitis (UC) and Crohn’s disease (CD). The label update allows physicians to prescribe Rinvoq prior to tumor necrosis factor (TNF) blocker therapy for patients who have
Biologics
FDA has approved Celltrion’s Eydenzelt (aflibercept-boav), a biosimilar referencing Regeneron and Bayer’s blockbuster Eylea (aflibercept) for treating several major retinal diseases, including neovascular (wet) age-related macular degeneration, macular edema following retinal vein occlusion, diabetic macular edema, and diabetic retinopathy. FDA’s decision was based on analytical,
Drugs
FDA has approved new labeling for Janssen Biotech’s Carvykti (ciltacabtagene autoleucel, including a Boxed Warning highlighting the risk of immune effector cell-associated enterocolitis (IEC-EC), a potentially life-threatening gastrointestinal complication. The warning comes after the agency reviewed reports from clinical trials and postmarketing data documenting cases of
Devices
FDA has approved SQ Innovation’s drug-device combination Lasix ONYU (furosemide injection) for treating edema (due to fluid overload) in adult patients with chronic heart failure. The product was developed to enable subcutaneous infusion of furosemide outside the healthcare setting for selected patients, according to the company. Lasix ONYU
FDA Policy/General
Fifty-one Republican senators are urging FDA to restrict access to abortion pills following the agency’s recent approval of a new generic version of mifepristone. The letter called on the agency to suspend approval of generic mifepristone until a planned safety review (see earlier story) is completed. It also
Drugs
FDA has granted Precision NeuroMed an orphan drug designation for IL13-PE38QQR (cintredekin besudotox), an investigational therapy targeting glioblastoma multiforme, an aggressive and often lethal form of brain cancer. The cytotoxic protein is engineered to attack cells expressing the IL-13 alpha-2 receptor — a marker commonly found on glioblastoma
Drugs
FDA says it is monitoring reports of contaminated children’s cough and cold medicines manufactured in India but confirmed that the affected products have not been shipped to the U.S. An agency notice says it is aware of an ongoing investigation in India into cough syrups contaminated with diethylene
FDA Policy/General
Americans’ ratings of FDA and five other major agencies (FEMA, CIA, CDC, EPA, and IRS) “have deteriorated markedly since last year,” according to a new Gallup poll. The pollsters say the six agencies are at or near their lowest point in Gallup’s trend. The report says the only agency
Drugs
FDA has granted Adcendo a fast track designation for ADCE-D01, an investigational antibody-drug conjugate (ADC) being developed for treating soft tissue sarcoma (STS). The company says the first-in-class ADC is designed to target urokinase plasminogen activator receptor-associated protein (uPARAP) conjugated to the Topoisomerase I inhibitor
Drugs
Nearly three out of four drug candidates that received an FDA breakthrough therapy designation (BTD) over the past decade eventually gained approval, according to a new analysis from investment firm Jefferies reported by BioSpace. The report examined 599 breakthrough designations issued between 2013 and year-to-date 2025, finding that
Devices
FDA has cleared a Methinks AI 510(k) for its Methinks CTA Stroke, an artificial intelligence–based software tool that detects suspected large vessel occlusions (LVOs) using CT angiography scans. The software demonstrated 98.2% sensitivity and 91.6% specificity, including detection of distal occlusions up to the MCA-M2
Drugs
FDA released on 10/8 a new table supporting the use of new approach methodologies and reduction in animal use in drug testing. An agency notice says the table contains an inventory of drug development contexts for which CDER is open to a streamlined nonclinical program. The notice says streamlined
Drugs
FDA has released the form FDA-483 with eight observations from an 8/25-9/5 inspection at the Aurobindo Pharma drug manufacturing facility in Bachupally, Hyderabad, India. The inspection observations were: * procedures designed to prevent microbiological contamination of drug products purporting to be sterile are not established and followed,