Drugs
FDA has published the International Council for Harmonization (ICH) E2D(R1) guidance, Postapproval Safety Data: Definitions and Standards for Management and Reporting of Individual Case Safety Reports. The document says it is important to establish an internationally standardized procedure to ensure the quality of postapproval safety information and to harmonize,
Drugs
FDA has just posted the form FDA-483 issued following a 1/15-1/19/2024 inspection at the Fukuzyu Pharmaceutical Co. facility manufacturing active pharmaceutical ingredients (API) in Toyama, Japan. The inspection observations were: * failure of the firm’s quality unit to always conduct investigations for out-of-specification
Drugs
FDA has cited a Novo Nordisk direct-to-consumer advertisement for its blockbuster diabetes drug Ozempic (semaglutide), calling the ad misleading and violating federal drug promotion rules. Specifically, the 2/26 untitled letter from CDER Office of Prescription Drug Promotion (OPDP) says a promotional video titled “Ozempic vs. – Random Guy
Biologics
A just-released complete-response letter is shedding light on FDA’s rejection last month of Regenxbio’s BLA for its gene therapy clemidsogene lanparvovec, citing fundamental concerns about the study population, choice of control, and use of a novel cerebrospinal fluid biomarker as the basis for accelerated approval. The
Drugs
HHS secretary Robert F. Kennedy Jr. said last week that FDA will soon make about 14 peptides “more accessible.” Politico reports Kennedy commented on Joe Rogan’s podcast. Politico says the move will please many of Kennedy’s Make America Healthy Again followers who promote the amino acids for a
Biologics
uniQure says FDA does not agree that data from the company’s Phase 1-2 studies — compared against an external control cohort — are sufficient to constitute the primary evidence of effectiveness required for a BLA for its Huntington’s disease gene therapy AMT-130. The agency strongly recommended that a
Drugs
Merck says that adding Welireg (belzutifan) to Keytruda as adjuvant therapy significantly reduced the risk of disease recurrence or death in certain patients with earlier-stage renal cell carcinoma (RCC), marking the first positive Phase 3 study of the HIF-2α inhibitor in early-stage disease. Results from the pivotal
Drugs
FDA has accepted for priority review a Takeda and Protagonist Therapeutics NDA for rusfertide, positioning the drug as a potential first-in-class treatment for adults with polycythemia vera (PV). The agency has set a user fee review action target date for the third quarter. Rusfertide is described as a
Biologics
Pfizer CEO Albert Bourla on Monday publicly criticized the leadership of FDA’s biologics center, saying the company “has a problem” with the agency’s top vaccines regulator. Speaking at the TD Cowen healthcare conference, Bourla said CBER director Vinay Prasad was dismissing the recommendations of career agency scientists. “I
Drugs
FDA has lifted a clinical hold against Intellia Therapeutics and its MAGNITUDE Phase 3 trial of nexiguran ziclumeran (nex-z), an experimental CRISPR-based therapy for patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The agency originally placed clinical holds on the MAGNITUDE and MAGNITUDE-2 trials in 10/2025
FDA Policy/General
Three former senior FDA officials have told a federal appeals court that the Trump administration mischaracterized the science on contraceptive safety when it expanded religious and moral exemptions to the Affordable Care Act’s birth control coverage mandate. In an amicus brief filed with the U.S. Court of Appeals
FDA Policy/General
HHS has directed FDA and other health agency employees to immediately stop using the artificial intelligence (AI) platform Claude, disabling access across its enterprise systems after the Trump administration labeled parent company Anthropic a “supply chain risk,” according to a post on NOTUS.org. In an email sent Monday by
Devices
FDA has approved a Synergy Spine Solutions PMA for its Synergy Disc, a motion-preserving artificial cervical disc indicated for single-level use between C3 and C7. The approval allows the device to be marketed for patients with degenerative disc disease (DDD) of the cervical spine requiring replacement at one
Drugs
FDA’s draft guidance endorsing minimal residual disease (MRD) as a potential basis for accelerated drug approvals in multiple myeloma marks a watershed moment for the field, according to leading myeloma researcher C. Ola Landgren. In an interview with Targeted Oncology, Landgren said the agency’s draft document reflects nearly
Drugs
FDA and drug industry negotiators working on the next reauthorization of the Prescription Drug User Fee Act (PDUFA) confronted lingering divisions over FDA’s “America First” proposals and the structure of hiring commitments, according to just-posted meeting minutes from a 1/29 virtual steering committee meeting. A central focus
Drugs
The U.S. Department of Justice (DoJ) says a Federal Circuit ruling on so-called generic drug “skinny labels” threatens to undermine the Hatch-Waxman framework for bringing generics to market by exposing compliant generic manufacturers to inducement claims based on ordinary, non-infringing behavior. A DoJ amicus brief argues
Drugs
Rare disease drug developers and patient advocates are applauding a series of new guidance documents from the U.S. Food and Drug Administration, telling Clinical Trials Arena that the policies bring long-awaited clarity to regulatory expectations and could help reduce risk in a high-stakes development landscape. Over the
Drugs
The U.S. Senate Special Committee on Aging is raising concerns about FDA’s handling of foreign drug manufacturing violations and how those decisions could affect military readiness and national security. In a 2/18 letter to Defense secretary Pete Hegseth, chairman Rick Scott (R-FL) and ranking member Kirsten
Drugs
FDA has accepted for priority review an Ionis Pharmaceuticals supplemental NDA for olezarsen for treating severe hypertriglyceridemia (sHTG). It's currently approved as Tryngolza for familial chylomicronemia syndrome in the U.S. The agency set a user fee review action target date of 6/30. Ionis said the submission’
Drugs
Eli Lilly says its investigational oral GLP-1 receptor agonist orforglipron delivered significantly greater reductions in blood sugar and body weight than oral semaglutide (Novo Nordisk’s Rybelsus) in a 52-week head-to-head Phase 3 trial of adults with Type 2 diabetes. Results from the ACHIEVE-3 study,
FDA Policy/General
FDA commissioner Marty Makary is defending the agency’s scientific standards and personnel, particularly CBER director Vinay Prasad, amid mounting political and investor criticism. In a 2/26 CNBC interview about recent FDA review policy changes (e.g., not requiring two pivotal trials for approval, and this week’s release
Drugs
FDA has granted Boehringer Ingelheim Pharmaceuticals an accelerated approval for Hernexeos (zongertinib) for adults with unresectable or metastatic non-squamous non-small cell lung cancer (NSCLC) harboring HER2 (ERBB2) tyrosine kinase domain-activating mutations, as detected by an FDA-authorized test. Zongertinib is a kinase inhibitor evaluated under the FDA