Drugs
Elevar Therapeutics and China's Hengrui Pharmaceuticals have resubmitted a rivoceranib plus camrelizumab as a first-line treatment for patients with unresectable or metastatic hepatocellular carcinoma (HCC). The companies’ resubmission marks the fourth attempt to win U.S. approval, and follows three previous complete response letters (CRLs) issued in
Biologics
Congressional Democrats are questioning the Trump administration's proposal to replace combination childhood vaccines with separate injections, a policy that could require new FDA regulatory reviews because single-disease measles, mumps and rubella vaccines are not currently available in the U.S. In a 10/7 letter to CDC
FDA Policy/General
Former FDA regulatory counsel and now-partner at Spencer Fane Brian Malkin is questioning whether the agency should become more directly involved in the Trump administration's drug-pricing initiatives, raising concerns about the potential direction of FDA policy under commissioner nominee Heidi Overton, whose nomination remains pending in
Drugs
FDA granted approval to Revolution Medicines' Rasonque (daraxonrasib) for metastatic pancreatic cancer despite substantial differences in treatment responses among patients with different KRAS mutations and limited evidence of effectiveness in patients without RAS mutations, according to a newly released approval document. The 321-page review shows that FDA considered
FDA Policy/General
The Trump Administration is developing a temporary policy that would allow compounding pharmacies to dispense certain unapproved peptides despite earlier FDA scientific assessments finding insufficient evidence of their safety and effectiveness, setting up a potential conflict between administration policy and the agency's scientific reviewers. Federal officials are considering
FDA Policy/General
A new Department of Justice directive identifying violations of the Federal Food, Drug, and Cosmetic Act (FDCA) among potential targets for fraud enforcement could expose FDA-regulated firms to increased federal investigations and criminal prosecutions, according to an analysis by attorneys at Holland & Knight. The 10/1 directive outlines
Biologics
FDA has released a final guidance entitled “Postapproval Manufacturing Changes to Biosimilar and Interchangeable Biosimilar Products — Questions and Answers.” The document explains how manufacturers should document and report changes to approved biosimilars, including changes involving manufacturing processes, production facilities, dosage forms and strengths. Regarding information required for postapproval manufacturing changes,
Devices
Drugs
U.S. representative Josh Gottheimer (D-NJ) and thirty-eight of his colleagues sent a bipartisan letter to HHS and FDA urging them to establish clear, consistent standards for defining rare disease patient populations. The lawmaker is the founder and co-chair of the Congressional Sarcoidosis Caucus. “When 95% of
Biologics
FDA has posted a final guidance entitled “Questions and Answers on Biosimilar Development and the BPCI Act.” The document provides answers to common questions from prospective applicants and other interested parties regarding section 351(k) of the Public Health Service Act, which was added by the Biologics Price Competition and
Independent news and analysis of FDA regulation, policy and enforcement
FDA has cited four deaths among patients treated with Newron Pharmaceuticals’ investigational schizophrenia drug evenamide as a potential cardiac safety signal, according to new details disclosed by the company concerning the agency's continuing clinical hold on its Phase 3 ENIGMA-TRS 2 study in the U.S. A
Rep. Nick Langworthy (R-NY) has introduced the Biosimilars Access and Affordability Act, bipartisan legislation that he says would expand access to lower-cost biosimilar prescription drugs and help bring them to market. In a statement, Langworthy says the bill would update Medicare policy to provide a clearer pathway for
HHS says it is taking a series of actions to “strengthen how the federal government identifies, documents, investigates, and responds to health problems reported following vaccination, with a focus on improving patient care and generating better evidence about potential vaccine-related injuries.” A department statement says HHS: * opened a National
FDA has released a final guidance entitled “Study Data Technical Conformance Guide - Technical Specifications Document” that provides specifications, recommendations, and general considerations on how to submit standardized study data using FDA-supported data standards located in the FDA Data Standards Catalog. The document supplements the guidance “Providing Regulatory Submissions in
Three stakeholders have submitted general and specific comments to FDA on the agency’s draft guidance on considerations for container closure and device constituent parts for biosimilar and interchangeable biosimilar products. The Biosimilars Forum comment letter made these general points: * other tools, including qualitative human factor studies, effectively evaluate user
FDA has approved Genentech’s Tecentriq (atezolizumab) for combination use with a fluoropyrimidine and oxaliplatin for the adjuvant treatment of adult and pediatric patients two years of age and older with Stage III mismatch repair deficient (dMMR) colon cancer. The agency also approved atezolizumab and hyaluronidase-tqjs (Tecentriq Hybreza) in
Ascendis Pharma is petitioning FDA to require additional information sufficient to evaluate unresolved safety questions before approving infigratinib to treat children with achondroplasia. The company’s 10/6 petition asks specifically that FDA: * include internal neurodevelopmental experts on the review team to consider the risks infigratinib’s mechanism of action
Ultragenyx Pharmaceutical has agreed to sell its Rare Pediatric Disease Priority Review Voucher to an undisclosed buyer for $210 million. FDA awarded the voucher in August upon approval of Genglycos (pariglasgene brecaparvovec-opnr), also known as DTX401, for use in adult and pediatric patients eight years and older with glycogen
A new Stanford Biodesign policy paper is calling for a dozen bipartisan FDA reforms aimed at accelerating clinical trials and drug reviews, including allowing some lower-risk Phase 1 trials to begin without waiting for prior FDA approval and expanding rolling review of marketing applications. The paper, Roadmap for FDA
FDA has warned O.N. Diagnostics that it made significant changes to its VirtuOst vertebral fracture assessment software — including a new machine-learning algorithm and conversion to a web-based platform — without obtaining required agency clearance. A just-posted 9/1 Warning Letter following a March inspection of the Berkeley,
MimiVax says it plans to seek a meeting with FDA to review positive results from its Phase 2b SURVIVE trial of SurVaxM, a novel cancer vaccine for patients with newly diagnosed glioblastoma. In a statement, company officials say they want to use the meeting to review the SURVIVE data and
Biostatisticians from FDA and several universities say interest in integrating randomized clinical trials (RCTs) with real-world data (RWD) continues to grow as stakeholders seek evidence that is both internally valid and relevant to clinical practice. Writing in an open-source article in Clinical Pharmacology & Therapeutics, the researchers say,