Drugs
FDA is reminding companies and the public that they have until 9/19 to submit comments on a draft guidance entitled E21 Inclusion of Pregnant and Breastfeeding Women in Clinical Trials. The document provides recommendations for the appropriate inclusion and retention of pregnant or breastfeeding women in clinical trials. The
Devices
FDA has cleared a Microbot Medical 510(k) for its Liberty Endovascular Robotic System, a single-use, remotely operated device designed for peripheral endovascular procedures. The pivotal trial supporting clearance demonstrated 100% success in robotic navigation to the target with no device-related adverse events, as well as a 92%
Biologics
The latest Make America Healthy Again (MAHA) Commission report, “Make Our Children Healthy Again: Assessment,” says FDA has a role to play in working with the National Institutes of Health to “build systems for real-world safety monitoring of pediatric drugs and create programs to independently replicate findings from industry-
Biologics
FDA has issued two final guidances related to biosimilar development. The final guidance Development of Therapeutic Protein Biosimilars: Comparative Analytical Assessment and Other Quality-Related Considerations provides recommendations on the design and evaluation of comparative analytical studies intended to support a demonstration that a proposed therapeutic protein product is biosimilar
Devices
Kimberly-Clark has agreed to pay $40 million under a deferred prosecution agreement to resolve a criminal charge that it sold adulterated MicroCool surgical gowns, according to the U.S. Department of Justice. Federal prosecutors accused the Dallas-based company of introducing the defective gowns into interstate commerce with intent
FDA Policy/General
Former FDA commissioner Scott Gottlieb is warning that HHS secretary Robert F. Kennedy Jr. may attempt to use a forthcoming HHS report on autism to challenge the scientific basis of the nation’s pediatric vaccine program. Speaking on CNBC 9/8, Gottlieb said he believes Kennedy intends to link aluminum
Drugs
FDA has issued an updated guidance entitled E6(R3) Good Clinical Practice (GCP) that describes GCP principles, emphasizing flexible, modern approaches to clinical trial design and conduct while maintaining participant safety and data integrity. The guidance, aligned with the International Council for Harmonization, notes that well-designed and well-executed
Drugs
Eli Lilly’s Bruton tyrosine kinase (BTK) inhibitor Jaypirca (pirtobrutinib) significantly extended progression-free survival in previously untreated patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), according to topline results from a Phase 3 study. The company says the BRUIN CLL-313 trial compared pirtobrutinib to chemoimmunotherapy
Drugs
Merck is reporting favorable results from the IDeate-Lung01 Phase 2 trial showing that ifinatamab deruxtecan (I-DXd) demonstrated clinically meaningful response rates in patients with previously treated extensive-stage small cell lung cancer (ES-SCLC). The potential first-in-class B7-H3 directed DXd antibody drug conjugate is being
Drugs
FDA has issued Saol Therapeutics a complete response letter (CRL) on its NDA for SL1009 (sodium dichloroacetate oral solution) for treating an orphan pediatric mitochondrial disease, Pyruvate Dehydrogenase Complex Deficiency (PDCD). There are about 1,000 children in the U.S. with PDCD, a rare and life-threatening genetic disorder
Drugs
FDA has launched a new “green list” import alert aimed at preventing unapproved and potentially unsafe GLP-1 drug ingredients from entering the U.S. The move comes as demand surges for drugs such as semaglutide and tirzepatide, which are FDA-approved for weight management and Type 2 diabetes. The
Drugs
FDA has granted CERo Therapeutics fast track status for its lead investigational compound, CER-1236, for treating acute myeloid leukemia. A first-in-human Phase 1/1b study, currently underway, is designed to evaluate the safety and preliminary efficacy of CER-1236 in patients with acute myeloid leukemia that is
Drugs
FDA’s recent use of ad hoc “expert panels” in place of traditional advisory committees risks undermining scientific rigor and public trust, according to an online opinion piece in STAT by Johns Hopkins Bloomberg School of Public Health’s drug safety expert G. Caleb Alexander. In recent months, the agency
Drugs
FDA says an evaluation of reports contained in its vaccines’ adverse event database shows there have been Covid-19 vaccine-related deaths in children and a “proper investigation” is underway that will generate a releasable report in the coming weeks. In a 9/4 appearance on The Lead with Jake
Drugs
BioNTech and Duality Biologics say their experimental antibody-drug conjugate, trastuzumab pamirtecan (BNT323/DB-1303), met its primary endpoint in a pivotal Phase 3 trial for patients with HER2-positive metastatic or unresectable breast cancer. The China-based study compared trastuzumab pamirtecan to the approved therapy trastuzumab emtansine (T-DM1)
Drugs
FDA has extended by three months its review of Agios Pharmaceuticals’ supplemental NDA seeking to expand the use of Pyrukynd (mitapivat) to adults with thalassemia. The extension, which was triggered by a Risk Evaluation and Mitigation Strategy (REMS) proposal that the company recently submitted, pushes back the user fee review
Drugs
FDA has granted breakthrough therapy designation to Lilly’s olomorasib in combination with the anti-PD-1 therapy Keytruda (pembrolizumab) for the first-line treatment of patients with unresectable advanced or metastatic non-small cell lung cancer with a KRAS G12C mutation and PD-L1 expression at or under 50%
Drugs
FDA has released the form FDA-483 issued following a 7/14-7/19 inspection at GenoGenix, a Boca Raton, FL, outsourcing facility. The 14 inspection observations were: * failure to include validation of the process in procedures designed to prevent microbiological contamination of drug products purporting to be sterile; * failure
Drugs
Sanofi says its investigational antibody amlitelimab met all primary and key secondary endpoints in a late-stage trial of patients with moderate-to-severe atopic dermatitis, reinforcing its potential as a first-in-class therapy with dosing as infrequent as four times a year. In the global COAST 1 Phase
FDA Policy/General
Health and Human Services secretary Robert F. Kennedy Jr. was grilled over his controversial policies and actions during a contentious three-hour Senate Finance Committee hearing 9/4 marked (XXX DELETE, INSERT XXX)by sharp bipartisan criticism and heated exchanges. Senators from both parties questioned Kennedy's recent public
Drugs
FDA has said it does not plan to convene an advisory committee to discuss the Aquestive Therapeutics NDA for its Anaphylm (dibutepinephrine) sublingual film to treat severe allergic reactions. A company statement says the PDUFA action date for the NDA remains 1/31/2026. The company says Anaphylm is a
Drugs
FDA says that going forward, complete response letters (CRLs) will be released promptly after they are issued to sponsors. In an announcement, the agency also said it had released 89 previously unpublished CRLs issued from 2024 to the present, associated with pending or withdrawn applications. “This is a milestone day