Novo Nordisk has received a Warning Letter from the US Food and Drug Administration (FDA) related to violations of postmarketing adverse drug experience (PADE) regulations. The letter relates to an inspection that took place in early 2025, in which the FDA found serious violations of PADE reporting… Read More
The US Food and Drug Administration (FDA) has placed a clinical hold on REGENXBIO’s investigational gene therapy, RGX-111, for the treatment of MPS I, following a single case of neoplasm (intraventricular CNS tumour) in a participant treated in its Phase I/II study. The FDA also placed a… Read More
Viking Therapeutics has released positive top-line results from its Phase II clinical trial of the oral tablet formulation of VK2735, the company’s dual agonist of the glucagon-like peptide 1 (GLP-1) and glucose-dependent insulinotropic polypeptide (GIP) receptors. VK2735 is being developed in both oral and subcutaneous formulations for… Read More
This is the latest episode of the free DDW narrated podcast, titled “Innovation enabled by new drug discovery technologies”, which covers two articles written for DDW Volume 24 – Issue 3, Summer 2023. They are called: “CRISPR breakthroughs: New solutions for common diseases” and “Re-assessing the risks of… Read More
A new study has demonstrated that the frequency of second primary malignancies (SPMs) is no higher with CAR-T cell therapies than with previous standard-of-care strategies. The authors found that T cell malignancies represented a small minority of adverse events at just 1.5%. The paper ‘… Read More
A new study has suggested a link between semaglutide (the active ingredient in Wegovy and Ozempic) and a disproportionately increased reporting of suicidality. Semaglutide has recently been treated as something of a wonder drug, with studies demonstrating it can reduce the risk of cardiovascular disease,… Read More
The US Food and Drug Administration (FDA) has announced its final decision to withdraw approval of Pepaxto (melphalan flufenamide), despite an appeal by manufacturer Oncopeptides. The drug was originally approved for use in combination with dexamethasone to treat some patients with multiple myeloma. Approval… Read More
The International Society for Cell & Gene Therapy (ISCT) has announced the publication in Nature Medicine of an expert consortium commentary addressing a recent statement from the US Food & Drug Administration (FDA) that it is investigating serious risk of T-cell malignancy… Read More
South Korean researchers have found that the use of Janus kinase (JAK) inhibitors as a treatment for atopic dermatitis (AD) is not associated with a greater risk of serious adverse events. While several biologic treatment approaches have been proposed in the recent years for the treatment… Read More
The Food and Drug Administration (FDA) said it will investigate the safety of BCMA- or CD19-directed autologous CAR-T cell immunotherapies, following reports of T cell malignancies, including chimeric antigen receptor CAR-positive lymphoma, in these patients. The reports were received from clinical trials and postmarketing adverse event… Read More
A tiny human heart model could potentially transform cardiovascular research and enable animal-free drug testing. A team of researchers, led by the Hebrew University of Jerusalem (HU), has developed a self-paced, multi-chambered human heart model, approximately half the size of a rice grain. “Integrating… Read More
Listen to this article on the DDW Podcast: Can potentially lethal arrhythmias induced by novel drugs be better predicted by using more relevant and sophisticated electrophysiological screening methods? By Dr J Mark Treherne, Dr W Jack Scannell and Dr Richard C Saumarez. Single-cell, electrophysiology-based… Read More
The European Medicines Agency (EMA) safety committee, the Pharmacovigilance Risk Assessment Committee (PRAC), is reviewing data on the risk of suicidal thoughts and thoughts of self-harm related to GLP-1 receptor agonists. The drugs under review include Ozempic (semaglutide), Saxenda (liraglutide) and Wegovy (semaglutide), used for weight… Read More
A cancer patient died and three others were hospitalised in the UK after they were administered an unlicensed version of chemotherapy cabazitaxel. According to The Guardian, which reported on the incident, the problem was caused by an issue at private healthcare company Sciensus’… Read More
