Bayer has started a phase 2a trial of an antibody that it hopes could provide a targeted, potentially disease-modifying therapy for Alport syndrome, a rare, genetic disease that leads to chronic kidney disease (CKD). Alport is caused by mutations in genes that are involved in the formation of type IV collagen, a protein integral to…
The FDA has revealed the latest element in its adoption of AI, an agentic AI platform that agency staffers can use to streamline complex tasks. Source link
The EMA has started a review of a regimen based on Pfizer and Astellas’ Padcev and MSD’s Keytruda that has been described as a game-changer in the treatment of muscle-invasive bladder cancer (MIBC). If approved, the combination of nectin-4-directed antibody-drug conjugate (ADC) Padcev (enfortumab vedotin) and PD-1 inhibitor Keytruda (pembrolizumab) could be used as a…
Belite Bio has said the results of the DRAGON trial of its oral therapy for sight-robbing disorder Stargardt disease type 1 (STGD1), tinlarebant, could support an FDA filing. The San Diego biotech said that DRAGON is the first successful pivotal trial in patients with STGD1, an eye disease that leads to progressive vision loss, usually…
A real-world study exploring the long-term effects of Eli Lilly’s weight-loss therapy tirzepatide in primary care settings has started in the UK. The five-year SURMOUNT-REAL UK trial, part of a collaboration signed last year between Lilly and the UK government, will chart the effects of tirzepatide on weight loss, diabetes prevention, and the prevention of…
Reports have emerged that Vinay Prasad, head of the FDA’s Center for Biologics Evaluation and Research (CBER), is planning to tighten up the regulations surrounding vaccines after concluding that COVID-19 shots resulted in the deaths of 10 children. The source of the reports is an internal email to CBER staff in which Prasad said that,…
Prostate cancer screening should not be made available to the vast majority of men across the UK, according to a committee set up to recommend a screening policy for the health service. Rather, the UK National Screening Committee (NSC) has recommended a targeted approach, only for men with a confirmed BRCA1 or BRCA2 gene variant…
Medical charity Wellcome has provided £2 million ($2.6 million) in additional funding to the UK’s MHRA medicines regulator and reimbursement authority NICE, to help advance the development of digital health technologies (DHTs) for mental health. The top-up cash will be used to continue an ongoing project by the MHRA and NICE to develop new regulatory…
Following its FDA approval in the summer, Tonix Pharma’s fibromyalgia therapy Tonmya has been launched onto the market in the US, becoming the first new drug for the painful condition in more than 16 years. Tonmya (cyclobenzaprine HCl) is a new 5.6 mg sublingual (under-the-tongue) formulation of a well-established tricyclic muscle relaxant drug, and has…
The FDA has started a priority review of BeOne Medicines’ BCL2 inhibitor sonrotoclax in relapsed or refractory mantle cell lymphoma (MCL), setting up a decision on US approval next year. Sonrotoclax is in the same class as AbbVie’s Venclexta (venetoclax), which isn’t approved to treat MCL, but has FDA clearance for various indications in chronic…