Brian Malkin and Sadaf Deedar, Spencer Fane LLP, unpack the recent changes to the FDA’s Priority Review Voucher scheme and the upcoming review of its effectiveness in promoting drug development for rare paediatric diseases. Congress recently enacted the Consolidated Appropriations Act, 2026 (CAA)… Read More
The US Food and Drug Administration (FDA) has granted Fast Tack Designation for an immune medicine targeting triple-negative breast cancer. RPTR-1-201 is a novel T cell receptor (TCR) bispecific immune medicine, developed by biotechnology company Repertoire Immune Medicines, that is currently in a Phase I/II trial for the… Read More
The US Food and Drug Administration (FDA) has granted Fast Track Designation to a treatment for amyotrophic lateral sclerosis (ALS). Tazbentetol, developed by biopharmaceutical company Spinogenix, is a once-a-day pill with potential to restore synapses, the key connections between neurons that allow people to think, plan, remember… Read More
The US Food and Drug Administration has announced two major steps as part of an initiative to advance the implementation of real-time clinical trials (RTCT). First, the agency unveiled the successful initiation of two proof-of-concept clinical trials that will report endpoints and data signals to the agency… Read More
The US Food and Drug Administration (FDA) has approved the first and only triple therapy for patients 12 years and older with asthma. AstraZeneca’s fixed-dose triple-combination therapy Breztri Aerosphere has been approved in the US for the maintenance treatment of asthma in adult and paediatric patients 12 years of age… Read More
The US Food and Drug Administration (FDA) has paused a trial evaluating a drug for treatment-resistant schizophrenia (TRS) after the death of a participant. Evenamide, developed by biopharmaceutical company Newron Pharmaceuticals, was being evaluated in the Phase III ENIGMA-TRS 2 study, targeting the modulation of excessive release of glutamate in… Read More
The world’s first and only malaria drug for newborns has been cleared globally, marking a crucial step towards addressing a long-standing gap in malaria care. Coartem (artemether-lumefantrine) Baby, developed by Medicines for Malaria Venture (MMV) and Novartis, has been prequalified by the World Health Organization (WHO) as the first… Read More
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending marketing authorisation for Novartis’ Itvisma (intrathecal onasemnogene abeparvovec). The opinion supports its use for the treatment of children two years and older, teens, and adults… Read More
The US Food and Drug Administration (FDA) has granted accelerated approval for Regeneron Pharmaceuticals’ Otarmeni (lunsotogene parvec-cwha), the first gene therapy and second new molecular entity approved under the FDA Commissioner’s National Priority Voucher programme. Otarmeni is an adeno-associated virus vector-based gene therapy indicated for the treatment… Read More
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has approved a clinical trial application for a therapy targeting advanced high-grade serous ovarian cancer. CryptiVax-1001, developed by biotechnology company Epitopea, is an off-the-shelf mRNA therapeutic vaccine. It is designed to drive targeted immune responses using a set of tumour-specific antigens… Read More
The UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted Clinical Trial Authorisation to a potential preventative therapy for Cytokine Release Syndrome (CRS). The TOPICAL trial will investigate Poolbeg Pharma’s therapy, POLB 001, in approximately 30 relapsed/refractory multiple myeloma patients receiving the approved bispecific antibody teclistamab. Read More
NHS patients are accessing groundbreaking treatments faster as the UK government fulfils its promise for a 150-day clinical trial target. The latest UK Clinical Research Delivery key performance indicators show the average time to set up a commercial interventional clinical trial has fallen from 169 days to 122 days when comparing the same six month period… Read More
Access to AstraZeneca’s hyperkalaemia treatment Lokelma has been expanded across England and Wales. AstraZeneca announced the National Institute for Health and Care Excellence (NICE) had issued a draft recommendation for lokelma (sodium zirconium cyclosilicate). Under the new willingness-to-pay threshold, more than double the number of patients could… Read More
The US Food and Drug Administration (FDA) has approved Foundayo (orforglipron) for adults with obesity, or overweight with weight-related medical problems. “People living with obesity need treatment options that meet them where they are – and for many, a once-daily pill that can be taken with no… Read More
The European Commission (EC) has granted marketing authorisation for Kygevvi (doxecitine and doxribtimine) for the treatment of thymidine kinase 2 deficiency (TK2d). The approval, making Kygevvi the first and only treatment for TK2d, can now be used to treat paediatric and adult patients with the condition with… Read More
The US Food and Drug Administration (FDA) has approved Lifyorli (relacorilant) in combination with nab-paclitaxel for the treatment of adults with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer. It is approved in patients who have received one to three prior systemic treatment regimens, at least… Read More
