Pharmaceutical Market Europe • June 2024 • 8-9
NEWS
Study reveals two copies of risk gene increase chances of Alzheimer’s
A study conducted by researchers in Spain has revealed that people who carry two copies of an Alzheimer’s risk gene could have a significantly higher chance of developing Alzheimer’s disease (AD).
Researchers based in Barcelona analysed medical records from over 10,000 people from across the US and Europe, as well as over 3,000 brain samples from people who had donated their brains to research.
Apolipoprotein E (APOE) is a protein that transports fatty molecules such as cholesterol to cells in the brain, to help maintain their structure, obtain energy and communicate with one another.
The three most common variants of APOE, APOE2, APOE3 and APOE4, are associated with people’s risk of AD in different ways and APOE4 has the biggest impact, with around one in 50 people carrying two copies.
The analysed brain tissue revealed that almost all APOE4 double-carriers had signs of AD in their brains by the age of 55.
In addition, APOE4 double-carriers who did develop AD symptoms developed them around the age of 65, seven to ten years earlier than those with other APOE variants.
Novartis shares results for ultra-rare kidney disease treatment
Novartis has shared positive results from a late-stage trial of its factor B inhibitor Fabhalta (iptacopan) in patients with the ultra-rare kidney disease complement 3 glomerulopathy (C3G).
C3G occurs when an over-activation of the alternative complement pathway causes deposits of C3 protein to build up in kidney glomeruli.
This triggers inflammation and glomerular damage that results in proteinuria (protein in urine), haematuria (blood in urine) and reduced kidney function.
The phase 3 APPEAR-C3G study has been evaluating the safety and efficacy of a twice-daily 200mg dose of Fabhalta in C3 patients.
Results, presented at this year’s European Renal Association Congress, showed that patients treated with Fabhalta in addition to supportive care achieved a 35.1% reduction in proteinuria at six months, compared to placebo plus supportive care.
Data on the secondary endpoint of estimated glomerular filtration rate, a measure of kidney function, also showed improvements over six months with Fabhalta compared to placebo, and Novartis’ therapy demonstrated a favourable safety profile with no new safety signals observed.
The trial will continue with an additional six-month, open-label period, in which all patients will receive Fabhalta.
Eli Lilly shares results for once-weekly insulin in type 2 diabetes
Eli Lilly has shared positive topline results from two late-stage studies evaluating its investigational once-weekly insulin efsitora (efsitora alfa) in adults with type 2 diabetes (T2D).
The phase 3 QWINT-2 trial randomised insulin-naïve patients to receive efsitora or once-daily insulin degludec for 52 weeks.
The primary endpoint of non-inferior A1C reduction was met, with efsitora reducing A1C by 1.34% compared to 1.26% for degludec. This resulted in an A1C of 6.87% and 6.95% for efsitora and degludec, respectively.
In a key secondary endpoint, efsitora was non-inferior to degludec in A1C change among patients using and not using GLP-1 receptor agonists.
Non-inferior A1C reductions were also seen in the phase 3 QWINT-4 trial, which compared efsitora to once-daily insulin glargine for 26 weeks in adults with T2D who had previously been treated with basal insulin and at least two injections per day of mealtime insulin.
Efsitora and glargine, both administered with insulin lispro, reduced A1C by 1.07%, resulting in an A1C of 7.12% and 7.11%, respectively.
It is hoped that a once-weekly insulin option could improve adherence rates and quality of life for patients.
WHO opens application period for 2025 Model Lists of Essential Medicines
The World Health Organization (WHO) has announced that the application period is now open ahead of its 25th meeting of the WHO Expert Committee on Selection and Use of Essential Medicines from 7 to 11 April 2025.
The deadline for submissions is 1 November 2024 and they will be used to revise and update the WHO Model Lists of Essential Medicines (EML) and Essential Medicines for Children (EMLc).
Every two years, WHO’s EML and EMLc lists are reviewed and updated based on disease prevalence, resource use, public health relevance, evidence of efficacy and safety, and comparative cost-effectiveness.
Currently, more than 130 countries use the WHO Model List to develop their national essential medicines list and to guide purchasing decisions.
The WHO Model Lists are used to provide countries with an evidence-based model to base their national essential medicine lists, guiding governments, health facilities and procurers to achieve universal health coverage.
Essential medicines on the lists can include anaesthetics, preoperative medicines and medical gases, pain and palliative care, antiallergics and medicines used in anaphylaxis, nervous system diseases and more.
EC finalises joint clinical assessment rules for faster access to medicines
The European Commission (EC) has finalised new rules for the joint clinical assessment of medicines to contribute to faster access to medicine for patients living in the EU.
The new approach aims to harness and pool expertise from across the EU to assess the effectiveness of new medicines compared to medicines already being used, while supporting healthcare systems to provide quality care for patients in the EU, utilising the latest technology.
Currently, new health technologies undergo national-level assessments to determine their cost-effectiveness. However, this process can be expensive and time-consuming.
The joint clinical assessment reports will be used to provide scientific evidence to Member States’ authorities at an early stage after a medicine’s marketing authorisation to help them decide whether to use it in the national health system.
The medicines will be assessed by their value, supported by robust evidence, ensuring more effective and timely decisions when bringing medicines to the market.
In addition, the rules set out how the EC will coordinate with the European Medicines Agency on exchanging information and selecting experts and patients to consult with on the assessments.
UK government launches five-year action plan for antimicrobial resistance
The UK government has announced the launch of a five-year national action plan aimed at tackling antimicrobial resistance (AMR).
Responsible for an estimated 1.27 million deaths globally in 2019, AMR occurs when bacteria, viruses, fungi and parasites change over time and find ways to resist the effects of antimicrobial drugs.
Inappropriate and excessive use of antimicrobials can accelerate the process, and the new national action plan will commit the UK to reducing its use of antimicrobials.
The commitments outlined in the plan also include strengthening the surveillance of drug-resistant infections before they emerge, supporting low- and middle-income countries to respond to the threat of AMR and incentivising the development of new vaccines, diagnostics and therapeutics, including alternatives to antimicrobials.
The initiative is the second of a series of five-year national action plans and builds on progress made towards the UK’s 20-year vision for AMR.
The latest plan could include the expansion of the “world-first subscription model” for antimicrobials, which would see more companies being paid a fixed annual fee for antimicrobials based primarily on their value to the NHS, instead of the volumes used.
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