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Business Wire
a day ago
- Business
- Business Wire
Genethon to Launch Pivotal Trial in Europe of GNT0004 a Low-Dose Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy
PARIS--(BUSINESS WIRE)--Genethon, a worldwide pioneer and leader in research and development in gene therapy for rare genetic diseases, has received approvals from regulatory authorities, MHRA and EMA*, to begin pivotal Phase 3 clinical trials in France and the UK of its gene therapy, GNT0004, for Duchenne muscular dystrophy (DMD). Genethon CEO Frederic Revah observed, 'We are delighted to be able to continue these trials and are determined to bring GNT0004 to market for young patients and their families who are waiting for a therapeutic solution. This marks a decisive step forward for our gene therapy program for DMD, which began in 2021 and has demonstrated extremely promising results in the first children treated in the Phase1/2 portion of our Phase1/2/3 study.' Dr. Revah added, 'In addition to the very positive results in patients treated in the early phases, one of the strengths of our product is the dose selected for the pivotal phase, which is lower than those used in other gene therapy trials for DMD. Approvals of our Phase 3 trials reflect the regulatory authorities' confidence in GNT0004 as well as the work accomplished by our teams.' The Phase 3 authorizations in Europe are based on the results of Phase 1/2 studies showing good tolerance of GNT0004 as well as efficacy in terms of microdystrophin expression, creatine phosphokinase (CPK) reduction, and motor function. Patients showed prolonged improvement or stabilization of motor functions and significant persistent reduction in CPK, a key marker of muscle damage. The Phase 3 double blind trials will begin in August and September in the UK and France using a single intravenous injection of GNT0004, which contains an optimized hMD1 transgene, a shortened (3x10¹³ vg/kg microdystrophin) but functional version of the gene encoding dystrophin in an AAV8 vector associated with transient immunological prophylactic treatment. The vector is designed to express itself in muscle tissue and the heart thanks to a Spc5-12 promoter sequence specific to these tissues. A total of 64 boys aged 6 to 10 with DMD who have retained their walking ability will be enrolled. * French ANSM as reporting member state for the EMA About Duchenne muscular dystrophy Duchenne muscular dystrophy is a rare progressive genetic disease that affects all the muscles in the body and mainly boys (1 in 5,000). It is caused by abnormalities in the gene responsible for the production of dystrophin, a structural protein essential for the stability of muscle fiber membranes and their metabolism. The absence of dystrophin leads to progressive degeneration of the skeletal and cardiac muscles, loss of walking and respiratory capacity, progressive heart failure, and death between the ages of 20 and 40. About Genethon A pioneer in the discovery and development of gene therapies for rare diseases, Genethon is a non-profit laboratory created by the AFM-Telethon. A first gene therapy drug, to which Genethon contributed, has been approved for marketing for spinal muscular atrophy. With more than 240 scientists and experts, Genethon's goal is to develop innovative therapies that change the lives of patients suffering from rare genetic diseases. Thirteen gene therapy products developed by Genethon or to which Genethon has contributed are currently undergoing clinical trials for diseases of the liver, blood, immune system, muscles, and eyes. Seven other products are in preparation for clinical trials over the next five years.
Yahoo
2 days ago
- Business
- Yahoo
Filgotinib Shows Positive Topline Results Across Full Spectrum of Axial Spondyloarthritis in OLINGUITO Phase 3 Study
Alfasigma plans to submit data from the OLINGUITO Phase 3 clinical trial to European Medicines Agency (EMA) and UK's Medicines Healthcare products Regulatory Agency (MHRA) to seek market authorization for filgotinib in the treatment of adults with active axial spondyloarthritis (axSpA). Filgotinib, an oral, once-daily JAK1 preferential inhibitor, met the primary endpoint in the OLINGUITO Phase 3 clinical trial, demonstrating efficacy across the full spectrum of axSpA (r-axSpA and nr-axSpA). The safety profile was consistent with previous filgotinib studies, with no unexpected events observed. AxSpA is a chronic inflammatory disease primarily affecting young adults, typically emerging during the third decade of life, with only 40-50% of patients with axSpA achieving adequate response with current treatments. If approved, filgotinib would offer a new oral treatment option for patients with axSpA expanding the treatment landscape in a therapeutic area marked by significant unmet needs. BOLOGNA, Italy, July 28, 2025--(BUSINESS WIRE)--Alfasigma S.p.A today announced positive topline results from the OLINGUITO Phase 3 clinical trial (NCT05785611; Eudra CT 2022-501354-10-01),i evaluating filgotinib (marketed as Jyseleca® in approved indications) to treat adult patients with active axial spondyloarthritis (axSpA). Filgotinib, an oral, once-daily JAK1 preferential inhibitor, met the primary endpoint in the OLINGUITO Phase 3 clinical trial in active axSpA. Efficacy was demonstrated across the full spectrum of axSpA, including both radiographic (r-axSpA) and non-radiographic (nr-axSpA) forms. The safety profile was consistent with previous studies, with no unexpected events Filgotinib is currently approved for the treatment of moderate to severe active rheumatoid arthritis (RA) and ulcerative colitis (UC).iii "These positive OLINGUITO topline results demonstrate filgotinib's potential to address this critical unmet need for patients with axial spondyloarthritis, with only half responding adequately to current therapies," said Daniele D'Ambrosio, Chief Development Officer at Alfasigma. "Based on these encouraging results, we intend to submit for an extension of filgotinib's current indications, offering a potential new treatment option for patients with axial spondyloarthritis who often struggle with debilitating symptoms from a young age. We thank all patients, investigators and staff at study sites whose participation made this important achievement possible." "These results from the OLINGUITO Phase 3 clinical trial clearly support the potential of filgotinib as a treatment option for patients living with axSpA at all stages of the disease. The burden of disease for these patients remains high as treatment options are limited. It is therefore encouraging that the primary endpoint for both axSpA indications was met in dedicated studies," said Professor Xenofon Baraliakos, Head of Rheumatology at the Rheumazentrum Ruhrgebiet, Herne, Germany, Professor for Internal Medicine and Rheumatology at the Ruhr-University Bochum, Germany. AxSpA is a chronic inflammatory disease primarily affecting young adults, typically emerging during the third decade of life. It primarily affects the axial skeleton (spine and sacroiliac joints) causing significant pain, stiffness and reduced Despite several efficacious anti-inflammatory treatment options, only about 40% to 50% of patients with axSpA achieve a relevant treatment response, and an even smaller proportion (approximately 10%-20%) reach remission or an inactive disease activity state within 16 to 24 weeks of treatment initiation.v About OLINGUITO The OLINGUITO Phase 3 clinical trial (NCT05785611; Eudra CT 2022-501354-10-01) was designed – in compliance with the European Medicines Agency's (EMA) guidelines – to evaluate the efficacy and safety of filgotinib in patients with active axial spondyloarthritis (axSpA). OLINGUITO was preceded by the TORTUGA Phase 2 clinical trial (NCT03117270), a randomized, double-blind, placebo-controlled clinical trial that demonstrated the safety and efficacy of filgotinib in adult patients with moderately to severely active ankylosing spondylitis (also known as r-axSpA, i.e., the damage caused by the disease can be seen on X-rays).vi The first patient entered the OLINGUITO Phase 3 clinical trial in April 2023. The trial consisted of two randomized, double-blind, multi-center, parallel-group studies of patients with active axSpA who had an inadequate response to conventional or biological treatments. Study A included 258 patients with r-axSpA, while study B included 237 patients with non-radiographic axSpA (nr-axSpA, i.e., the patient had symptoms of axSpA but damage was not yet visible on X-rays). After enrollment, patients in each study were randomized (1:1) to receive treatment with oral filgotinib 200 mg, or matching placebo, once daily for 16 weeks. The primary endpoint for both studies was the proportion of patients who achieved an Assessment of SpondyloArthritis international Society 40% improvement (ASAS40) at Week Thereafter, patients without risk factors entered an open-label treatment period in which they received filgotinib 200 mg once daily up to Week 52. Patients from study A and study B who achieved sustained low disease activity or inactive disease during the open-label period were re-randomized (1:1) at Week 52 to receive double-blind filgotinib 100 mg or 200 mg up to Week 104. In patients above 65 years of age and those with increased risk factors for cardiovascular disease or malignancies, at Week 16 in the case of achievement of disease control after treatment with 200 mg once daily, the dose was reduced to 100 mg once daily for up to Week 104. All patients receiving filgotinib 100 mg daily had the option to increase to 200 mg daily in case of flares. Week 52 visits for all patients were completed in May 2025. After Week 104, select eligible patients who achieve ASAS40 response without high disease activity, per investigators' judgment, will enter the Open Label Extension (OLE) study. This OLE is designed to collect additional long-term safety and efficacy data for a duration of approximately 2.5 years. About Filgotinib Filgotinib (marketed as Jyseleca®) is currently approved by the relevant regulatory authorities in the European Union, United Kingdom, Japan, Taiwan, South Korea and Singapore. In Europe, United Kingdom, Japan, Taiwan, South Korea and Singapore, filgotinib is approved for the treatment of moderate to severe active rheumatoid arthritis in adults who have not responded adequately or cannot tolerate other disease modifying anti-rheumatic drugs (DMARDs). Filgotinib is also approved in Europe, United Kingdom, Japan Taiwan, South Korea and Singapore for the treatment of adult patients with moderate to severe active ulcerative colitis who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a biologic agent. Filgotinib 100mg and 200mg are registered in the above-mentioned territories. The European Summary of Product Characteristics for filgotinib, which includes contraindications and special warnings and precautions, is available at The United Kingdom Summary of Product Characteristics for filgotinib can be found at The interview form from the Japanese Ministry of Health, Labor and Welfare is available at The Taiwan Food and Drug Administration Assessment Report for filgotinib can be found at The Korean Ministry of Food and Drug Safety report on filgotinib can be found here The Singapore Summary of Product Characteristics for filgotinib can be found at About axial spondyloarthritis Axial spondyloarthritis (axSpA) is a chronic inflammatory condition that primarily affects the axial skeleton (spine and sacroiliac joints). While persistent back pain and spinal stiffness are common initial symptoms, the disease often also presents with peripheral manifestations such as enthesitis, arthritis, and dactylitis, as well as extra-musculoskeletal features including uveitis, inflammatory bowel disease and psoriasis. AxSpA comprises the whole spectrum of patients with and without radiographic sacroiliitis, that is, radiographic axSpA (r-axSpA; also known as ankylosing spondylitis, i.e., the damage caused by the disease can be seen on X-rays) and non-radiographic axSpA (nr-axSpA), respectively. AxSpA usually starts during the third decade of life; r-axSpA is more common in men than women, whereas there is an equal sex distribution among patients with About Alfasigma Alfasigma is a global pharmaceutical company founded over 75 years ago in Italy, where it is headquartered (in Bologna and Milan). The Group operates in over 100 markets spanning Europe, North and South America, Asia, and Africa. It has offices in several countries, including Italy, the US, Spain, Germany, Mexico, and China; production sites in Italy (Pomezia, RM; Alanno, PE; Sermoneta, LT; Trezzano Rosa, MI), Spain (Tortosa, Baix Ebre), and the United States (Shreveport, Louisiana); and R&D labs in Italy (Pomezia and Bergamo). Alfasigma employs approximately 4,000 people dedicated to research, development, production, and distribution of medicinal products, contributing to its mission, to provide better health and a better quality of life for patients, caregivers, and healthcare providers'. It focuses on three main therapeutic areas: Gastroenterology, Vascular and Rheumatology. Its portfolio spans from primary care to specialty care, rare disease medications, and consumer health products, including medical foods and nutraceuticals. For more information, please visit Forward-Looking Statements This release may contain forward-looking statements based on current assumptions and forecasts made by Alfasigma. Various known and unknown risks, uncertainties, and other factors could lead to material differences between the actual future results, financial situation, development, or performance of the company and the estimates given here. Alfasigma assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments. References i OLINGUITO Clinical Trial (NCT05785611) Study Details | A Study Evaluating the Effect of Filgotinib in Participants With Active Axial Spondyloarthritis | Accessed July 2025. ii The topline results of OLINGUITO Phase 3 clinical trial will become available in future scientific conferences and published in a peer-reviewed journal in due course. iii Accessed July 2025. iv Navarro-Compán V, et al., Axial spondyloarthritis. Lancet 2025; 405: 159–72 v Poddubnyy D, et al. Ann Rheum Dis 2025; 84(4): 538-546. doi: 10.1016/ vi Van der Heijde D, et al. Lancet 2018; 392(10162): 2378-2387. doi: 10.1016/S0140-6736(18)32463-2 vii J. Sieper, et al. Ann Rheum Dis. 2009 Jun:68 Suppl 2:ii1-44. doi: 10.1136/ard.2008.104018. Based on the ASAS (Assessment of SpondyloArthritis International Society) assessment that includes four aspects (domains): patient global assessment, pain, function and inflammation. In order to meet ASAS40 response, 3 of the 4 domains should improve by at least 40% with a minimum 2-unit change on a scale of 0 to 10. For the remaining domain, there should be no deterioration (worsening) from baseline. View source version on Contacts Contact Press Office:Gea Annie Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Evening Standard
2 days ago
- Health
- Evening Standard
Pharmacies warn of unsustainable demand for weight loss medication
Mounjaro and Wegovy are licensed by the Medicines and Healthcare products Regulatory Agency (MHRA) for use in patients with a BMI of over 30 or between 27 and 30 but with a weight-related co-morbidity. This occurs when an individual who has obesity develops another medical condition due to their weight.
Daily Mail
3 days ago
- Health
- Daily Mail
Health officials were warned of rogue Botox beauticians a YEAR ago - but failed to act
Drug safety chiefs were warned last year about rogue beauty therapists injecting illegal Botox-style products but failed to act before the jabs hospitalised dozens. The procedures, carried out by beauticians using unlicensed versions of botulinum toxin, left 38 people across the North East, North West, East of England and Midlands seriously ill with paralysis-like symptoms and breathing difficulties. The alarming outbreak this month has exposed 'gaping holes' in regulation, claim senior doctors now calling on the Government to ban medically unqualified practitioners from administering cosmetic injections. Dr Steven Land, a Newcastle-based expert in aesthetic medicine, said he first raised concerns in November 2023 with the Medicines and Healthcare products Regulatory Agency (MHRA). 'Our only surprise is that this didn't happen sooner,' he said. 'I was getting WhatsApp messages and emails every week from people selling knock-off toxin – illegal, unlicensed products that you can buy online with no checks.' Dr Land said he provided the MHRA with names, phone numbers, pricing information and screenshots of practitioners openly advertising cut-price anti-wrinkle jabs using unlicensed botulinum toxin. He added: 'When I followed up in April, they told me the matter would be dealt with 'in due course'. Nothing happened. If action had been taken when I raised the alarm, dozens might not have ended up in hospital.' MHRA was asked to comment. Due to the severity of the situation, the UK Health Security Agency (UKHSA) announced this month it had launched an inquiry. The practitioners involved are said to be cooperating, although no further detail has been given. Dr Land said two beauty therapists operating in his area had been identified as the primary source of the outbreak. Both had used a Korean-manufactured product called Toxpia. 'Neither had any medical qualifications – just basic beauty therapy training – and both were injecting clients in salons and private homes using the same unlicensed product,' he said. 'We believe they were buying this toxin online and administering it with no clinical oversight.' HJ Corporations Co. Ltd, the maker of Toxpia, was approached for comment. More than seven million people in the UK undergo cosmetic treatments – including Botox and fillers – each year and demand is growing. The industry is now worth an estimated £3.6billion. Yet in Britain, anyone can legally offer cosmetic injections regardless of medical training – a situation many doctors have long condemned. A recent study by University College London found that two in three procedures are carried out by non-medics, including hairdressers and beauty therapists. Dr Christopher Rowland Payne, president of the Royal Society of Medicine Section of Aesthetic Medicine and Surgery, said: 'The current situation is quite terrifying. It is irresponsible for the Government to allow this to continue. 'Britain is the odd man out in Europe in allowing non-medics to perform these procedures.' Dr Rowland Payne, who gave evidence to the women and equalities committee on the issue, also highlighted the stark differences in training between medics and non-medics offering Botox. Botulinum toxin – the active ingredient in Botox – is one of the most powerful poisons known, being found in soil and contaminated food. When consumed, it can cause botulism – a potentially fatal illness that leads to muscle paralysis and respiratory failure. Its potency is staggering: gram-for-gram, botulinum toxin is estimated to be 100,000 times more toxic than cyanide. Experts stress that the risk is not with approved brands – such as Botox, Dysport, Xeomin and Jeuveau – but with counterfeit or unlicensed versions used by untrained individuals. Toxpia is not licensed for use in the UK though industry insiders say it is available to buy online. In 2023, the Government launched a consultation on new rules for the aesthetics sector and Health Secretary Wes Streeting suggested this month a crackdown was imminent. Kaylie Bailey, 36, from Peterlee, County Durham, was left fighting for her life and now wears an eye patch after developing paralysis and losing the ability to breathe. She had paid £75 for cut-price anti-wrinkle jabs from beautician Gemma Gray – who has since admitted the product caused widespread harm but claimed it was a 'nationwide problem'. 'I remember lying on the bed thinking 'I'm dying',' added Ms Bailey, who spent three days in intensive care with botulism. We're having to live with what she [the beautician] did to us. I nearly died because of it.'
The Sun
5 days ago
- Health
- The Sun
Urgent recall for asthma medication taken by thousands as health chiefs warn of wrong dose instruction
AN urgent warning has been issued for a common inhaler used by asthma sufferers due to a mistake on the labelling. The error affecting Flutiform 250 micrograms inhalers means asthma patients could be informed of the wrong dose of the drug. 1 In its alert, UK medicines regulator, the MHRA said: "CD Pharma Ltd [the inhaler's manufacturer] have notified the MHRA of an error on the outer carton of the product for the batches listed in this notification. "While the total active content statement is correct, the delivered dose content statement is incorrect." The other product details on the inhaler carton, however, including the name, strength and pharmaceutical form of the medicine, are correct. And the quality of the medicine has not been impacted by the labelling defect. Patients have been advised to continue to take the medication as prescribed by their healthcare professionals. Meanwhile, healthcare professionals have been advised to stop supplying the above batch immediately. The recall urged: "Quarantine all stock and return it to your supplier using your supplier's approved process." "This is a wholesale and pharmacy level recall that will be actioned by a healthcare professional," added the MHRA. "There is no quality issue with the product and patients can continue to take their medicine as prescribed." The labelling mistake means patients may have thought they were only receiving half the necessary dose of the medication. Early Warning Signs of an Asthma Attack As part of the recall, patients who do experience adverse reactions or have any questions about the medication are advised to seek medical attention. Any suspected adverse reactions should also be reported via the MHRA Yellow Card Scheme. Asthma is a common condition. In the UK, 7.2 million people have asthma. This is about eight in every 100 people, according to Asthma + Lung UK. It affects the airways that carry air in and out of your lungs, causing them to become inflamed. This makes the airways narrower so less air gets into and out of the lungs. Symptoms, like wheezing, breathlessness, a cough or a tight chest, can be triggered by things like exercise, allergens or changes in weather. At the moment there is no cure for asthma, but most people with asthma can control their symptoms well with asthma inhalers. Depending on how severe your symptoms are, the NHS says you may be offered either: an inhaler to use only when you get symptoms – this is called an anti-inflammatory reliever (AIR) inhaler an inhaler to use every day to help prevent symptoms, as well as when you get symptoms – this is called a maintenance and reliever therapy (MART) inhaler two separate inhalers – a preventer inhaler to use every day to help prevent symptoms, and a blue reliever inhaler to use when you get symptoms (you should not be given a blue reliever inhaler to use on its own) The NHS also advises: "It's important to use your inhaler correctly so that your medicine works properly. "There are different types, including dry powder inhalers, pMDIs and soft mist inhalers.
