Latest news with #LudovicFenaux
Yahoo
31-01-2025
- Health
- Yahoo
A £1.65m hope for blood disorder patients is now on offer in Manchester
A £1.65 million treatment has been approved for use for some NHS patients, offering hope of a cure to those with an inherited blood disorder. Campaigners have shared their joy as health officials approved a gene-editing therapy for certain children and adults with severe sickle cell disorder. It is estimated that 1,700 people could be eligible after the National Institute for Health and Care Excellence (Nice) approved Casgevy for certain patients with the genetic condition. NHS officials estimate that around 50 people a year will receive treatment. Casgevy, also known as exa-cel, was the first treatment to be licensed using gene-editing tool Crispr, which earned its inventors the Nobel Prize for chemistry in 2020. It works by editing the faulty gene in a patient's own stem cells. READ MORE: Suspect named and set to face court following alleged M6 pursuit READ MORE: Knifeman left friend fighting for his life following unexplained attack Cells are taken from a patient to a laboratory where the Crispr technology is used. The edited cells are then infused back into the patient, which prompts the body to produce healthy red blood cells. The only curative treatment currently available for people with sickle cell in the UK is a donor stem cell transplant. But it is not given to many patients due to the risks involved. Casgevy is an option for patients when a stem cell transplant is suitable but no donor can be found. In March last year, Nice rejected Casgevy for people with severe sickle cell disease. Nice said it required more information on the drug's effectiveness and a commercial agreement, with its £1.65 million list price. Now, a confidential deal has now been struck with its creators, Vertex, on how much the NHS will pay for the treatment under a "managed access scheme". Nice has endorsed the treatment for some people aged 12 and over who suffer from "recurrent vaso-occlusive crises" and have a specific genotype. Dr Samantha Roberts, Nice's chief executive, said: "Exa-cel could represent a potential cure for some people with severe sickle cell disease, freeing people from the burden of complications as well as addressing Nice's aim of reducing health inequalities associated with the condition and getting the best care to patients fast." Ludovic Fenaux, senior vice president at Vertex International, celebrated the announcement, saying: "Today is an important day for the sickle cell community who have gone too long without treatments that address the underlying cause of their devastating disease." In the UK, approximately 17,500 individuals live with sickle cell disease, which is particularly common among those with African or Caribbean heritage. The disease can lead to intense pain, life-threatening infections, and anaemia. Mehmet Tunc Onur Sanli, 42, from London, who has been battling sickle cell disease since he was diagnosed at age 11, explained: "Because of my illness, I often experience pain in my chest, bones, and muscles. "I had surgery on my spleen when I was six and a hip replacement at 22 – I will probably need another hip replacement in the next few months or years. Speaking about the frequency and severity of his sickle cell crises, he said: "I also suffer from regular sickle cell crises – last year, I had to go to the hospital at midnight after waking up in severe pain, and overall, I had to visit the hospital five or six times due to crises." "The pain is the worst I have ever felt in my life – it's hard to put into words." 'Not having to go to hospital for regular transfusions or taking medicine anymore would be a dream to me – gene therapy could offer that – but there's still a lot to consider in terms of the side effects that could come with this treatment and whether it would be the right choice for me.' In clinical trials, all patients who received exa-cel also avoided hospital admission for a year following treatment – and almost 98% had still avoided being admitted to hospital around 3.5 years later. The treatment will be offered at specialist NHS centres in London, Manchester and Birmingham. NHS England chief executive Amanda Pritchard said: 'This is a leap in the right direction for people with sickle cell disease – which can be an extremely debilitating and painful condition. 'This innovative, gene-editing therapy offers hope of a cure for people facing a severe form of the disease and could be absolutely transformative – it could enable patients to live free from the fear of sickle cell crises hanging over them. 'We are funding this new treatment option straight away so patients can benefit from the enhanced quality of life it offers.' John James, chief executive of the Sickle Cell Society, said: 'We are absolutely thrilled to see this groundbreaking gene therapy treatment available on the NHS from today. 'The significance of this milestone for the sickle cell community cannot be understated – today's result will give hope to many and is the result of determined campaigning.' The Medicines and Healthcare products Regulatory Agency (MHRA) approved the treatment in November 2023 for patients aged 12 and over after a 'rigorous assessment of its safety, quality and effectiveness'. It was the first regulator in the world to approve the treatment. Yasmin Sheikh, head of policy and public affairs at Anthony Nolan, said: 'This groundbreaking decision to fund the UK's first ever Crispr-based therapy for patients with sickle cell disorder represents a leap forward in the treatment of this debilitating and life-threatening condition. 'Previously, only individuals fortunate enough to have a stem cell donor match could access stem cell transplants as a potential cure. 'With Casgevy, we now have a treatment that offers hope to more patients, the majority of whom are from African and African-Caribbean backgrounds and have experienced years of feeling ignored. 'This treatment has the potential to transform lives and offers a glimpse into the exciting possibilities of gene therapy.' Nice approved the treatment for certain patients with a severe form of the blood disorder thalassemia in August last year. The NHS spending watchdog said that it will collect more data while patients receive the treatment on the NHS before it evaluates the medicine again. It said that collecting more data through a managed access agreement may resolve some uncertainty in the evidence, particularly about how long the benefits of treatment with exa-cel last. Health minister Andrew Gwynne said: 'By offering a treatment that could allow patients to live a life free of debilitating illness, we will give people with conditions like sickle cell disease more freedom and independence, all whilst protecting vital NHS emergency services.' Professor Bob Klaber, director of strategy, research and innovation at Imperial College Healthcare NHS Trust – which led the UK arm of the clinical trials for exa-cel, said: 'Together with patients and industry partners, we are proud to be part of the ground-breaking research and international academic collaboration that has made this treatment possible. 'The treatment is an example of true medical innovation and will provide patients with no other options a potential cure for the painful, debilitating symptoms of their diseases. 'It also offers promising research avenues for other genetic diseases.'
Yahoo
31-01-2025
- Health
- Yahoo
Vertex Announces CASGEVY® Reimbursement Agreement for the Treatment of Sickle Cell Disease in England
- Agreement means eligible sickle cell disease (SCD) patients in England now have access to CASGEVY - - Agreement for CASGEVY in transfusion-dependent beta thalassemia (TDT) was previously reached in August 2024 - LONDON, January 31, 2025--(BUSINESS WIRE)--Vertex Pharmaceuticals (Nasdaq: VRTX) announced today a reimbursement agreement with NHS England for eligible sickle cell disease (SCD) patients to access the CRISPR/Cas9 gene-edited therapy, CASGEVY® (exagamglogene autotemcel). The reimbursement agreement comes as the National Institute for Health and Care Excellence (NICE) issues positive guidance recommending CASGEVY's use in the NHS. It means that eligible SCD patients in England now have access to the therapy following the prior agreement for transfusion-dependent beta thalassemia (TDT) patients announced last August. "Today is an important day for the sickle cell community who have gone too long without treatments that address the underlying cause of their devastating disease," said Ludovic Fenaux, Senior Vice President, Vertex International. "We are pleased to have reached this new agreement that ensures both eligible SCD and TDT patients can now be treated with CASGEVY, recognizing the value a one-time treatment can provide to patients, their families and the healthcare system." The administration of the therapy requires experience in stem cell transplantation and the management of hemoglobinopathies; therefore, Vertex is continuing to engage with experienced hospitals throughout England to establish a network of independently operated authorized treatment centers (ATCs). About Sickle Cell Disease (SCD) SCD is a debilitating, progressive, life-shortening genetic disease. SCD patients report health-related quality of life scores well below the general population and significant health care resource utilization. SCD affects the red blood cells, which are essential for carrying oxygen to all organs and tissues of the body. SCD causes severe pain, organ damage and shortened life span due to misshapen or "sickled" red blood cells. The clinical hallmark of SCD is vaso-occlusive crises (VOCs), which are caused by blockages of blood vessels by sickled red blood cells and result in severe and debilitating pain that can happen anywhere in the body at any time. SCD requires lifelong treatment and significant use of health care resources, and ultimately results in reduced life expectancy, decreased quality of life and reduced lifetime earnings and productivity. In Europe, the mean age of death for patients living with SCD is around 40 years. Stem cell transplant from a matched donor is a potentially curative option but is only available to a small fraction of people living with SCD because of the lack of available donors. About CASGEVY® (exagamglogene autotemcel) CASGEVY® is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient's own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved for eligible SCD and TDT patients 12 years and older by multiple regulatory bodies around the world. The Conditional Marketing Authorization in Great Britain for CASGEVY is for the treatment of patients 12 years of age and older with either TDT or SCD (with recurrent VOCs who have the βS/βS, βS/β+ or βS/β0 genotype), for whom hematopoietic stem cell transplantation is appropriate and a human leukocyte antigen matched related hematopoietic stem cell donor is not available. For full details about access eligibility please refer to the NICE final draft guidance issued today. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple serious diseases and conditions — cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain — and continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 15 consecutive years on Science magazine's Top Employers list and one of Fortune's 100 Best Companies to Work For. (VRTX-GEN) Vertex Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the statements by Ludovic Fenaux, in this press release, and statements regarding Vertex's expectations for and the anticipated benefits of CASGEVY, expectations for access to CASGEVY for eligible SCD patients in England, and Vertex's plans to continue to engage with experienced hospitals throughout England to establish an ATC network. While we believe the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy, and other reasons, and other risks listed under the heading "Risk Factors" in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at and available through the company's website at You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. View source version on Contacts Vertex Pharmaceuticals Incorporated Investors: InvestorInfo@ Media: mediainfo@ orInternational: +44 20 3204 5275 Sign in to access your portfolio
The Independent
31-01-2025
- Health
- The Independent
£1.65m gene-editing therapy offers hope of cure for some blood disorder patients
A £1.65 million treatment has been approved for use for some NHS patients, offering some with an inherited blood disorder hope of a cure. Campaigners reacted with joy as health officials approved a gene-editing therapy for certain children and adults with severe sickle cell disorder. It is thought that 1,700 people could be eligible after the National Institute for Health and Care Excellence (Nice) approved Casgevy for certain patients with the genetic condition. NHS officials estimate that around 50 people a year will receive treatment. Casgevy, also known as exa-cel, was the first treatment to be licensed using gene-editing tool Crispr, which earned its inventors the Nobel Prize for chemistry in 2020. Exa-cel could represent a potential cure for some people with severe sickle cell disease, freeing people from the burden of complications Dr Samantha Roberts, Nice It works by editing the faulty gene in a patient's own stem cells. Cells are taken from a patient to a laboratory where the Crispr technology is used. The edited cells are then infused back into the patient, which prompts the body to produce healthy red blood cells. The only curative treatment currently available for people with sickle cell in the UK is a donor stem cell transplant. But it is not given to many patients due to the risks involved. Casgevy is an option for patients when a stem cell transplant is suitable but no donor can be found. In March last year, Nice rejected Casgevy for people with severe sickle cell disease. At the time Nice said that it needed further detail about the effectiveness of the treatment, also known as exagamglogene autotemcel (exa-cel) and made by Vertex. It also needed a commercial agreement for the drug, which has a list price of £1.65 million. Now health officials have reached a confidential agreement with Vertex on how much the NHS will pay for the treatment. Nice said that the treatment can be offered under a 'managed access scheme' for treating severe sickle cell disease in some people 12 years and over with 'recurrent vaso-occlusive crises' who have a certain genotype. 'Exa-cel could represent a potential cure for some people with severe sickle cell disease, freeing people from the burden of complications as well as addressing Nice's aim of reducing health inequalities associated with the condition and getting the best care to patients fast,' said Nice chief executive Dr Samantha Roberts. Ludovic Fenaux, senior vice president at Vertex International, added: 'Today is an important day for the sickle cell community who have gone too long without treatments that address the underlying cause of their devastating disease.' There are around 17,500 people with sickle cell disease in the UK. It is particularly common in people with an African or Caribbean family background. Symptoms can include very severe pain, serious and life-threatening infections and anaemia. Mehmet Tunc Onur Sanli, 42, from London, who was diagnosed with sickle cell disease aged 11, said: 'Because of my illness, I often experience pain in my chest, bones, and muscles. 'I had surgery on my spleen when I was six and a hip replacement at 22 – I will probably need another hip replacement in the next few months or years. 'I also suffer from regular sickle cell crises – last year, I had to go to the hospital at midnight after waking up in severe pain, and overall, I had to visit the hospital five or six times due to crises. 'The pain is the worst I have ever felt in my life – it's hard to put into words. 'Not having to go to hospital for regular transfusions or taking medicine anymore would be a dream to me – gene therapy could offer that – but there's still a lot to consider in terms of the side effects that could come with this treatment and whether it would be the right choice for me.' In clinical trials, all patients who received exa-cel also avoided hospital admission for a year following treatment – and almost 98% had still avoided being admitted to hospital around 3.5 years later. The treatment will be offered at specialist NHS centres in London, Manchester and Birmingham. NHS England chief executive Amanda Pritchard said: 'This is a leap in the right direction for people with sickle cell disease – which can be an extremely debilitating and painful condition. 'This innovative, gene-editing therapy offers hope of a cure for people facing a severe form of the disease and could be absolutely transformative – it could enable patients to live free from the fear of sickle cell crises hanging over them. 'We are funding this new treatment option straight away so patients can benefit from the enhanced quality of life it offers.' John James, chief executive of the Sickle Cell Society, said: 'We are absolutely thrilled to see this groundbreaking gene therapy treatment available on the NHS from today. 'The significance of this milestone for the sickle cell community cannot be understated – today's result will give hope to many and is the result of determined campaigning.' The Medicines and Healthcare products Regulatory Agency (MHRA) approved the treatment in November 2023 for patients aged 12 and over after a 'rigorous assessment of its safety, quality and effectiveness'. It was the first regulator in the world to approve the treatment. Yasmin Sheikh, head of policy and public affairs at Anthony Nolan, said: 'This groundbreaking decision to fund the UK's first ever Crispr-based therapy for patients with sickle cell disorder represents a leap forward in the treatment of this debilitating and life-threatening condition. 'Previously, only individuals fortunate enough to have a stem cell donor match could access stem cell transplants as a potential cure. 'With Casgevy, we now have a treatment that offers hope to more patients, the majority of whom are from African and African-Caribbean backgrounds and have experienced years of feeling ignored. 'This treatment has the potential to transform lives and offers a glimpse into the exciting possibilities of gene therapy.' Nice approved the treatment for certain patients with a severe form of the blood disorder thalassemia in August last year. The NHS spending watchdog said that it will collect more data while patients receive the treatment on the NHS before it evaluates the medicine again. It said that collecting more data through a managed access agreement may resolve some uncertainty in the evidence, particularly about how long the benefits of treatment with exa-cel last. Health minister Andrew Gwynne said: 'By offering a treatment that could allow patients to live a life free of debilitating illness, we will give people with conditions like sickle cell disease more freedom and independence, all whilst protecting vital NHS emergency services.' Professor Bob Klaber, director of strategy, research and innovation at Imperial College Healthcare NHS Trust – which led the UK arm of the clinical trials for exa-cel, said: 'Together with patients and industry partners, we are proud to be part of the ground-breaking research and international academic collaboration that has made this treatment possible. 'The treatment is an example of true medical innovation and will provide patients with no other options a potential cure for the painful, debilitating symptoms of their diseases. 'It also offers promising research avenues for other genetic diseases.'
