{"id":195897,"date":"2025-06-19T01:45:14","date_gmt":"2025-06-19T01:45:14","guid":{"rendered":"https:\/\/www.europesays.com\/uk\/195897\/"},"modified":"2025-06-19T01:45:14","modified_gmt":"2025-06-19T01:45:14","slug":"ive-dreamed-of-this-moment-first-ever-patient-gets-worlds-most-expensive-drug-on-the-nhs-it-costs-2-6million-per-dose","status":"publish","type":"post","link":"https:\/\/www.europesays.com\/uk\/195897\/","title":{"rendered":"‘I’ve dreamed of this moment’: First ever patient gets ‘world’s most expensive drug’ on the NHS – it costs \u00a32.6million per dose"},"content":{"rendered":"

A 44-year-old man has become the first ever patient to receive a life-changing dose of the ‘world’s most expensive drug’ on the NHS<\/a>.<\/p>\n

Called Hemgenix, the drug\u2014which costs an estimated\u00a0\u00a32.6million per patient\u2014was administered yesterday at St Thomas’ NHS Foundation Trust in London<\/a>.\u00a0<\/p>\n

Delivered as a one-off IV drip, it is the only treatment of its kind for haemophilia B.<\/p>\n

This is a bleeding disorder where the body doesn’t make enough\u2014or any\u2014of a vital protein critical to clotting.\u00a0<\/p>\n

Clotting stops wounds from bleeding, so those with the disorder risk suffering severe and even life-threatening blood loss from even minor injuries.<\/p>\n

Patients also run the risk of what are called ‘spontaneous bleeds’, which can be triggered without a direct injury and even prove deadly if they occur in a vital organ.<\/p>\n

Prior to Hemgenix, all haemophilia B patients needed regular weekly injections of an artificial clotting agent to keep their risk of catastrophic injury to a minimum.<\/p>\n

The patient, speaking anonymously, was diagnosed with haemophilia B when he was just 18 months old, and said he was now looking forward to a life ‘free of worry’.<\/p>\n

\"A <\/p>\n

A 44-year-old man has become the first ever patient to receive a life-changing dose of the ‘world’s most expensive drug’ on the NHS. Credit: Supplied<\/p>\n

‘I’ve always had to be more cautious and to plan ahead. There is a level of anxiety in that and being overly cautious has often led to missed opportunities and things I can\u2019t do, like contact sports,’ he told MailOnline.\u00a0<\/p>\n

‘To experience a life free of the worry and to do things that I wouldn’t normally do will also be amazing.’<\/p>\n

He added: ‘Not needing to plan ahead for treatment deliveries or looking up hospitals in foreign destinations when going on holiday, or having to tell people “sorry I can’t do that I’ve got haemophilia”, will be something I’ve always dreamed of.’\u00a0<\/p>\n

Medics and charities have hailed the roll-out of the therapy as a critical milestone for helping those with the condition lead more fulfilling lives.\u00a0<\/p>\n

Dr Pu-Lin Luo, the consultant haematologist at Guy\u2019s and St Thomas\u2019 who administered the treatment yesterday said it represented an ‘exciting’ step in treating the condition.\u00a0<\/p>\n

‘This is a big step forward in our ability to manage haemophilia B and could change the lives of some of our patients.\u00a0<\/p>\n

‘It is also a testament to the advancement of cell and gene therapies in the UK,’ she said.\u00a0<\/p>\n

Kate Burt, chief executive of charity The Haemophilia Society, also added:\u00a0‘Today marks an important milestone not just for this patient, but for all those living with haemophilia B in the UK.’<\/p>\n

\"Called <\/p>\n

Called Hemgenix the drug costs \u00a32.6million per patient, but experts insist it could save the NHS money in the long term\u00a0<\/p>\n

‘The current treatment of lifelong intravenous injections can place a significant burden on those living with haemophilia and it has an impact on broader family, relationships and work.\u00a0<\/p>\n

‘The availability of gene therapy for haemophilia B as a one-time infusion will allow those eligible for treatment to expand their horizons and live life to the full, free from the restrictions of regular injections.’<\/p>\n

Despite being approved for use on the NHS last year<\/a>, yesterday’s treatment is the first time Hemgenix has been used by the health service.<\/p>\n

It’s also the first time the drug has been used in the UK outside of a clinical trial.\u00a0<\/p>\n

Hemgenix is the brand name of the drug\u00a0etranacogene dezaparvovec.<\/p>\n

It works by replacing a patient’s defective gene\u2014which is incapable of producing the clotting protein\u2014with one that can, eliminating the need for regular injections.<\/p>\n

Studies on the gene therapy have shown the protective effect lasts for at least three years, but the hope is it could work for even longer.<\/p>\n

There are approximately 2,000 people with haemophilia B in the UK.\u00a0<\/p>\n

\"Hemgenix <\/p>\n

Hemgenix works by replacing the patient’s defective gene, which is incapable of producing the clotting protein, with one that can, eliminating the need for frequent injections<\/p>\n

Yet only around 260 with ‘moderately severe or severe haemophilia B’ are currently eligible for Hemgenix on the NHS.<\/p>\n

This puts the potential total bill to the taxpayer at about \u00a3676million.\u00a0<\/p>\n

MailOnline understands, however, that the NHS acquired the drug at an undisclosed discount, so the actual cost is likely to be less.\u00a0<\/p>\n

Additionally, while the cost of the drug is roughly \u00a32.6million per patient, medics claim the treatment actually saves the NHS money in the long term.<\/p>\n

The lifetime cost of providing a patient the alternative weekly clotting injections has been estimated to be \u00a38million.\u00a0<\/p>\n

This sum doesn’t include the cost of life-saving interventions and surgeries haemophilia B may also need.\u00a0<\/p>\n

Hemgenix is made by Philadelphia-based pharmaceutical company CSL Behring.<\/p>\n

Reacting to the news today the firm’s general manager for UK and Ireland, Eduardo Cabas, said: ‘This patient receiving etranacogene dezaparvovec is a testament to the collaborative efforts of the haemophilia community, NICE and NHS England to ensure that patients in the UK are able to access this one-time treatment option.’<\/p>\n

Hemgenix was initially described as the ‘most expensive drug in the world’ when it originally came onto the scene in 2022.<\/p>\n

However, other drugs\u2014also gene therapies\u2014have since eclipsed it in terms of overall cost.\u00a0\u00a0<\/p>\n

Like all medications Hemgenix does come with the risk of side effects.\u00a0<\/p>\n

Patients administered the drug take regular liver function tests to ensure the vital organ is functioning correctly.<\/p>\n

This is because there is a risk the medication could trigger a potentially dangerous immune response.<\/p>\n

These tests are held one a week for the first three months before they are reduced to once a year.<\/p>\n

Patient information leaflets distributed with the drug also highlight a potential increased risk of cancer due to the fact it inserts itself into the DNA of cells.<\/p>\n

However, clinical studies have\u2014so far\u2014shown no cancers have been caused by Hemgenix use.<\/p>\n

Those taking Hemgenix must also use contraceptives like condoms for at least a year following treatment, due to a process called ‘shedding’\u2014where the drug can be passed through bodily fluids like semen.<\/p>\n

Patients are also forbidden from donating blood for this reason.<\/p>\n","protected":false},"excerpt":{"rendered":"A 44-year-old man has become the first ever patient to receive a life-changing dose of the ‘world’s most…\n","protected":false},"author":2,"featured_media":195898,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[4315],"tags":[92,105,4326,16,15],"class_list":{"0":"post-195897","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-medication","8":"tag-dailymail","9":"tag-health","10":"tag-medication","11":"tag-uk","12":"tag-united-kingdom"},"share_on_mastodon":{"url":"https:\/\/pubeurope.com\/@uk\/114707505210640587","error":""},"_links":{"self":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/195897","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/comments?post=195897"}],"version-history":[{"count":0,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/195897\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media\/195898"}],"wp:attachment":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media?parent=195897"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/categories?post=195897"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/tags?post=195897"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}