Latest news with #Hemgenix
Daily Mail
11 hours ago
- Health
- Daily Mail
EXCLUSIVE 'I've dreamed of this moment': First ever patient gets 'world's most expensive drug' on the NHS - it costs £2.6million per dose
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. Called Hemgenix, the drug—which costs an estimated £2.6million per patient—was administered yesterday at St Thomas' NHS Foundation Trust in London. Delivered as a one-off IV drip, it is the only treatment of its kind for haemophilia B. This is a bleeding disorder where the body doesn't make enough—or any—of a vital protein critical to clotting. Clotting stops wounds from bleeding, so those with the disorder risk suffering severe and even life-threatening blood loss from even minor injuries. 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. 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. 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'. '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't do, like contact sports,' he told MailOnline. 'To experience a life free of the worry and to do things that I wouldn't normally do will also be amazing.' 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.' 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. Dr Pu-Lin Luo, the consultant haematologist at Guy's and St Thomas' who administered the treatment yesterday said it represented an 'exciting' step in treating the condition. 'This is a big step forward in our ability to manage haemophilia B and could change the lives of some of our patients. 'It is also a testament to the advancement of cell and gene therapies in the UK,' she said. Kate Burt, chief executive of charity The Haemophilia Society, also added: 'Today marks an important milestone not just for this patient, but for all those living with haemophilia B in the UK.' '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. '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.' Despite being approved for use on the NHS last year, yesterday's treatment is the first time Hemgenix has been used by the health service. It's also the first time the drug has been used in the UK outside of a clinical trial. Hemgenix is the brand name of the drug etranacogene dezaparvovec. It works by replacing a patient's defective gene—which is incapable of producing the clotting protein—with one that can, eliminating the need for regular injections. 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. There are approximately 2,000 people with haemophilia B in the UK. Yet only around 260 with 'moderately severe or severe haemophilia B' are currently eligible for Hemgenix on the NHS. This puts the potential total bill to the taxpayer at about £676million. MailOnline understands, however, that the NHS acquired the drug at an undisclosed discount, so the actual cost is likely to be less. Additionally, while the cost of the drug is roughly £2.6million per patient, medics claim the treatment actually saves the NHS money in the long term. The lifetime cost of providing a patient the alternative weekly clotting injections has been estimated to be £8million. This sum doesn't include the cost of life-saving interventions and surgeries haemophilia B may also need. Hemgenix is made by Philadelphia-based pharmaceutical company CSL Behring. 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.' Hemgenix was initially described as the 'most expensive drug in the world' when it originally came onto the scene in 2022. However, other drugs—also gene therapies—have since eclipsed it in terms of overall cost. Like all medications Hemgenix does come with the risk of side effects. Patients administered the drug take regular liver function tests to ensure the vital organ is functioning correctly. This is because there is a risk the medication could trigger a potentially dangerous immune response. These tests are held one a week for the first three months before they are reduced to once a year. 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. However, clinical studies have—so far—shown no cancers have been caused by Hemgenix use. Those taking Hemgenix must also use contraceptives like condoms for at least a year following treatment, due to a process called 'shedding'—where the drug can be passed through bodily fluids like semen. Patients are also forbidden from donating blood for this reason.
Yahoo
13-05-2025
- Health
- Yahoo
Top 10 most expensive prescription drugs in the US by price and by sales volume
On the heels of President Donald Trump's Monday announcement of an executive order that will slash prescription drug prices in the U.S., the spotlight is on current costs and how much Americans could save. The president's order calls for "most favored nations drug pricing" — which means "the lowest price paid for a drug in other developed countries, that is the price that Americans will pay," he said. "Some prescription drug and pharmaceutical prices will be reduced almost immediately by 50 to 80 to 90%," Trump said. President Trump Takes On 'Big Pharma' By Signing Executive Order To Lower Drug Prices Katy Dubinsky, a New York pharmacist and founder and CEO of Vitalize, applauded the move to reduce prescription prices, noting that Trump's order tackles a long-standing problem. "But this will not be simple to accomplish," she told Fox News Digital. Read On The Fox News App "The executive order doesn't reduce costs immediately," she said. "It directs government agencies to start drafting the rules, which may take months." Here are the five most expensive prescription drugs in the U.S. by price — followed by five by volume. Dubinsky detailed some of the most expensive prescription drugs in the country today and what conditions they treat. 1. Lenmeldy (atidarsagene autotemcel) by Orchard Therapeutics – $4.25 million This medication is used to treat metachromatic leukodystrophy (MLD), a rare genetic disorder that damages the nervous system, Dubinsky said. "It is given once and is supposed to stop or slow down the disease in young kids," she noted. Top 10 'Allergy Capitals' Of The Us, Plus 4 Tips To Manage Symptoms 2. Hemgenix (etranacogene dezaparvovec-drlb) by CSL Behring – $3.5 million This medication is prescribed for people with hemophilia B, a bleeding disorder. "This one-time treatment helps the body make its own clotting factor, so patients don't need regular infusions," said Dubinsky. 3. Elevidys (delandistrogene moxeparvovec-rokl) by Sarepta Therapeutics – $3.2 million This prescription medication, intended for young boys, treats Duchenne muscular dystrophy (DMD), a condition that weakens muscles over time. "It aims to slow down how fast the disease progresses," Dubinsky said. 4. Skysona (elivaldogene autotemcel) by Bluebird Bio – $3 million "This medication is used for cerebral adrenoleukodystrophy (CALD), a serious brain disease in boys," said Dubinsky. "This therapy tries to slow the damage before symptoms get worse." 5. Zynteglo (betibeglogene autotemcel) by Bluebird Bio – $2.8 million Zynteglo is for beta-thalassemia, a blood condition that usually requires regular transfusions. "This gene therapy can help patients make healthy red blood cells on their own and reduce how often they need treatment," said Dubinsky. John Stanford, executive director of Incubate, a Washington-based coalition of early-stage life-science investors, shared his thoughts on the top five most expensive drugs by sales volume. "Typically, when the government is focused on the most expensive drugs, they're focused on the metric based on sales volume rather than, for instance, a rare disease therapy with a high list price but smaller patient pool," he told Fox News Digital. "Often, officials are focused on total drug spending by Medicare or other government programs." 1. Keytruda (pembrolizumab) by Merck — $25 billion revenue (2023) Keytruda is an immunotherapy medication used to treat a variety of cancers, including melanoma, non-small cell lung cancer, liver cancer and others. "Keytruda has become Merck's crown jewel, helping the company expand its cancer treatment portfolio with more than 1,000 active clinical trials," Stanford told Fox News Digital. Terminal Colon Cancer Patient Saved By Breakthrough Treatment 2. Eliquis (apixaban) by Bristol Myers Squibb and Pfizer — $18.95 billion Eliquis (apixaban) is an "anchor drug" for both BMS and Pfizer, according to Stanford. Apixaban is prescribed to prevent the formation of blood clots and to treat deep vein thrombosis and pulmonary embolism (a blood clot in the lungs). 3. Ozempic (semaglutide) by Novo Nordisk — $13.93 billion Prescribed for type 2 diabetes, the semaglutide medication Ozempic has become widely popular for its weight-loss effects and other health benefits. "Ozempic's sales are powering Novo Nordisk's broader foray into GLP-1s for obesity, heart disease and liver conditions — all areas with high development costs and uncertain scientific outcomes," Stanford told Fox News Digital. "The money has gone toward scaling up production to meet demand for GLP-1s and avoid supply shortages." 4. Humira (AbbVie) — $14.4 billion (U.S. 2023 revenue) "Humira has been one of the highest-grossing drugs in history, generating over $200 billion during its exclusivity period," Stanford said. The injectable medication, which contains the active ingredient adalimumab, is used to treat rheumatoid arthritis and other inflammatory conditions. Click Here To Sign Up For Our Health Newsletter 5. Biktarvy by Gilead — $11.85 billion Biktarvy is an HIV treatment that includes the three ingredients bictegravir, emtricitabine and tenofovir alafenamide. "Biktarvy isn't just a leading HIV treatment — it's the financial backbone for Gilead's move into cancer research," Stanford said. Dr. Jacob Glanville, CEO of Centivax, a San Francisco biotechnology company, said vaccines and most generic drugs would not likely be changed by the executive order. "Most vaccines that Americans take cost less than a hundred dollars, while generic drugs are often less than a dollar a pill," he told Fox News Digital. What would be affected, Glanville predicted, are newer brand-name drugs still under IP exclusivity, antibody therapies, cellular therapies, gene therapies and personalized cancer vaccines. "Some of these are excruciatingly expensive — $100,000 to $500,000 for a treatment course for a patient. However, they are also often the most effective treatments for certain cancers, autoimmune disorders or rare diseases." The pharmaceutical industry might argue that lowering the prices on these medicines will result in a "dramatic reduction of investment" in creating such breakthroughs, said Glanville. The industry may also argue that these medicines eventually become generic — at which point the prices drop, according to the expert. For more Health articles, visit "From a patient's perspective, the price of medical care in the United States is unsustainable, and extremely expensive medicine is part of that," he said. But "the insurance system and the hospital business also contribute." "If the prices of new medicines are capped, then effort should be made to reduce the cost of clinical trials and drug GMP manufacturing. Otherwise, we will lose a lot of innovation." Greg Norman of Fox News Digital contributed article source: Top 10 most expensive prescription drugs in the US by price and by sales volume
Medscape
12-05-2025
- Health
- Medscape
Hemophilia B: Post-Beqvez, What's Next for Gene Therapy?
In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later, the Pfizer treatment — expected to cost $3.5 million for a one-time dose — got a boost from a phase 3 study published in The New England Journal of Medicine that found annualized bleeding rates fell by 71% in 45 treated men, although 21% resumed prophylaxis. Then, in April 2025, a follow-up analysis of patients from a phase 1-2a study appeared in NEJM . It tracked 14 patients for at least 3 years and reported none had serious treatment-related adverse events after year 1. The newer findings were good news, but they came too late to matter. Pfizer had killed off Beqvez 2 months earlier. 'The company said the discontinuation was due to several reasons,' Reuters news service reported, 'including limited interest in gene therapies for the bleeding disorder.' (Pfizer had earlier dumped a gene therapy for hemophilia A, also blaming a lack of interest.) In fact, it appears that Beqvez was never administered outside clinical research. What does the loss of this treatment mean for the estimated 7250 patients with hemophilia B in the United States and the hematologists who treat them? Here are some questions and answers about the state of gene therapy in hemophilia B. How Does Gene Therapy for Hemophilia Work? Gene therapy, which is now FDA-approved for hemophilia A and hemophilia B, deliver genes for factor VIII or IX via adeno-associated viruses. The liver then begins producing factor, potentially allowing patients to stop or reduce their use of prophylactic therapy. 'In the best-case scenario, [gene therapy] will provide factor levels in the normal range and essentially normal hemostasis without having to give yourself medication for many years,' explained pediatric hematologist Benjamin Jacob Samelson-Jones, MD, PhD, of the Perelman School of Medicine at the University of Pennsylvania and Children's Hospital of Philadelphia, both in Philadelphia, in an interview for a January 2025 Medscape Medical News article. What Made Beqvez Unique? Not a lot compared with its sole competitor. The FDA approved etranacogene dezaparvovec (Hemgenix), the first gene therapy for hemophilia B, in November 2022. It remains available. Beqvez required a smaller dose, and this was thought to be a potential benefit, said hemophilia specialist Steven Pipe, MD, professor of pediatrics and pathology at the University of Michigan, Ann Arbor, Michigan, in an interview. 'There was a hypothesis that lower dosing could provide a theoretical safety advantage — less vector-related toxicity that may manifest with liver toxicity or the overall number of DNA integration events.' (In gene therapy, 'potential integration of therapeutic transgene into host cell genomes is a serious risk' that can lead to mutations and the creation of tumors, according to a 2023 report.) Safety data have not shown a 'clear signal' of a difference in liver toxicity between the therapies, Pipe said. 'Still, we had hoped that clinicians could have a choice to put before patients.' Were There Other Differences Between Beqvez and Hemgenix? According to Pipe, 'the patient eligibility is reduced compared to Hemgenix since it requires patients to have neutralizing antibody (Nab) titers < 1:5 against the SPK100 vector. Best estimates are that 60% of those screened would be eligible. Nab positivity was not an exclusion for Hemgenix.' The costs for the treatments were both around $3.5 million. Why Didn't Beqvez Catch On? 'There are probably multiple reasons for this, including high cost, logistical challenges for centers wanting to offer gene therapy, caution on the part of patients about long-term safety, and the availability of other good treatment options,' said Adam Cuker, MD, professor of medicine in the Department of Pathology and Laboratory Medicine and chief of the Section of Hematology at the University of Pennsylvania, in an interview. He led the 2024 NEJM phase 3 study of Beqvez. In an interview, Samelson-Jones said it's a shame that Beqvez left the market. 'Worldwide, having two safe and very effective gene therapies for hemophilia B would have decreased costs and increased patient access in the long run. To me, this is why this is a loss to the community.' How Do Competing, Non-Gene Therapy Treatments Fit Into the Picture? The FDA recently approved several new subcutaneous treatments as therapies for both hemophilia A and hemophilia B: Marstacimab (Hympavzi), fitusiran (Qfitlia), and concizumab (Alhemo). 'These drugs add to the growing list of treatment options for patients with hemophilia B,' Cuker said. 'It would not be surprising if some patients who otherwise would have been interested in gene therapy choose one of these subcutaneous treatments instead.' Subcutaneous therapy 'may decrease treatment burden enough that a one-and-done treatment like gene therapy is less attractive,' Samelson-Jones said. 'Hympavzi and other new nonfactor therapies may be a bridge that patients want to try before committing to gene therapy. However, nothing prevents bleeding like factor IX, which is what gene therapy provides.' What Should Hematologists Know About Gene Therapy in Hemophilia B? 'The data for gene therapy in hemophilia B are robust,' Cuker said. 'They show impressive efficacy with a durable response in most patients and a favorable safety profile. For my patients who have received gene therapy, the results have been nothing short of life-changing.' He added: 'Patients with hemophilia B deserve to hear about gene therapy as a treatment option from their hemophilia provider. If a patient is potentially eligible for and interested in gene therapy and it is not offered at their home institution, they should be referred to a center with expertise and experience in gene therapy.' What's Next for Gene Therapy in Hemophilia B? Regeneron is exploring second-generation factor IX gene therapy that relies on gene editing, Pipe said. 'This protocol is looking to establish durable, stable expression. If safe and effective, it has the potential to treat pediatric patients.' An ongoing trial seeks to enroll 120 patients and to conclude by April 2026. Meanwhile, another therapy under investigation 'harvests the patient's own B cells by apheresis,' Pipe said. 'The cells are gene edited at a manufacturing facility so that they express factor IX, then the cells are differentiated to long-lasting plasma cells and given back to the same patient. No immunosuppression required since this is autologous cellular therapy.' This therapy 'as the potential for repeat treatment if expression declines over years and can also be dose-titrated to desired expression level,' Pipe said. Be Biopharma has begun a trial of this therapy.
Yahoo
28-03-2025
- Health
- Yahoo
US FDA approves Sanofi's bleeding disorder therapy
(Reuters) -The U.S. Food and Drug Administration approved French drugmaker Sanofi's hemophilia therapy, paving the way for a new type of treatment for patients with the rare blood clotting disorder, the regulator said on Friday. Sanofi's subcutaneous, or under-the-skin, therapy helps prevent bleeding and lowers antithrombin, a protein that delays blood clotting, for hemophilia patients aged 12 years and older. It is a first-in-class, antithrombin-lowering therapy for people with hemophilia A or B, with or without inhibitors. The hemophilia market has been dominated by factor replacement therapies for decades. However, recently, gene therapies such as CSL's Hemgenix and BioMarin Pharmaceutical's Roctavian have been approved as an alternative in the United States. Sanofi's therapy Qfitlia is to be used once every two months, compared to other available treatments such as Pfizer's once-a-week injection Hympavzi and Novo Nordisk's once-daily Alhemo. "Today's approval of Qfitlia is significant for patients with hemophilia because it can be administered less frequently than other existing options," said Tanya Wroblewski, deputy director of the Division of Non-Malignant Hematology in the FDA's Center for Drug Evaluation and Research.
Yahoo
27-02-2025
- Health
- Yahoo
Pfizer's bet on gene therapies in haemophilia has been a bust
Pfizer has ended the global commercialisation of its haemophilia B (factor IX deficiency) gene therapy Beqvez, citing soft demand for gene therapies among haemophilia patients and physicians. This announcement comes after other news that Pfizer ended its partnership with Sangamo Therapeutics for the haemophilia A (factor XIII deficiency) gene therapy giroctocogen fitelparvovec in December 2024. The therapy had received positive Phase III results and was likely to receive US Food and Drug Administration (FDA) approval. However, Pfizer determined that the cost of launching and commercialising the drug would exceed its anticipated sales. Both Biomarin, which markets the haemophilia A gene therapy Roctavian, and CSL Behring, which markets the haemophilia B gene therapy Hemgenix, have announced that the uptake and sales for their respective therapies have been below expectations. The commercial failure of these adeno-associated virus (AAV) gene therapy programmes has come as a surprise to those who were familiar with the opinions of patients and key opinion leaders during clinical trials, as they were generally very positive. However, opinions changed when longer-term efficacy and safety data was published, as many patients who received haemophilia A gene therapies eventually returned to prophylactic treatment as expression of factor XIII reduced over time. Even if patients are interested in these therapies, they may be ineligible. Patients who receive Roctavian must test negative for the AAV serotype used in the gene therapy. Patients with factor inhibitors or certain liver conditions were also excluded from haemophilia gene therapy trials. Even if the patient is eligible, the high list price has discouraged payers from reimbursing the therapies, limiting uptake. Patients may decide to wait for a potential future generation of gene therapies with better safety and efficacy, especially considering the fact that the current prophylaxis treatments have a lower disease burden than previous options. Roche's Hemlibra can be dosed subcutaneously once per month, which is a significant improvement over the multiple infusions per week that were common when these gene therapy programmes started. Pfizer has not abandoned the haemophilia market completely. Instead, it has shifted its focus to Hympavzi, an antibody that received FDA approval for the treatment of haemophilia A and B without factor inhibitors, which is dosed subcutaneously once per week. However, both Pfizer and Roche will closely be watching Sanofi's fitusiran, a small interfering ribonucleic acid therapy that is currently in preregistration for haemophilia A and B with or without inhibitors, and which can be dosed as infrequently as once every two months, significantly reducing the treatment burden on patients. Pfizer's exit from the gene therapy space has reduced the competition for eligible interested patients. Leading data and analytics company GlobalData's analyst consensus forecast predicts that sales for Roctavian will reach $337m in 2030 while Hympavzi will reach sales of $251m in the same year. It is possible that innovative reimbursement strategies such as outcome-based agreements could speed up their slow start in sales. It remains to be seen whether or not Pfizer's strategy is the correct one. "Pfizer's bet on gene therapies in haemophilia has been a bust" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.
