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.
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Yahoo
26 minutes ago
- Yahoo
Family and friends shoulder the real cost of dementia − $224B in unpaid care
About 5.5 million Americans live with dementia, requiring US$53 billion in annual medical spending on doctor visits, hospitalizations, medications, home health aides and nursing homes. But the true cost of dementia care in the U.S. is far higher because it relies heavily on unpaid care from family and friends. I am a researcher who studies health spending, and my colleagues and I set out to quantify the true costs of dementia care – not just to the health system, but to families and communities. After factoring in the value of unpaid care, we found that the total cost of dementia care increases fivefold to $277 billion per year. Our first-of-its-kind estimate of dementia care costs by state also revealed dramatic cost disparities by geography. In the District of Columbia, the average annual cost for a person living with dementia – including both medical spending and the cost of unpaid care – is $37,000, while in West Virginia it is $61,000. Moreover, we found that the total cost was highest in some of the poorest states, with West Virginia and Kentucky in the top three most expensive states despite a low cost of living. The biggest contributing factor to costs was the hours of unpaid care provided. Cost-of-illness studies often include a monetary value of unpaid care, which is referred to by economists as an indirect cost. While no money is changing hands, unpaid care represents the use of a resource – caregivers' time. Caregiving is often rewarding, but it also has tangible impacts on caregivers' well-being. Recent work by our team found that 12% of all caregivers have had to reduce their work hours or quit their jobs altogether. Caregivers who remain in the workforce often miss days of work due to caregiving responsibilities, or they experience reduced productivity while they are at work. Caregivers who aren't working for pay often have to give up other activities, such as volunteer work or leisure time. The indirect cost of care is particularly important in the context of dementia care. Direct medical spending for dementia is relatively small because there aren't many treatments available. While a new Alzheimer's drug, Leqembi, has recently been approved by the FDA, it is available only for a small portion of people living with dementia. People living with dementia also require significantly more hours of care than older adults without dementia. While many elderly adults eventually need help with activities of daily living, such as eating and bathing, people living with dementia often need constant or near-constant supervision. The fact that our study also found indirect costs varying widely by state raises important questions about why hours of unpaid care varies so much geographically. Here, several contributing factors are worth considering. First, many of the states above average in unpaid-care hours also have high rates of chronic conditions such as diabetes. People living with dementia coupled with other illnesses will require more care. Similarly, the severity of dementia affects the amount of care needed. Dementia progresses from mild to moderate to severe, with each stage needing progressively more care. The severity of dementia cases likely varies by state due to different rates of smoking, obesity and other dementia risk factors. And finally, available support from government and community − including the extent of programs such as Meals on Wheels − varies widely in each state. Researchers should continue to look at dementia care costs in the post-COVID-19 era. The stressors on caregivers have likely increased. Almost all states have a shortage of home health workers, and many states have experienced closures of adult day centers, assisted-living facilities and home health agencies over the past year. One-third of Meals on Wheels programs had a waitlist as of 2024. These shortfalls may worsen amid large-scale cuts to federal programs by the Trump administration. The Department of Health and Human Services recently announced plans to dissolve the Administration for Community Living, which oversees the Area Agencies on Aging, a network of nonprofit agencies that administer Meals on Wheels as well as other services aimed to maximize the independence of older adults. Our study highlights the massive contribution of family caregivers and the disproportionate reliance on unpaid care in states with some of the highest poverty rates in the U.S. More work is needed to pinpoint the factors driving these differences and to improve the well-being of caregivers. The Research Brief is a short take on interesting academic work. This article is republished from The Conversation, a nonprofit, independent news organization bringing you facts and trustworthy analysis to help you make sense of our complex world. It was written by: Amy Lastuka, University of Washington Read more: Older adults with dementia misjudge their financial skills – which may make them more vulnerable to fraud, new research finds How being friends with someone who has dementia can be good for you both Does forgetting a name or word mean that I have dementia? Amy Lastuka does not work for, consult, own shares in or receive funding from any company or organization that would benefit from this article, and has disclosed no relevant affiliations beyond their academic appointment.
Yahoo
an hour ago
- Yahoo
Theranica to Present Three Real-World Evidence Studies at AHS Scientific Meeting Highlighting the Impact of the Nerivio® REN Wearable on Migraine Treatment
New studies confirm the clear benefit of early acute treatment, the consistent effectiveness of REN over years, and its clinical benefits in migraine both with and without aura BRIDGEWATER, N.J. and NETANYA, Israel, June 19, 2025 (GLOBE NEWSWIRE) -- Theranica, a pioneer in drug-free neuromodulation treatments for idiopathic pain conditions, today announced the presentation of three new real-world studies at the 2025 American Headache Society (AHS) 67th Annual Scientific Meeting, held in Minneapolis, Minnesota. The posters highlight the safety, efficacy, and versatility of the company's prescribed, FDA-cleared Nerivio® REN wearable, the first-and-only remote electrical neuromodulation (REN) therapy indicated for both acute and preventive treatment of migraine in people aged 8 and older. Among the highlights is a long-term study demonstrating that the REN wearable maintains consistent efficacy for migraine treatment over three years of use, with no signs of tachyphylaxis or 'treatment fatigue.' Throughout the study period, patients experienced consistent Pain Relief, Functional Recovery (see Figure), and freedom from migraine-associated symptoms such as photophobia, phonophobia, and nausea/vomiting.'These findings speak to a critical need in migraine treatment: durability with continued use,' said Stephanie J. Nahas, MD, MSEd, FAHS, FAAN, Assistant Director, Headache Medicine Fellowship Program, Jefferson Headache Center and Professor, Department of Neurology, Thomas Jefferson University. 'Migraine disease is complex and non-curable, yet too often patients struggle to find safe, well-tolerated treatments with sustained efficacy over time, something that is necessary to mitigate chronification and maintain quality of life. Seeing ongoing patient adherence with consistent results over a three-year span, without the risk of treatment fatigue, rebound symptoms, or gradually degraded response confirms the REN wearable as a sustainable, non-drug option for the long-term management of a complex condition such as migraine.' Another poster compares REN's impact in treating migraine with and without aura (MWA and MWoA) in nearly 32,000 patients. Despite having more severe baseline symptoms, patients with aura experienced treatment outcomes that were comparable, and in some measures slightly better than in patients without aura. Pain Relief, Functional Recovery, and freedom from migraine-associated symptoms were evident in both groups, reinforcing the REN wearable's reliable performance across these two major migraine subtypes. 'These studies represent some of the most comprehensive real-world analyses to date on the use of REN in migraine treatment. Together, they demonstrate that REN is not only effective when used early for acute treatment—but also durable over time and beneficial across different migraine subtypes,' said Alit Stark-Inbar, Ph.D., VP Medical Information and Research at Theranica. 'Presenting these data at AHS is an excellent vehicle to share clinically meaningful insights with the professional headache community, and to further support physicians in making evidenced-based decisions when taking care of their patients, being well educated about their treatment options'. These recent clinical data reinforce the REN wearable's unique position as a non-drug treatment of migraine, clinically proven to work both acutely and preventively. For patients facing intolerability or lack of efficacy with medications, or seeking non-pharmacological alternatives, Nerivio delivers safe, effective, and non-disruptive relief, without compromising outcomes. As an active participant in this year's AHS 67th Annual Scientific Meeting, held at the Minneapolis Convention Center from June 18 to 22, Theranica reaffirms its commitment to scientific exchange, clinical innovation, and engagement with the broader headache community. AHS remains a cornerstone in advancing migraine intervention and education, and Theranica is proud to contribute meaningful data that help shape the future of the field. Attendees are invited to visit Theranica at booth #501 to learn more. Information can also be found at About NerivioThe Nerivio® REN wearable is an acute and preventive (dual-use) prescription migraine treatment that works without drugs, needles, or invasive procedures. FDA-cleared for patients 8 and above, it uses gentle electrical pulses on the arm to activate the brain's natural pain regulation system, relieving migraine symptoms during an attack, and reducing the frequency and burden of future episodes when used preventively. Controlled by a smartphone app, the Nerivio REN wearable offers a safe, effective, and easy-to-use way to manage migraine—without the risk of systemic side effects or drug interactions. About TheranicaTheranica is a neuromodulation therapeutics company pioneering drug-free treatments for idiopathic pain conditions. Its FDA-cleared flagship product, Nerivio®, is the first-and-only prescribed REN wearable for both acute and preventive migraine care. Used in more than one million treatments across the U.S., Nerivio provides a much-needed option for migraine patient populations with unique qualities, including children, veterans, individuals managing comorbidities, and women of childbearing age. Dedicated to modernizing pain management without drugs or needles, Theranica continues to develop cutting-edge neuromodulation therapies that reshape the way pain is treated. Theranica Contact: Ronen Jashekronenj@ Media Contact:Grey Matter Marketingmedia@ A photo accompanying this announcement is available at: 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
Medscape
an hour ago
- Medscape
Vaccination Tied to Better Survival in COVID-Linked AKI
Among patients hospitalized with COVID and acute kidney injury (AKI), unvaccinated individuals had longer hospital stays, more frequent need for continuous renal replacement therapy, higher dependence on dialysis at discharge, and substantially higher rates of in-hospital and long-term mortality than their vaccinated peers. METHODOLOGY: AKI affects up to 28%-46% of patients hospitalized with COVID, with effects ranging from mild decline in renal function to dependency on dialysis; however, long-term renal and survival outcomes of these patients remain poorly understood. Researchers conducted a single-center cohort study to evaluate renal outcomes in 972 vaccinated and unvaccinated patients with COVID and AKI admitted to a large urban academic quaternary care hospital from March 1, 2020, to March 30, 2022. Among the included patients, 411 (median age at COVID diagnosis, 67 years; 37% women) were unvaccinated, whereas 467 (median age at COVID diagnosis, 67.4 years; 38.5% women) had received at least two primary-series doses of the Pfizer-BioNTech or Moderna mRNA COVID vaccine or a single dose of the Johnson & Johnson Janssen COVID vaccine prior to hospitalization. The primary outcome was in-hospital mortality, whereas secondary outcomes included long-term mortality, length of hospital stay, and need for renal replacement therapy at discharge. The median follow-up duration was 232 days. TAKEAWAY: Unvaccinated patients had significantly higher rates of in-hospital mortality (adjusted hazard ratio [HR], 5.54; 95% CI, 3.36-9.13) and long-term follow-up mortality (adjusted HR, 4.78; 95% CI, 3.39-6.73) than vaccinated patients. They were also more likely to require continuous renal replacement therapy during hospitalization than vaccinated patients (15.8% vs 10.9%; P = .03); the need for this therapy was associated with increased risks for in-hospital death (adjusted HR, 2.82; 95% CI, 1.88-4.25) and long-term mortality (adjusted HR, 2.44; 95% CI, 1.73-3.42). = .03); the need for this therapy was associated with increased risks for in-hospital death (adjusted HR, 2.82; 95% CI, 1.88-4.25) and long-term mortality (adjusted HR, 2.44; 95% CI, 1.73-3.42). Unvaccinated patients had higher odds of being discharged on renal replacement therapy (odds ratio, 2.56; 95% CI, 1.52-4.30) and had a significantly longer median length of hospital stay (12 days vs 8 days) than vaccinated patients. IN PRACTICE: 'Patients who completed the primary vaccine series with COVID-19 infection and AKI had a lower mortality rate and were less likely to remain on renal replacement therapy at the time of discharge,' the authors wrote. SOURCE: This study was led by Niloofar Nobakht, MD, Division of Nephrology, Department of Medicine, David Geffen School of Medicine at the University of California, Los Angeles. It was published online on June 17, 2025, in Kidney Medicine . LIMITATIONS: Missing baseline creatinine data limited the ability to stage AKI and likely contributed to the significant differences in AKI staging observed between the vaccinated and unvaccinated cohorts. This study lacked data on ventilation status and other indicators of infection severity in patients admitted to ICUs who received continuous renal replacement therapy. Information regarding COVID booster vaccinations was not available. DISCLOSURES: One author received support from the National Institutes of Health, the Smidt Family Foundation, the Factor Family Foundation, the Davita Allen Nissenson Research Fund, and the Ralph Block Family Foundation.
