Latest news with #haemophilia
France 24
6 days ago
- Business
- France 24
Pharma giant Roche sees income soar in first half
It also reported that four potentially "practice-changing" drugs -- to treat Alzheimer's, Parkinson's, haemophilia and an antibiotic to fight a drug-resistant bacteria strain -- had advanced to the final stage of clinical development. Net income jumped 17 percent to reach 7.8 billion Swiss francs ($9.85 billion), while the Basel-based group confirmed its full-year outlook. Its sales exceeded forecasts, rising four percent to 30.9 billion Swiss francs -- or a seven-percent hike excluding currency effects, the world leader in cancer treatments said in a statement. Roche has two branches: pharmaceuticals and diagnostics. Its pharmaceuticals division saw sales rise 10 percent, at constant exchange rates, to 24 billion francs, primarily thanks to five "growth driver" medicines, including for breast cancer and asthma, which achieved total sales of 10.6 billion francs. That was 1.7 billion more than during the first half of 2024. Sales in its diagnostics division, however, were flat at constant exchange rates, at 6.95 billion francs. "We received numerous important approvals and reported positive data in disease areas with high unmet medical need," said Roche chief executive Thomas Schinecker. "Over the past six months, we have made significant progress in our pipeline and advanced four potentially practice-changing therapies into the final phase of clinical development," he said. These are NXT007 in haemophilia A, trontinemab in Alzheimer's disease, prasinezumab in early-stage Parkinson's disease, and zosurabalpin, "a novel antibiotic that could become the first in over 50 years to tackle a type of bacteria that has become resistant to most other treatments". For the full year, Roche said it was still targeting mid-single-digit sales growth excluding currency effects, equivalent to around five percent growth. In April, Roche announced plans to invest $50 billion in the United States over the next five years, following in the footsteps of Swiss rival Novartis, with US President Donald Trump's tariff war fuelling uncertainty in the sector. The United States is a key market for the pharmaceutical industry, representing around half of sales in Roche's pharmaceutical division.
Telegraph
09-07-2025
- Health
- Telegraph
Factor VIII was advertised as a wonder drug
Scroll to begin It was said to offer people with haemophilia a passport to a normal life But in reality, it was a poison made by American pharmaceutical companies using thousands of high-risk donors One infected plasma donor was all it took to contaminate an entire batch Supplies were imported into Britain in the 1970s and 1980s for use by doctors facing a shortage of blood-clotting treatments Thousands of people were infected with HIV and hepatitis C as a result Many died. Others struggle on, still awaiting justice This is their story Cara McGoogan 09 July 2025 2:00pm BST Gift this article free The infected blood scandal can only be described as a catalogue of heartbreak. More than 30,000 people in the United Kingdom were affected by contaminated blood products or transfusions, nearly 3,000 of whom have died. More die each year, while survivors continue their decades-long battle for truth and compensation in the face of cover-up and dismissal. In May 2024, after five years of investigation, an inquiry into the worst treatment disaster in NHS history will publish its final report. Victims are hoping for answers on how the crisis was allowed to unfold. But the heart of the story had already been written, in the shape of distorted lives, pain, stigma, sickness and loss. And as a new report released on Wednesday said, its victims are being "harmed further" still by long waits for money promised to them by the state. Gwynneth and Steven Walker 'There was never a future for him, it was always just a few more months or another year if you were lucky.' Steven Walker was three years old in 1983 when he was found to have severe haemophilia A. The diagnosis came after months of being shuttled back and forth to the doctors with unexplained bruises, a result of the genetic bleeding disorder, which prevents the body from forming clots. It was almost a relief to his mother, Gwynneth, who had faced accusations of harming her son. 'We tried to wrap him up in cotton wool,' she recalls. With a diagnosis, Steven could start treatment with cryoprecipitate, a frozen plasma product made from the blood of a single donor. But in June 1984, after the family had moved to Norwich, he began a new regime – one that would ultimately prove deadly. Steven received his first dose of Factor VIII on the recommendation of a local doctor, who suggested he be treated at home with the blood-clotting protein. Like many parents of children with haemophilia, Gwynneth learnt to mix the Factor VIII concentrate with sterile water and inject it into her son's arm. While she was completely unaware of the risk in doing so, by 1984, it was already known that acquired immunodeficiency syndrome (Aids) was caused by a blood-borne virus, HTLV-III, which could be passed on through the type of treatment he was receiving. At that stage, there was an alternative, safe version of the imported Factor VIII available, which had been heat-treated to kill off potential viruses. The UK had been slow to fully adopt it, in part because of cost, but haemophilia treatment guidelines set out that new patients should be given the heat-treated product or cryoprecipitate. Still, Gwynneth took her doctor's word, and did as they had suggested, unwittingly using the contaminated version of Factor VIII. Many other patients would receive the same, into 1985, because of a sluggish response by officials and the NHS to adopt the new, more expensive but safe treatment. Within a year, serious illness had gripped the family. In December 1985, her skin turned yellow with jaundice. While receiving treatment in hospital, she was told she should be tested for hepatitis B – a liver infection – because of Steven's haemophilia. 'I had never heard of hepatitis,' Gwynneth, 67, says. Doctors and the government had known Factor VIII could contain hepatitis since the 1970s, but had continued to import higher risk American supplies, rather than building up UK stocks of the plasma treatment. Both Gwynneth and Steven, then four, tested positive for the illness. She had contracted it by removing a splinter from her son's finger with her mouth. 'You wouldn't do that if you thought you were at risk, would you?' she says. But worse was still to come. Further tests revealed Steven had contracted HIV. In the wake of the diagnosis, Gwynneth was told by doctors that she had to protect her son from his younger brother, then aged one. Steven was given 'two years to live', she says, 'and not much more'. For six weeks Gwynneth locked herself away at home with her sons, trying to process the news. She decided not to tell Steven the news of his diagnosis, choosing to put off doing so for as long as she could. 'I was absolutely distraught,' Gwynneth says. 'I was looking at my child, planning his funeral. And wondering about the little one and how I could protect him. 'You look at your own flesh and blood and you're frightened to cuddle him. It ended up crippling me. You put your trust in doctors and they told me untruths.' In an attempt to slow his deterioration to Aids-related illness, Steven was prescribed the antiretroviral drug AZT, a retired cancer treatment that often had the opposite effect because it was prescribed in dangerously high doses in the early days of the Aids crisis. But the entire course of his life had changed and by 13, he was too frail to go to school, forced to watch on as his little brother ticked off childhood landmarks, such as riding a bike and getting a first girlfriend, that he was unable to match. 'He was brutally wounded by that,' says Gwynneth. 'He was starting to get angry and annoyed, so it seemed he was getting to an age where maybe he needed to know [about his diagnosis].' On learning that he had HIV, Steven was 'horrified'. 'He felt cheated,' says Gwynneth, and retreated into himself as his teenage years progressed. She taught her son to lie about his condition, in order to try to protect him from the stigma that surrounded it. 'I'd been teaching them [her sons] how to keep secrets since they were small about trivial stuff, so that when the biggie came along they'd keep the secret,' she says. He couldn't make friends, go to university or get a job because he was too unwell. The only guests at his 18th birthday party were his nurse and his driving instructor. 'There was never a future for him, it was always just a few more months or another year if you were lucky. There are several times I reflect back on and wish he'd died sooner because he suffered so much and was so ill,' says Gwynneth. 'From 13, his life was really over. He died at 37, but after 13 the rest of it was just suffering.' Gwynneth recalls one of several recurring nightmares Steven would endure – of making breakfast for two daughters he would never be able to have. 'He felt that his life had been robbed from him and he felt angry,' she says. As an adult, Steven turned to alcohol and drugs, including heroin. For the last six years of his life, he would fall asleep on his sofa each night after drinking several bottles of wine, topped off with whiskey. 'We just watched him waste away,' says Gwynneth. 'He was about 5ft 11 and when he died he was under seven stone.' In July 2017, two months before Steven died, then-prime minister Theresa May announced the Infected Blood Inquiry. It was the smallest measure of relief, Gwynneth says, for a life pockmarked by avoidable agony. The inquiry had raised Steven's hopes for compensation, which he dreamed of using to buy himself an apartment, but years of government inaction meant nothing was forthcoming before he died. 'He used to say: 'The government has done this to me',' she says. 'At every step of the way there were people at fault: from the doctors who knew [the risks and didn't tell us] to the government ministers who have failed us ever since. 'I feel like he was murdered,' says Gwynneth. 'He didn't live long enough, and now my son will never get justice.' Jo-Anne Cohrs and Keith Proud 'I whispered in his ear that Jesus loved him, that he'd done a good job, and to go when he'd had enough.' Keith Proud entered Jo-Anne Cohrs' life with uncharacteristic confidence in 1980, when he gatecrashed her 21st birthday party. Keith was 10 years older but, like Jo-Anne, an undergraduate at Sunderland Polytechnic. He joined as a mature student after a childhood interrupted by haemophilia A. Keith was a shy media studies student. Jo-Anne was a lively musician and member of a Christian rock band. They were exact opposites: she thought he was quiet, he didn't quite know what to make of her high energy. 'But funnily enough we both rubbed off on each other quite well,' says Jo-Anne, 64. 'I sort of tamed down a bit as I got to know him and he became a bit more outward.' After university, Jo-Anne and Keith toured Europe, driving to Italy and back again. Every other day on the trip, Keith would inject himself with Factor VIII, which he had been using since it first became available in the mid-1970s. Within three years, they were planning their wedding. But shortly before the big day in December 1983, Keith came down with what appeared at first to be a heavy flu. 'He was very cold,' says Jo-Anne. 'Looking back, I think he might have even had it then.' It was a new illness that had emerged in the US among gay men – Aids. Totally unaware of the true severity of his condition at the time, Keith eventually bounced back from what had developed into a bout of pneumonia and returned to work as a lecturer in 1984. For a few months, life for the couple was good. Jo-Anne was a teacher's special needs assistant and after years of muddling their way through studies, the couple suddenly felt flush with cash. 'We had so much money we didn't know what to do with it,' she says. For Christmas, they splashed out on gifts for their family. But almost immediately after their 'fantastic' festive period with loved ones, the couple received a life-altering letter from Keith's local hospital. 'He had HIV,' says Jo-Anne. 'We were both shocked.' 'Over the next two or three months, Keith started to get very, very afraid,' she says. 'He started to come down with things: red dots in his mouth, then his skin had lumps and bumps from Kaposi's sarcoma, lots of candida and thrush in his mouth. He couldn't keep anything down, the tablets made him sick; everything made him sick.' Physical pain and symptoms were accompanied by the vicious stigma that defined the early days of the Aids crisis, leaving many who suffered with the condition socially outcast and isolated. Jo-Anne tried to shield Keith from as much of it as she could. She even stopped letting him watch live television at home, for fear he would see a public health advert or coverage of the crisis that would upset him. 'I didn't want him to be frightened,' she says. 'I wanted him at least to have peace at home.' But the outside world was impossible to control, and when Keith told his dentist he had HIV, they refused to treat him. They told him 'don't bring your wife back either', says Jo-Anne. 'Those words are cemented in my brain.' Within a year of his diagnosis Keith and Jo-Anne were forced to leave their jobs; he had become too unwell to work and she opted to stay at home to care for him. Their social life ground to a complete halt and their increasingly precarious financial situation forced the couple to apply for benefits. The local church became a vital lifeline of support. Fellow congregants would visit daily, and go to the shops on their behalf. Meanwhile, doctors advised Jo-Anne to wear gloves to protect herself from Keith. She refused. 'I'm not going to treat my husband with gloves,' she recalls thinking. 'If he needs a cuddle or a kiss or closeness, whatever he needs I'll be there.' Despite Jo-Anne's care, Keith's health continued to decline over the next two years and by the beginning of 1987, the pair became aware his death was fast approaching. He passed away in the spring, on March 6, with his wife, then 27, and family at his side. 'I whispered in his ear that Jesus loved him, that he'd done a good job, and to go when he'd had enough,' Jo-Anne says. After years of anguish that followed, Jo-Anne painstakingly rebuilt her life, remarrying and having a daughter with her new partner. But she still only lives half a mile from where she first met Keith at the party in Sunderland. 'I'm not over how things turned out,' says Jo-Anne, adding that what upsets her most deeply now is the 'incredulous amount of time' it has taken the Government to compensate victims. The government has set aside £11.8bn for those affected by the scandal, but many have expressed concerns about the way the scheme has been managed to date. The long wait times some have endured, or continue to endure, for payment is a particular issue. 'There are still a lot of people who are seriously ill and who are going to meet an untimely, undignified death,' says Jo-Anne. 'And for those people, they should be given everything they need until they die. It's upsetting for me. But at least I'm not facing a relative who's going to slip away with that cruel death right in front of me, right now.' Christopher, Gary and Kelvin Marsh 'I'm still here and no one else is. My world has just fallen apart.' Haemophilia often affects more than one child within a family, passing most commonly from mothers to sons. That was the case for the Marsh family – Gary, Kelvin and their youngest brother Christopher, all three of whom were born with haemophilia A. As a child, Christopher says, he hated having treatment for his illness. 'I used to run around the hospital not wanting it,' he says. 'But Mum and Dad would say I'd have to sleep there if I didn't.' He remembers being given cryoprecipitate. His brothers, meanwhile, received Factor VIII. What they shared was a refusal to let illness get in the way of life – playing rugby, skateboarding and riding motorbikes instead. That was, until Gary and Kelvin tested positive for HIV – a by-product of the Factor VIII they had been given – in the 1980s. Christopher had avoided contracting the illness but was found to have been infected with hepatitis C, which slowly attacks the liver and can lead to cirrhosis and cancer. 'It changed our lives instantly,' says Christopher, 49. 'We were living with the fact that they were like a ticking time bomb and could die at any time.' While Christopher's elder brothers both went on to marry and have families of their own, the family's worst fears were realised when Gary became the first of the three to fall seriously ill at the beginning of the 1990s. He initially tried to maintain the partygoer spirit that had defined his life until that point, but eventually realised those days were behind him when he was forced to leave his stepdaughter's wedding because of sickness. 'That really upset me because I couldn't understand,' says Christopher. 'He had been so healthy and loved life, then I saw him deteriorating.' His brother, 13 years his elder, suffered with ulcers, contracted pneumonia and 'became gaunt' as his condition took hold. On Boxing Day 1992, Gary died from Aids-related illness, aged 30. He was never able to take the trip to the US and the Bahamas the siblings had been planning together, shortly before he passed away. 'Kelvin said, 'Gary would have wanted us to go',' recalls Christopher. 'So we still went.' But being positive for HIV himself, Kelvin couldn't help but see his own future in his brother's death. He was in his late-20s and married with two young children, a girl and a boy. 'I remember being on holiday and him sitting there saying he wasn't scared of dying himself, but for the ones he would leave behind,' says Christopher. Eight years later, Kelvin's HIV progressed to Aids. After a failed liver transplant, he died in April 2000, aged 35. By the end of the year, their mother had also passed away. 'Everyone said it was from a broken heart, but obviously you can't prove that,' says Christopher. In the wake of repeated tragedy, Christopher sunk into depression. 'I thought, as soon as I hit my 30s I'm going to die,' he says. 'Because both my brothers were in their 30s when they died. 'We never got a choice. We believed we were given the best care we could have been, not knowing it was contaminated.' Christopher is currently undergoing treatment to try to clear his hepatitis C, but had to stop work as a painter and decorator last year because of pain in his lower abdomen. His life has been curtailed in more profound ways, too. While Christopher has a partner, step-children and grandchildren, he never had children of his own. He was too scared to try, in case he transmitted his illness to them via his partner. And like so many others in the infected blood scandal, he remains plagued by survivor's guilt. 'I'm still here and no one else is,' he says. 'My world has just fallen apart. I feel alone. I had two brothers and now it's just me and my sister left. I miss them so much.' Gary Webster 'We'd had Factor VIII pumped into us; people were going yellow; people were getting ill and we knew there was something wrong.' Life changed overnight for Gary Webster in 1975 when, aged nine, he started boarding at the Lord Mayor Treloar College in Hampshire, a school for children with physical disabilities. He learnt to sail, struck up friendships and ran wild in the dormitories at night. He also had his first dose of Factor VIII. The treatment initially gave him a sense of freedom after a childhood defined by haemophilia – one spent waiting hours for cryoprecipitate to defrost, transfuse and take effect. Similarly relishing in the newfound liberation was Gary's best friend Stephen, whose family were farmers in Norfolk. The pair stayed up late talking to local people on their CB radio and, when they were older, snuck up to London to watch David Bowie in concert. They planned to live together after leaving school at 18. Whenever they had a bleed, day or night, they could go to the onsite NHS-run haemophilia centre for treatment. But amid the idyllic life Treloar's initially appeared to offer, there were also signs that things were, in fact, going badly wrong. Gary and other boys were regularly bed-ridden for weeks with serious illnesses, including hepatitis and glandular fever. And by the outset of the 1980s, Gary noticed the amount of Factor VIII they were given dramatically increased. 'I'd have to give myself injections every other day,' he says. 'It was so monotonous. I thought, 'Why do I need it when I haven't got a bleed?'' When Gary questioned the school's doctors over the regime, he was told it was to prevent bleeds. But his friend Stephen had another theory: 'I'm sure they're bloody experimenting on us,' he said. In their final few months at the school, in springtime 1983, Gary and Stephen, then 17, were abruptly called out of class. The doctor wanted to see them. 'I've got something to tell you,' said Dr Mounir Wassef. Both the boys had signs of a newly emerging illness, Aids. 'It is incurable and we cannot guarantee you will be alive in two to three years,' he said. They were among the first people in Britain to be diagnosed with the life-threatening condition, a year before HTLV-III was discovered and the link between Factor VIII and the illness was accepted by the government. 'We just couldn't believe what he was saying,' recalls Gary, 59. 'We were shocked but we were young. We looked at each other and half smiled. Then we went back to class.' The doctors left it to Gary and Stephen to inform their parents. 'My mum and dad had to inject me when I was a young lad,' says Gary. 'They put their trust in the college, the doctors and the staff. They felt guilty and upset.' His parents were told little about their son's treatment by the doctors at Treloar's. They didn't know what brand of Factor VIII he was receiving, or how often he was being treated, and nothing of the associated risks. They were even kept in the dark when Gary contracted hepatitis and glandular fever. 'If it wasn't for me telling them what was going on while I was at the college, they wouldn't know anything,' he says. After receiving their diagnoses, Stephen's health declined more rapidly than Gary's and by 1991 he was suffering with Aids-related illness. For a while, he tried to hide it, but eventually he confided in his friend. 'I think I've got it,' Stephen said. 'Join the club,' replied Gary, whose own descent into illness was also quickening. Later that year, Gary was told by Stephen's father that his friend had been hospitalised with pneumonia. Knowing Stephen would never make it out, he made the long, sorrowful journey from Hampshire to Norfolk to say goodbye. Like other Treloar's boys, Gary had grown accustomed to seeing school friends grow poorly, witnessing their decline at regular reunions, and paying his respects at the funerals that followed. After Stephen's death he stopped going to both. 'It got too hard,' he says. 'Every time you went to the reunions there were less and less people. The funerals were every year from the late Eighties into the Nineties. 'I had a big group of friends who were haemophiliacs – and [then] there weren't that many of them any more.' In 1996, an effective antiretroviral treatment based on a new combination of drugs was introduced, helping Gary to come back from the brink. 'It was pot luck that treatment came in when it did,' he says. 'Others got ill too early.' But 15 years later, in 2011, he was forced to retire, aged 46, from his job at the Red Cross because of hepatitis C. It was a second, major professional blow, having earlier been forced to abandon his dream career as a tailor in the 1990s. 'It was so difficult to get up and go to appointments,' he says. 'The fatigue and brain fog was just awful.' Without work, Gary turned his focus to campaigning, joining a group of boys from Treloar's who had come together to demand answers over how 122 of the school's pupils had been allowed to contract viruses including hepatitis C and HIV. He was fuelled by anger about his own condition, and the fate met by Stephen, one of 80 boys from the school who died as a direct result of the infected blood scandal. 'I went on my first campaign demonstration to [then-Prime Minister] David Cameron's constituency,' he says. In 2017, Gary was one of more than 1,000 survivors and bereaved relatives who came together and sued the Department of Health for misfeasance in public office. Soon after, Theresa May announced the Infected Blood Inquiry. 'I campaigned to get the inquiry going with Theresa May,' says Gary. 'I've made posters and press releases, written to MPs and travelled to London with my MP and local councillor.' The inquiry opened in 2019. Two years later, it dedicated a week of hearings to Treloar's, during which evidence emerged confirming that children at the school had been part of research, including how effective preventative treatment was for haemophiliacs, and what brands of Factor VIII were more likely to transmit hepatitis. Both these trials involved being injected with high-risk Factor VIII imported from the United States and, at times, placebos. Present at the hearings, Gary realised he had been put onto a trial in his first year at the school, without being told. Its former headmaster, Alec Macpherson, said he had known about the research but avoided probing the school's NHS-affiliated doctors because he trusted them. 'We knew our own story; we'd lived it,' says Gary. 'We'd had Factor VIII pumped into us; people were going yellow; people were getting ill; we knew there was something wrong. 'We also knew we could have been on cryoprecipitate. But that week, Alec Macpherson said 'doctors are gods' and [admitted] that he didn't question them in his weekly meetings. We found out we had been on placebos for trials.' On the back of the revelations, Gary moved to sue Treloar's for breaching its duty of care. 'Treloar's have to answer for what happened under their watch,' he says. 'We were at school without our parents – they basically were our parents.' Looking ahead, he wants to see a memorial built at Treloar's for the pupils who died – a measure of justice, in other words, at long last. 'It has to be a life-changing amount of money,' he says of the promised compensation. 'Eighty boys have passed and that's only going to increase over the next few years. People's lives have been ruined; people have lost loved ones – how much [value] do you put on that?' Listen to Bed of Lies, a six-part Telegraph podcast laying bare one of the biggest medical disasters in history, on Apple Podcasts, Spotify or your preferred podcast app. Header video credit: Bad Blood: A Cautionary Tale, Blood Brothers - The America Blood Scandal / Image Productions film / H.O.P.E films.
Yahoo
20-06-2025
- Health
- Yahoo
Sobi will showcase extensive research and clinical outcomes at ISTH 2025
STOCKHOLM, June 20, 2025 /PRNewswire/ -- Sobi® (STO: SOBI) will be presenting a wide range of clinical study updates at the annual International Society on Thrombosis and Haemostasis (ISTH) Congress in Washington (21-25 June). With ten (10) presentations, Sobi's clinical teams will showcase new data for avatrombopag to treat children with ITP, surgery outcomes using efanesoctocog alfa, and abstracts on specific long-term haemophilia studies. "Attending ISTH is an important part of our commitment to collaboration and knowledge sharing so we can further advance treatments for those living with haemophilia, immune thrombocytopenia, and other rare blood conditions", said Lydia Abad-Franch, MD, Head of R&D and Medical Affairs, and Chief Medical Officer at Sobi. "We are proud to be showcasing abstracts as part of the ISTH programme, including Doptelet abstracts that present new data related to children living with ITP, Altuvoct outcomes for perioperative management, and joint health outcomes from a long-term extension study, as well as final outcomes from the B-MORE study. Many people living with rare conditions have unmet needs, and we are proud to present additional data advancing treatments for these rare conditions." Data to be presented at ISTH 2025 ALTUVOCT® (efanesoctocog alfa) OC 64.4: Major Surgical Outcomes with Efanesoctocog Alfa: 4 Years' Experience in the XTEND Clinical Program Presenting author: Liane Khoo Oral presentation Session date: Tuesday 24 June Session time: 14:45 - 16:00 EDT Location: 151 A&B, Walter E. Washington CC Convention Center OC 20.5: Treatment of Bleeding Episodes with Efanesoctocog Alfa in Children: XTEND-ed Second Interim Analysis Presenting author: Lynn Malec Oral presentation Session date: Sunday 22 June Session time: 14:45 - 16:00 EDT Location: Ballroom A-C, Walter E. Washington CC Convention Center PB0847: Minor Surgeries Outcomes with Efanesoctocog Alfa: 4 Years' Experience in the XTEND Clinical Program Presenting author: Pratima Chowdary Poster presentation Session date: Monday 23 June Session time: 13:45 – 14:45 EDT Location: Exhibition Hall PB1425: Joint Health Outcomes with Efanesoctocog Alfa in Adults/Adolescents from XTEND-1 Continuing XTEND-ed Presenting author: Christoph Königs Poster presentation Session date: Tuesday 24 June Session time: 13:45 – 14:45 EDT Location: Exhibition Hall General Haemophilia PB0778: Addressing unmet medical needs and health inequities in haemophilia A: expertconsensus statements Presenting author: Cédric Hermans Poster presentation Session date: Monday 23 June Session time: 13:45 – 14:45 EDT Location: Exhibition Hall PB0816: Extravascular distribution of factor IX: evidence and relevance for haemophilia B replacement therapy Presenting author: Cédric Hermans Poster presentation Session date: Monday 23 June Session time: 13:45 – 14:45 EDT Location: Exhibition Hall PB1439: Monitoring Joint Health in Haemophilia Patients in Spain: Updated Analysis of the JOIN-US Project Presenting author: María Teresa Álvarez Román Poster presentation Session date: Tuesday 24 June Session time: 13:45 – 14:45 EDT Location: Exhibition Hall ALPROLIX® (rFIXFc) PB0868: Real-World Effectiveness and Usage of Recombinant Factor IX Fc: Final Data from the B-MORE Study Presenting author: Heidi Glosli Poster presentation Session date: Monday 23 June Session time: 13:45 – 14:45 EDT Location: Exhibition Hall Doptelet® (avatrombopag) PB0348: Consistent Response to Avatrombopag for the Treatment of Children with ITP Across Various Baseline Characteristics Presenting author: Rachael F. Grace Poster presentation Session date: Sunday 22 June Session time: 13:45 - 14:45 EDT Location: Exhibition hall PB0364: Evaluation of Efficacy and Safety of Avatrombopag in Children with ITP Based on Disease Duration Presenting author: Rachael F. Grace Poster presentation Session date: Sunday 22 June Session time: 13:45 - 14:45 EDT Location: Exhibition hall OC 65.3: Real-World Outcomes of Avatrombopag Treatment in Primary ITP Stratified by Prior TPO-RA Exposure Presenting author: Srikanth Nagalla Oral presentation Session date: Tuesday 24 June 2025 Session time: 14:45 – 16:00 EDT (Presentation time currently in programme: 15:15 – 15:30 EDT) All abstracts are accessible through the official ISTH website. However, any late-breaking abstracts will only be made available later. About ALTUVOCT® (efanesoctocog alfa) ALTUVOCT® (efanesoctocog alfa) is indicated for the treatment and prophylaxis of bleeding in patients with haemophilia A. ALTUVOCT can be used for all age groups and any disease severity. About Elocta®/Eloctate® (efmoroctocog alfa) Elocta®/Eloctate® (efmoroctocog alfa) is a treatment and prophylaxis of bleeding in patients with haemophilia A. Elocta/Eloctate can be used for all age groups. About Alprolix® (eftrenonacog alfa) Alprolix® (eftrenonacog alfa) is a treatment and prophylaxis of bleeding in patients with haemophilia B. Alprolix can be used for all age groups. About Doptelet® (avatrombopag) Doptelet® (avatrombopag) is indicated for the treatment of primary chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments, and a treatment of severe thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo an invasive procedure. About Sobi Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Sobi has approximately 1,900 employees across Europe, North America, the Middle East, Asia and Australia. In 2024, revenue amounted to SEK 26 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at and LinkedIn. About Sanofi Sanofi is an R&D driven, AI-powered biopharma company committed to improving people's lives and delivering compelling growth. Sanofi apply deep understanding of the immune system to invent medicines and vaccines that treat and protect millions of people around the world, with an innovative pipeline that could benefit millions more. Sanofi's team is guided by one purpose: to chase the miracles of science to improve people's lives; this inspires Sanofi to drive progress and deliver positive impact for Sanofi's people and the communities Sanofi serve, by addressing the most urgent healthcare, environmental, and societal challenges of our time. Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY About the Sanofi and Sobi collaboration Sobi and Sanofi collaborate on the development and commercialisation of Alprolix and Elocta/Eloctate. The companies also collaborate on the development and commercialisation of efanesoctocog alfa, or ALTUVIIIO® in the US, Japan, and Taiwan, and Altuvoct in Europe. Sobi has final development and commercialisation rights in the Sobi territory (essentially Europe, North Africa, Russia, and most Middle Eastern markets). Sanofi has final development and commercialisation rights in North America and all other regions in the world excluding the Sobi territory. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information was brought to you by Cision The following files are available for download: Sobi will showcase extensive research and clinical outcomes at ISTH 2025 View original content:
News.com.au
21-05-2025
- Health
- News.com.au
Aussie researchers and ASX biotechs driving paediatric drug breakthroughs
Australia has become well-known for its paediatric drug discovery with several life-altering treatments CSL is a global leader in developing therapies for children with haemophilia and other serious blood disorders Neurotech International is at forefront of developing cannabinoid-derived therapies for paediatric neurological disorders Australia may lack the size and funding of larger global biotech hubs, but it consistently punches above its weight in paediatric drug discovery. From life-altering treatments for rare genetic conditions to vaccines that have reshaped public health, Aussie researchers and ASX-listed companies have and continue to deliver breakthroughs improving and in some cases saving young lives globally. Among these advancements is Australia's largest biotech CSL (ASX:CSL), which has become a global leader in developing therapies for children with haemophilia and other serious blood disorders. Its paediatric haemophilia products — including Afstyla and Hemlibra — allow young patients to live more freely and safely. CSL also played a pivotal role in bringing Gardasil to the world – the revolutionary human papillomavirus (HPV) vaccine co-developed by former Australian of the Year Professor Ian Frazer at the University of Queensland. The vaccine is helping to protect millions of girls and boys from HPV-related cancers. In the 1990s Dr Sandra Anderson and colleagues at Sydney's Royal Prince Alfred Hospital (RPAH) started research into how inhaled mannitol can be used to diagnose asthma. Their work resulted in development of two new pharmaceutical products: • Aridol – to identify bronchial hyperresponsiveness and assist in asthma diagnosis. • Bronchitol – to treat patients with bronchiectasis and cystic fibrosis (CF) by helping clear mucus from their lungs. In 2001 Pharmaxis now Syntara (ASX:SNT) licensed patents for respiratory products from the Central Sydney Area Health Service (which included RPAH). Pharmaxis funded later stage clinical trials necessary to get Aridol and Bronchitol, which are now available world-wide, approved. Its shares peaked in 2007 at over $4 a share – for a roughly $800 million market cap – mainly on its Bronchitol hopes. The company announced sale of its mannitol respiratory unit, which included Aridol and Bronchitol, in October 2023 along with its rebrand to Syntara and change of focus to haematological malignancies. On the ASX, several healthcare players are cooking up the next-gen of paediatric therapeutics. Neuren targets various rare diseases In 2023 Neuren Pharmaceuticals (ASX:NEU) became the darling of the Aussie bourse after its US partner Acadia Pharmaceuticals secured US Food and Drug Administration (FDA) approval for trofinetide to treat Rett syndrome. Marketed as Daybue, it is the first drug to treat the rare neurological disorder which emerges in infancy and is a profoundly debilitating neurological condition affecting predominately girls. And Neuren hasn't stopped there. It's also advancing NNZ-2591 in clinical trials for a suite of other rare paediatric syndromes – including Angelman, Phelan-McDermid, and Pitt-Hopkins — for which there are currently no approved treatments options. Neuren is also developing NNZ-2591 to treat neonatal hypoxic-ischemic encephalopathy (HIE). "HIE is a devastating type of brain injury caused when a baby's brain does not receive enough oxygen or blood flow before or shortly after birth," CEO and managing director Jon Pilcher told Stockhead. "It is one of the leading causes of neonatal death and neurodevelopmental disability. He said Neuren aimed to provide a highly differentiated form of treatment continuing beyond the neonatal intensive care unit to target the long-term outcomes for these children. "Neuren's whole business is aimed at trying to improve the lives of the children and their families that are so impacted by all of these conditions," he said. "The whole team is highly motivated by that mission, building on scientific foundations from Australia and New Zealand." Argenica Therapeutics (ASX:AGN) has been targeting neonatal HIE for nearly a decade, alongside other acute neurological conditions such as stroke and traumatic brain injury, with its novel therapeutics aimed at reducing brain tissue death. Its first published paper out of the Perron Institute in Western Australia was in 2018. The study was run by Dr Adam Edwards, who leads Argenica's preclinical neonatal HIE program. Both Argenica's therapeutic candidates ARG-006 and ARG-007 are being investigated for their respective safety and efficacy profiles in HIE, offering different therapeutic options for the treatment of the condition. The FDA has granted both ARG-006 and ARG-007 ODD and Rare Paediatric Disease Designations (RPDD), qualifying Argenica for significant incentives. Neurotech tackles paediatric neuro disorders Neurotech International (ASX:NTI) is at the forefront of developing cannabinoid-derived therapies for paediatric neurological disorders, led by its proprietary oral formulation, NTI164. Derived from a unique cannabis strain with ultra-low THC ( Following the path of successful companies like Neuren, Neurotech is advancing treatments for Rett syndrome, having completed a phase I/II trial with promising results and an extension study currently underway. NTI164 has been granted orphan drug designation (ODD) by both the US FDA and the European Commission, reinforcing its potential as a differentiated therapy in this rare disease. Beyond Rett, Neurotech has completed multiple paediatric clinical programs with NTI164, including: • A phase I/II and phase II/III trial in Autism Spectrum Disorder (ASD) • Phase I/II trials in PANDAS/PANS (Paediatric Autoimmune Neuropsychiatric Disorders and Acute-Onset Neuropsychiatric Syndrome) "Neurotech is redefining the potential of cannabinoid-derived therapies in paediatric neurology," newly appointed CEO and managing director Dr Anthony Filippis told Stockhead. "With NTI164, we're not just targeting symptoms, we're aiming to shift the treatment paradigm for conditions like Rett syndrome, ASD, and PANDAS/PANS. "The orphan drug designations in the US and Europe affirm the unique promise of our approach, and we're committed to advancing NTI164 towards approval and access for the families who need it most." Dimerix tackles leading cause of kidney failure in children Dimerix (ASX:DXB) is focused on the rare kidney disease focal segmental glomerulosclerosis (FSGS) with its lead phase III drug candidate DMX-200. CEO and managing director Dr Nina Webster told Stockhead FSGS affects both adults and children damaging filtering units of the kidneys, leading to permanent damage and eventual organ failure. 'FSGS is one of the leading causes of kidney failure in children, with 20% of all presentations of nephrotic syndrome in paediatric patients caused by the disease,' she said. 'This is a disease for which there are no treatments available anywhere in the world and 60% of those who do get a transplant get reoccurring disease in the transplanted kidney. 'This is a very poor prognosis and in particular for children who will live with life-long dialysis.' Dimerix's phase III study of lead drug DMX-200 in FSGS is titled Angiotensin II Type 1 Receptor (AT1R) & Chemokine Receptor 2 (CCR2) Targets for Inflammatory Nephrosis – or ACTION3 for short. The second interim analysis of its ACTION3 trial is forecast in around mid-CY25. DMX-200 has been granted ODD in the US and Europe, as well as the equivalent Innovative Licensing and Access Pathway (ILAP) designation in the UK. Mesoblast gains world-first approval for treatment Mesoblast (ASX:MSB) has developed Ryoncil (remestemcel-L) for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in paediatric patients two months and older, which is the first FDA approved mesenchymal stromal cell (MSC) therapy. Resulting from bone marrow transplants, GvHD is a life-threatening condition with high mortality rates. Mesoblast said in the US about 10,000 patients undergo a bone marrow transplant annually with donated tissue, 1500 of whom are children. About half of them will develop GvHD and 50% of these won't respond to steroids. Ryoncil applies to patients resistant to steroids, the standard-of-care treatment. Mesoblast is also targeting the children's disorder hypoplastic left heart syndrome, for which it has rare paediatric disease designation from the US FDA for its allogeneic cell therapy Revascor.
Telegraph
20-05-2025
- Health
- Telegraph
The British Blood Scandal, review: a story to fill you with rage
It's a dull cliché to compare every national scandal to Mr Bates vs the Post Office, but The British Blood Scandal: Poisoned at School (ITV1) had enough jaw-dropping moments for a Gwyneth Hughes or a Jack Thorne to spin out half a dozen establishment-shaming mini-series. Unlike when Mr Bates aired, however, the campaigning is largely done, last year's Infected Blood Inquiry having found catastrophic failings and the government announcing a £11.8bn compensation fund for victims. That doesn't stop this documentary from filling you with the same eye-pricking, skin-tingling rage that the Horizon scandal did. The story it tells is unfathomable. The Lord Mayor Treloar's College in Hampshire, set up in 1907, is a specialist boarding school for children with various disabilities. In the 1970s and 1980s it was a 'haven' for boys with haemophilia, with the 24-hour care allowing them to treat bleeds instantly and giving the pupils the chance at a normal life. In an idyllic countryside setting, the boys could sail, fish, ride bikes and play football. Better still, they had access to what they were told was a revolutionary 'wonder' treatment, Factor Eight. There were gifts for taking part in the treatment, punishments if you missed it. 'It was really pushed, everyone was on it,' said Richard, one of four former Treloar's boys – all of whom contracted hepatitis, HIV or both at the school – who made the backbone of this devastating film. The plasma – much of which was imported from the US, where private companies would often recruit from the homeless – was infected. Doctors at Treloar's, whose haemophilia unit was run by the NHS from 1978, were aware of the high risk of infection with the imported plasma. Not only did they not change anything about the treatment, they doubled down, using the boys for research projects. 'Guinea pigs. Lab rats. They exploited that opportunity to do research on children,' said Steve, another former pupil, barely able to get that final word out of his mouth. 'I'll never forgive them for that.' The truly sickening thing is the implication that at every level – pharmaceutical, government, the school – the risk of infection was known. All four of the courageous men here – Richard, Steve, Ade and Gary – told their stories with dignity, but they never stopped reminding us what a privilege it was that they could do so at all. 'We used to come back to the school for reunions every year,' said Ade, 'and every year we'd lose two or three guys.' Of the 122 haemophiliac boys to have gone to Treloar's, around 90 have died. There were details in Tom Whitaker's film that hit like a hammer. Ade remembers being invited to one of his doctor's houses to play with the doctor's son, and finding the man crying. 'We f--ked up,' he said, 'we f--ked up, boys.' Another pupil, Neil, was diagnosed at the school as HIV+ when he was 12. His mother, Angela, was not told for three years. 'I sent him to his death,' she said. 'He was a good boy.' It was 1983 when the Mail on Sunday ran the front-page headline, 'Hospitals using killer blood'. The teachers at Treloar's hid the newspapers that day. It wasn't until 2024 when Richard, Ade, Steve and Gary got to give their evidence to an inquiry, now grey-haired men in their 50s, most of their mates dead.
