Eftrenonacog alfa

STOCKHOLM, April 23, 2025 /PRNewswire/ -- Sobi® (STO: SOBI) presents clinical data aimed at updating the global haemophilia community attending this year's WFH 2025 Comprehensive Care Summit in Dubai from the 23 – 25 April. New and updated outcomes and analyses will be presented from the XTEND phase 3clinical program testing the effectiveness of Altuvoct® (efanesoctocog alfa) treatment for haemophilia A, including updates on patients' joint health and surgical outcomes in patients. "At WFH 2025 CCS, Sobi will present highlights from the XTEND phase 3 program and discuss the new treatment paradigm with FVIII levels in the non-haemophilia range. Sharing these clinical outcomes with the wider haemophilia community will help to ensure the latest treatment approaches are widely known and understood," said Lydia Abad-Franch, MD, MBA, Head of Research, Development, and Medical Affairs (RDMA), and Chief Medical Officer at Sobi. Key data to be presented at WFH 2025 Comprehensive Care Summit, Dubai, UAE Altuvoct® (efanesoctocog alfa) About efanesoctocog alfa Efanesoctocog alfa [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein] (formerly BIVV001) is a novel and significant part of the week factor VIII therapy. It has the potential to deliver near-normal factor activity levels for significant parts of the week, improving bleed protection in a once-weekly dose for people with haemophilia A. Efanesoctocog alfa builds on the innovative Fc fusion technology by adding a region of von Willebrand factor and XTEN® polypeptides to extend its time in circulation. It is the only therapy that has been shown to break through the von Willebrand factor ceiling, which imposes a half-life limitation on current factor VIII therapies. It was approved as ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl] by Sanofi in the US in February 2023. About the Sobi and Sanofi 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, referred to as ALTUVIIIO™ in the USA and ALTOVUCT in Europe. An investigational factor VIII therapy with the potential to provide high sustained factor activity levels with once-weekly dosing for people with haemophilia A. 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. Sobi® Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Providing sustainable access to innovative medicines in the areas of haematology, immunology and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East and Asia. In 2022, revenue amounted to SEK 18.8 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com, LinkedIn and YouTube. Contact 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: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

The FDA on Friday said it approved a new RNAi-based hemophilia treatment from Sanofi that the company licensed from Alnylam. Patients with hemophilia A or B will now be able to take Qfitlia (pronounced cue-FIT-lee-uh), whose scientific name is fitusiran, to treat their disease. A Sanofi spokesperson said the average cost of Qfitlia for a majority of patients will be $642,000 per year. The drug will come with a boxed warning for thrombotic events and gallbladder disease, as some patients needed their gallbladders removed, according to an agency press release . There are two major subtypes of hemophilia, but most hemophilia drugs on the market only treat one, by replacing the missing clotting proteins that cause the condition’s hallmark bleeding. Patients with hemophilia A are missing Factor VIII, while those with hemophilia B don’t properly produce Factor IX. Qfitlia, on the other hand, is designed to lower a coagulation-blocking protein called antithrombin and treat hemophilia A or B prophylactically, said Craig Benson, who steered fitusiran development at Sanofi. It can also be used with or without other inhibitors, helpful for some patients whose immune systems neutralize the factor proteins, he added. “They’ve been stuck using other therapies that require very frequent injections, they don’t work quite as well,” Benson said. “So fitusiran is really the first that can treat all forms of hemophilia.” Two other types of hemophilia, hemophilia C and acquired hemophilia, are much rarer. The FDA’s indication is limited to hemophilia A and B regardless of inhibitor usage, and Sanofi did not evaluate the other types of patients in its trials. Although Qfitlia is the first RNAi drug approved for hemophilia, several other companies have won recent approvals with next-generation mechanisms. In October, Pfizer’s Hympavzi was approved in patients with either hemophilia A or B, becoming the first anti-tissue factor pathway inhibitor. And in 2022, uniQure secured an approval for Hemgenix, a gene therapy for hemophilia B. Novo Nordisk is also developing a factor VIII mimetic called Mim8, and expects to file for approval this year. The king of the hemophilia market remains Roche’s Hemlibra, which was approved in 2017 for hemophilia A and recorded sales of 4.5 billion Swiss francs ($5.09 billion) last year. Hemlibra is also a Factor VIII mimetic that doesn’t try to replace Factor VIII, but mimics its function. In an investor note earlier this week, TD Cowen analyst Steve Scala said he expected Qfitlia to compete with Hemlibra, though he noted a boxed warning may complicate the speed of its launch. Qfitlia’s development saw multiple pauses after cases of blood clots arose in 2017 and 2020. The approval also builds on Sanofi’s presence in the disease following the 2023 approval of Altuviiio, a Factor VIII inhibitor only indicated for hemophilia A patients. Sanofi got Altuviiio in its $11.6 billion buyout of Bioverativ in 2018, a deal that also included two other hemophilia drugs (Eloctate and Alprolix) that had already been approved. Qfitlia development has been a longer time coming. Sanofi originally partnered with Alnylam in 2014, paying it $700 million for a 12% stake. Then, in 2018, the companies adjusted their collaboration, with Sanofi getting full rights to fitusiran while Alnylam took on development responsibilities for the programs that would become Onpattro and Amvuttra.