Systemic onset juvenile chronic arthritis

STOCKHOLM, Oct. 30, 2024 /PRNewswire/ -- Sobi® (STO: SOBI) today announced that new research showing the effect of emapalumab in patients with macrophage activation syndrome (MAS) in Still's disease, including systemic juvenile idiopathic arthritis (sJIA) and adult-onset still's disease (AOSD) who had an inadequate response to high-dose glucocorticoids (GCs) will be presented at the American College of Rheumatology (ACR) Convergence in Washington D.C. The data were pooled from two open-label, single-arm interventional studies, the phase 3 NI-0501-14 study [EMERALD; NCT05001737] and NI-0501-06 [NCT03311854], including 39 patients with MAS. Key results: Complete response at week 8: At week 8, 21 patients (53.8%) achieved a complete response (95% confidence interval (CI): 37.2–69.9%). Complete response at any time: 33 patients (85%) achieved a complete response at any time during the studies. Glucocorticoid tapering: Weekly mean glucocorticoid doses were reduced by 70.1% after 2 weeks of treatment. By week 8, glucocorticoids were tapered to a clinically meaningful dose of ≤1 mg/kg/day in 28 patients (72%) and to ≤0.5 mg/kg/day in 17 patients (44%). Safety: No new safety concerns were identified during the studies. "MAS in Still's disease is a severe condition characterised by intense hyperinflammation and multiple organ failure. Those affected, including young children, often experience high fevers, liver and spleen enlargement, severe cytopenias, and neurological symptoms," said Lydia Abad-Franch, MD, Head of Research & Development and Medical Affairs and Chief Medical Officer at Sobi. "The results to be presented at ACR indicate that emapalumab could potentially provide a new therapeutic option, offering effective control of MAS symptoms and possibly reducing the need for high-dose glucocorticoids. This represents an important development in the treatment landscape for MAS." Additional key results: Overall response (OR) by week 8: 32 patients (82.4%) achieved an overall response by week 8. OR was observed as early as day 5, with a median time to first OR of 2.3 weeks. Clinical MAS remission: 32 patients (82.1%) achieved investigator-assessed clinical MAS remission at any time , with a median time to clinical remission of 3.3 weeks. Survival rate: 37 patients (94.9%) were alive at week 8. Emapalumab is a monoclonal antibody that neutralises interferon gamma (IFNγ), a key cytokine which contributes to the inflammation and tissue damage seen in MAS. The purpose of these two prospective studies is to assess the efficacy and safety of emapalumab in controlling MAS in patients with Still's disease, including sJIA and AOSD, who had an inadequate response to high-dose glucocorticoids. In May 2024, the US FDA granted emapalumab fast track designation as a potential therapeutic option in patient with MAS. Sobi plans to submit an sBLA for this indication to the FDA in 2024. The data will be presented by Alexei A. Grom, MD, Professor of Paediatrics, Research Director Division of Rheumatology, Cincinnati Children's Hospital Medical Centre, in a session entitled "Efficacy and Safety of emapalumab in children and adults with macrophage activation syndrome (MAS) in Still's disease: Results from a phase 3 study and a pooled analysis of two prospective trials" on Tuesday, 19 November at ACR Convergence. About macrophage activation syndrome (MAS) Macrophage activation syndrome (MAS) is a severe complication of rheumatic diseases, most frequently systemic juvenile idiopathic arthritis (sJIA) – a rare systemic disorder of auto-inflammatory nature with common clinical manifestations such as daily spiking fever, typical transient cutaneous rash, arthritis, lymphadenopathy, hepatosplenomegaly and serositis. MAS is characterised by fever, hepatosplenomegaly, liver dysfunction, cytopenias, coagulation abnormalities and hyperferritinaemia, possibly progressing to multiple organ failure and death. MAS is classified as a secondary form of haemophagocytic lymphohistiocytosis (HLH). About emapalumab Emapalumab (Gamifant®) is an anti-interferon gamma (IFNγ) monoclonal antibody that binds to and neutralises IFNγ. In the USA, emapalumab is indicated for the treatment of adult and paediatric (newborn and older) patients with primary haemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. Primary HLH is a rare syndrome of hyperinflammation that usually occurs within the first year of life and can rapidly become fatal unless diagnosed and treated. The FDA approval is based on data from the phase 2/3 studies (NCT01818492 and NCT02069899). Gamifant is indicated for administration through intravenous infusion over one hour twice per week until haematopoietic stem cell transplantation (HSCT). About Sobi® Sobi® is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable 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, Asia, and Australia. In 2023, revenue amounted to SEK 22.1 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. 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: SOURCE Swedish Orphan Biovitrum AB WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Giuseppe Menin, life sciences and process industry manager at COPA-DATA, highlights how an automation integration layer can streamline data management, ensuring compliance with the ALCOA principles—Attributable, Legible, Contemporaneous, Original, and Accurate. Pharmaceutical companies are under increasing pressure to meet the rising demand for diverse medications. According to IQVIA, global medicine usage is projected to grow by 12% by 2028, driven largely by innovative therapeutics and the availability of more affordable generics and biosimilars. This surge necessitates handling larger volumes of data, underscoring the importance of robust data management systems. The ALCOA principle and its expansion ​ ALCOA, recently expanded to ALCOA+ to include Complete, Consistent, Enduring, and Available, forms the foundation of data integrity standards set by regulatory bodies like the FDA and EMA. Non-compliance can lead to severe repercussions, including warning letters, non-compliance reports, fines, and even criminal prosecution. Despite the awareness of data integrity’s importance, pharmaceutical manufacturers often struggle with accurate and consistent data collection, analysis, and storage. Traditional paper-based processes pose significant risks, including human errors and the potential for tampering. Challenges in data integrity Legacy equipment and the lack of standardized protocols further complicate data integrity efforts. Even with a Manufacturing Execution System (MES), manual data transcriptions, known as "paper-on-glass" practices, remain error-prone. Additionally, older machinery from various vendors often lacks the ability to communicate seamlessly, adding another layer of complexity. googletag.cmd.push(function () { googletag.display('text-ad1'); }); The role of automation integration ​ Automating the integration of data across all production stages is crucial for overcoming these challenges. COPA-DATA’s zenon Automation Integration Layer (AIL) software exemplifies this approach, providing connectivity to machines from multiple vendors, including older equipment. Zenon ensures that data is automatically contextualized and archived, minimizing the risk of data loss or inconsistency. This automation detects incomplete or incorrect entries, enabling real-time data access and immediate issue resolution. Operators can rectify faults during production, significantly reducing time to market and associated costs. Ensuring accurate recipe management ​ In pharmaceutical manufacturing, precise recipe management is critical. Zenon allows operators to select pre-set recipes, eliminating human errors during production setup. The software guides users through each step, ensuring data completeness and adherence to specified tolerances. Towards automated data integrity ​ As pharmaceutical companies expand their product lines, maintaining compliance is essential. Efficient data management through automation not only ensures compliance with data integrity standards but also reduces costs and accelerates time to market. Menin emphasizes that automation integration is key to meeting ALCOA+ requirements. "Automated systems like zenon not only enhance data accuracy but also provide real-time access, ensuring compliance and efficiency in pharmaceutical production," he notes. Tofidence, a biosimilar to Actemra, is approved for treating moderately to severely active rheumatoid arthritis, polyarticular juvenile idiopathic arthritis, and systemic juvenile idiopathic arthritis. This FDA-approved biosimilar offers an affordable alternative, helping to alleviate the financial burden on patients and the healthcare system. Automating data integrity processes is not just about compliance; it’s about improving overall operational efficiency and ensuring that patients receive safe, effective, and high-quality medications.

Tyenne® is the first tocilizumab biosimilar available with both an intravenous and subcutaneous formulation approved by the FDA. The subcutaneous formulation of the tocilizumab biosimilar provides increased access and an affordable treatment option for U.S. patients. Subcutaneous formulation was approved by FDA and is available in a self-administered prefilled syringe and an autoinjector. Third Fresenius Kabi biosimilar in the U.S. and second in its immunology portfolio. LAKE ZURICH, Ill.--(BUSINESS WIRE)-- Fresenius, via its operating company Fresenius Kabi, announced today the immediate availability in the U.S. of its biosimilar Tyenne® (tocilizumab-aazg), in a subcutaneous formulation, which will increase access to affordable and cost-effective treatment options for use in the treatment of chronic autoimmune diseases. This press release features multimedia. View the full release here: Tyenne® from Fresenius Kabi is the first tocilizumab biosimilar approved by the FDA that is available in both an intravenous and subcutaneous formulation. The subcutaneous formulation is now available in a self-administered prefilled syringe and an autoinjector. (Photo: Business Wire) Tyenne is indicated for use in the treatment of chronic autoimmune diseases for certain indications of the reference product, Actemra® (tocilizumab). Fresenius Kabi previously launched Tyenne® in an intravenous (IV) formulation in April of this year. “After successfully launching Tyenne in more than 10 countries, we are on track to continue the momentum in the U.S with the launch of a second formulation,” said Sang-Jin Pak, M.D., President of Biopharma at Fresenius Kabi. “Introducing our subcutaneous formulation demonstrates our continued commitment to providing greater access to more patients living with autoimmune diseases.” Trial data that compared Tyenne® to the reference product, Actemra® showed similar safety and tolerability. Tyenne® is the first tocilizumab biosimilar with an intravenous and subcutaneous formulation approved by the FDA. Tyenne® received FDA approval on March 5, 2024, and an IV formulation was launched in April 2024 for use in the treatment of several inflammatory autoimmune diseases, including rheumatoid arthritis, giant cell arteritis, polyarticular juvenile idiopathic arthritis, and systemic juvenile idiopathic arthritis. To learn more about how Fresenius Kabi provides comprehensive patient support for Tyenne® in the U.S. please click here. About Tyenne®, a Tocilizumab Biosimilar Tyenne® (tocilizumab-aazg), a biosimilar to Actemra® (tocilizumab), is a prescription medicine called an Interleukin-6 (IL-6) receptor antagonist. It was developed by Fresenius Kabi using advanced analytical and manufacturing technologies for use in the treatment of several autoimmune diseases, including rheumatoid arthritis, giant cell arteritis, polyarticular juvenile idiopathic arthritis, and systemic juvenile idiopathic arthritis. Tyenne® includes a Boxed Warning for serious infections leading to hospitalization or death including tuberculosis (TB), bacterial, invasive fungal, viral, and other opportunistic infections that have occurred in patients receiving tocilizumab products. Tyenne® is contraindicated in patients with known hypersensitivity to tocilizumab products. For more information about Tyenne®, please see the important safety information and full prescribing information for the U.S. About Fresenius Kabi Fresenius Kabi is a global healthcare company that specializes in lifesaving medicines and technologies for infusion, transfusion and clinical nutrition. The company’s products and services are used for the therapy and care of critically and chronically ill patients. Its product portfolio comprises a range of highly complex biopharmaceuticals, clinical nutrition, medical technologies, and I.V. generic drugs. Within biopharmaceuticals, Fresenius Kabi offers, among others, biosimilar drugs with a focus on autoimmune diseases and oncology. The company’s clinical nutrition offering includes a wide selection of enteral and parenteral nutrition products. In the segment of medical technologies, its offering includes vital disposables, infusions pumps, apheresis machines, cell therapy devices, and more. Fresenius Kabi puts essential medicines and technologies in the hands of people who help patients and finds the best answers to the challenges they face. Following its strategy “Vision 2026”, which is a key part of the #FutureFresenius program of the Fresenius healthcare group, the company is furthermore committed to increase efficiencies in the therapy and care of patients and improve access to high-quality healthcare around the globe. Fresenius Kabi aspires to be leader globally in its product segments – all for the benefit of patients, its customers, and its stakeholders. Fresenius SE & Co. KGaA (Frankfurt/Xetra: FRE) is a global healthcare company headquartered in Bad Homburg v. d. Höhe, Germany. In the 2023 fiscal year, Fresenius generated €22.3 billion in annual revenue with its more than 190,000 employees. Fresenius offers solutions to the social challenges posed by a growing and ageing population and the resulting need for affordable, high-quality healthcare. The Fresenius Group comprises the operating companies Fresenius Kabi and Fresenius Helios as well as the investment companies Fresenius Vamed and Fresenius Medical Care. With 140 hospitals and countless outpatient facilities, Fresenius Helios is the leading private hospital operator in Germany and Spain, treating around 26 million patients every year. Fresenius Kabi’s product portfolio includes a range of highly complex biopharmaceuticals, clinical nutrition, medical technology, and generic intravenous drugs. Fresenius was established in 1912 by the Frankfurt pharmacist Dr. Eduard Fresenius. After his death, Else Kröner took over management of the company in 1952. She laid the foundations for a global enterprise that today pursues the goal of improving people’s health. The largest shareholder is the non-profit Else Kröner-Fresenius Foundation, which is dedicated to advancing medical research and supporting humanitarian projects. For more information visit the company website at . Follow us on social media: . This release contains forward-looking statements that are subject to various risks and uncertainties. Future results could differ materially from those described in these forward-looking statements due to certain factors, e.g., changes in business, economic and competitive conditions, regulatory reforms, results of clinical trials, foreign exchange rate fluctuations, uncertainties in litigation or investigative proceedings, the availability of financing and unforeseen impacts of international conflicts. Fresenius does not undertake any responsibility to update the forward-looking statements in this release. Fresenius SE & Co. KGaA Registered Office: Bad Homburg, Germany / Commercial Register: Amtsgericht Bad Homburg, HRB 11852 Chairman of the Supervisory Board: Wolfgang Kirsch General Partner: Fresenius Management SE Registered Office: Bad Homburg, Germany / Commercial Register: Amtsgericht Bad Homburg, HRB 11673 Management Board: Michael Sen (Chairman), Pierluigi Antonelli, Sara Hennicken, Robert Möller, Dr. Michael Moser Chairman of the Supervisory Board: Wolfgang Kirsch Actemra® is a registered trademark of Chugai Seiyaku Kabushiki Kaisha Corp., a member of the Roche Group. Tyenne® is a registered trademark of Fresenius Kabi Deutschland GmbH.