Systemic onset juvenile chronic arthritis

Pictured: Fresenius Kabi's office in Toronto iStock/JHVEPhoto Another biosimilar approval from the FDA was handed down Thursday, as the regulator approved Fresenius Kabi’s Tyenne biosimilar for Genentech’s Acterma (tocilizumab). According to Fresenius Kabi, Tyenne is the first biosimilar to Acterma which has both IV and subcutaneous formulations approved by the FDA. However, as part of a patent settlement with Genentech, the German pharma has a license to market its biosimilar products in the U.S., which will start on confidential license dates. The biosimilar is now approved to treat inflammatory and immune diseases such as rheumatoid arthritis, polyarticular juvenile idiopathic arthritis, giant cell arteritis and systemic juvenile idiopathic arthritis. “The FDA’s approval of our tocilizumab biosimilar is a breakthrough in bringing high-quality, affordable, and accessible autoimmune treatment options to patients and healthcare providers,” Michael Schönhofen, Fresenius Kabi president of biopharma, said in a statement. “We are expanding our biosimilars portfolio for immunology and oncology-related diseases, and we are committed to improving the quality of patients’ lives and lightening the burden on healthcare systems around the world.” The Tyenne biosimilar has already been launched in 10 countries but Fresenius Kabi plans to launch it in additional nations this year and in 2025. Acterma pulled in over CHF 1.2 billion ($1.39 billion) in sales in the U.S. in fiscal year 2023 and CHF 2.6 billion ($2.9 billion) in global sales. “Tyenne met the rigorous FDA requirements for biosimilarity and is another treatment option for patients and their healthcare providers,” Fabrice Romanet, senior vice president of innovation and development at Fresenius Kabi Biopharma, said in a statement. Tyenne is the third FDA-approved biosimilar for Fresenius Kabi as Stimufend, a Neulastat biosimilar, was approved in February 2023 and the Humira biosimilar Idacio was approved in July 2023. The Tyenne approval comes as the FDA gave its approval to two of Sandoz’s biosimilars this week. The regulator approved Sandoz’s Jubbonti, which will go up against Amgen’s Prolia in treating osteoporotic men and postmenopausal women at risk of fracture. The other approved biosimilar is Wyost, similar to Amgen’s Xgeva, which is intended to prevent skeletal-related events in patients with multiple myeloma and solid tumors with bone metastases. Tyler Patchen is a staff writer at BioSpace. You can reach him at tyler.patchen@biospace.com. Follow him on LinkedIn.

Detailed results from the NIH-sponsored Phase III OUtMATCH study showed treatment with Xolair increased the amount of peanuts, tree nuts, egg, milk and wheat that people as young as 1 year consumed without an allergic reaction The U.S. FDA recently approved Xolair as the first and only medicine for children and adults with one or more food allergies Allergic reactions can be life-threatening and it is estimated that food-related anaphylaxis results in 30,000 medical events treated in emergency rooms in the U.S. each year1-3 Basel, 25 February 2024 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today data from Stage 1 of the National Institutes of Health (NIH)-sponsored pivotal Phase III OUtMATCH study evaluating the efficacy and safety of Xolair® (omalizumab) in patients allergic to peanuts and at least two other common foods were published in the New England Journal of Medicine (NEJM) and featured in a late-breaking symposium at the 2024 American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting. The study showed treatment with Xolair increased the amount of peanuts, milk, egg, wheat and tree nuts (cashew, hazelnut and walnut) that it took to cause moderate to severe allergic reactions in multi-food allergic people as young as 1 year. Safety findings were consistent with the known safety profile of Xolair across its approved indications and in previous clinical trials.4 The U.S. Food and Drug Administration (FDA) recently approved the expanded use of Xolair in children and adults with IgE-mediated food allergies based on the OUtMATCH data. “Over the past 35 years, I have seen how debilitating food allergies can be for patients and their loved ones, as they are consumed by the fear of accidental exposure,” said Robert Wood, M.D., director of the Eudowood Division of Allergy, Immunology and Rheumatology at Johns Hopkins Children's Center, and principal investigator of the OUtMATCH study. “While allergic reactions to exposures are common and often severe, there have been limited treatment advancements for food allergy. The results of the OUtMATCH study showed that anti-IgE therapy could significantly reduce the occurrence of allergic reactions across multiple foods in the event of an accidental exposure.” “Living with food allergies has a profound impact on patients and their families, causing significant stress and requiring constant vigilance,” said R. Sharon Chinthrajah, M.D., associate professor of medicine, Stanford School of Medicine, Sean N. Parker Center for Allergy and Asthma Research and OUtMATCH co-lead study investigator. “The OUtMATCH study demonstrated that anti-IgE therapy increased most patients’ threshold for an allergic reaction. This presents an important new treatment option for patients and families in its potential to reduce the risk of allergic reactions from accidental exposures they may face in day-to-day life.” One hundred eighty patients ages 1 to 55 years old entered Stage 1 of the OUtMATCH study unable to tolerate up to 100 mg of peanut protein (equivalent to about one third of a peanut), and up to 300 mg of at least two other food proteins among milk, egg, cashew, walnut, hazelnut and wheat. After 16 to 20 weeks of treatment with Xolair or placebo, each participant completed four separate blinded food challenges (including a placebo ingredient) to assess patients’ ability to consume a single dose of at least 600 mg of peanut protein (primary endpoint), and a single dose of at least 1,000 mg of milk, egg, wheat, cashew, hazelnut or walnut protein (secondary endpoints) without experiencing moderate to severe allergic reactions. Results showed that, compared to placebo, a statistically significant (p<0.001) higher proportion of patients receiving Xolair were able to consume at least 600 mg of peanut protein and at least 1,000 mg of milk, egg and cashew protein without experiencing moderate to severe allergic reactions. Additionally, compared to placebo, a higher proportion of patients receiving Xolair were also able to consume at least 1,000 mg of walnut, hazelnut and wheat protein without experiencing moderate to severe allergic reactions. Detailed results are included in the table below. OUtMATCH Primary and Secondary Endpoints Results in Paediatric Population (n=177)Percentage of Patients Successfully Consuming Predefined Threshold Dose of Seven Foods Approximate Equivalents5 Xolair Treated Patients Placebo Treated Patients Number of Patients Primary Endpoint Peanut (≥600 mg) 95% CI for difference: 47 to 70; p<0.001 2.5 peanuts or 1/2 teaspoon of regular peanut butter 67% 7% 177 Key Secondary Endpoints Cashew (≥1,000 mg) 95% CI for difference: 19 to 52; p<0.001 3.5 cashews 41% 3% 99 Egg (≥1,000 mg) 95% CI for difference: 46 to 79; p<0.001 1/4 of an egg 67% 0% 71 Milk (≥1,000 mg) 95% CI for difference: 30 to 74; p<0.001 2 tablespoons of 1% milk 66% 10% 62 Additional Secondary Endpoints Walnut (≥1,000 mg) 95% CI for difference: 27 to 68 3 walnut halves 64% 13% 78 Hazelnut (≥1,000 mg) 95% CI for difference: -2 to 78 4.5 hazelnuts 65% 14% 24 Wheat (≥1,000 mg) 95% CI for difference: 13 to 88 3.5 saltine crackers 75% 13% 20 Additionally, out of three adult OUtMATCH participants, two completed Stage 1 of the study; the adult who received Xolair met the primary endpoint compared to the placebo-enrolled adult patient. Adverse event rates in the study were similar between Xolair and placebo. The most common adverse event in Xolair-treated children and adolescents was injection site reaction (9%). “The Phase III OUtMATCH results represent a major advance and reinforce our commitment to addressing critical gaps in care for conditions like food allergy,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “Now recently FDA-approved, Xolair provides a new way to help manage food allergies for children and adults who live with the constant fear of life-threatening allergic reactions.” While efficacy cannot be established from uncontrolled, open-label studies, for 38 children who continued Xolair for 24-28 weeks in an open-label extension, the percentage of patients who were able to consume 600 mg or more of peanut protein and 1,000 mg or more of egg, milk and/or cashew protein without moderate to severe dose-limiting symptoms was maintained. About 3.4 million children and 13.6 million adults in the U.S. will have been diagnosed with IgE-mediated food allergies, based on estimates for 2024.1,2 Food allergy prevalence has been on the rise for the past 20 years.6 There are 160 different foods that cause IgE-mediated food allergy.7 Allergic reactions can range from mild to moderate, including hives and swelling, to severe and life-threatening, such as anaphylaxis.3 More than 40% of children and more than half of adults with food allergies have experienced a severe reaction at least once, and it is estimated that food-related anaphylaxis results in 30,000 medical events treated in emergency rooms in the U.S. each year.1-3 On 16 February, 2024, the FDA approved Xolair for the reduction of allergic reactions, including anaphylaxis, that may occur with accidental exposure to one or more foods in adult and paediatric patients aged 1 year and older with IgE-mediated food allergy. People taking Xolair for food allergies should continue to avoid all foods they are allergic to (commonly referred to as “food allergen avoidance”). Xolair should not be used for the emergency treatment of any allergic reactions, including anaphylaxis. Xolair is the first and only FDA-approved medicine to reduce allergic reactions in people with one or more food allergies. In the U.S., Genentech, a member of the Roche Group, and Novartis Pharmaceuticals Corporation work together to develop and co-promote Xolair. About the OUtMATCH StudyThe Omalizumab as Monotherapy and as Adjunct Therapy to Multi-Allergen Oral Immunotherapy in Food Allergic Children and Adults (OUtMATCH; NCT03881696) study is an NIH-sponsored, three-stage, multicentre, randomised, double-blind, placebo-controlled study evaluating Xolair safety and efficacy in patients aged 1 to 55 years who are allergic to peanuts and at least two other common foods. Stage 1 included 180 patients (177 children and adolescents; 3 adults) who were randomly assigned to receive placebo or Xolair injections either every two weeks or every four weeks for 16 to 20 weeks. The Xolair dose and dosing interval were determined by total serum IgE level and body weight at baseline. After 16 to 20 weeks of treatment with Xolair or placebo, each participant completed four separate blinded food challenges where they were given gradually increasing amounts of peanut protein, two other food proteins they were allergic to, and a placebo ingredient. The food challenges were conducted in a carefully controlled setting with investigators looking for signs and symptoms of allergic reaction to assess patients’ ability to consume a single dose of at least 600 mg of peanut protein (primary endpoint), and a single dose of at least 1,000 mg of milk, egg, wheat, cashew, hazelnut or walnut protein (secondary endpoints) without experiencing dose-limiting symptoms, which were defined as moderate to severe allergic reactions, including skin, respiratory or gastrointestinal symptoms. The OUtMATCH study is being sponsored and funded by the National Institute of Allergy and Infectious Diseases (NIAID), part of the NIH, and conducted by the NIAID-funded Consortium for Food Allergy Research (CoFAR) at 10 clinical sites across the U.S. led by Johns Hopkins Children's Center and co-led by Stanford School of Medicine. The study is also supported by Genentech and Novartis Pharmaceuticals Corporation. About Xolair Xolair is the only approved antibody designed to target and block immunoglobulin E (IgE). By reducing free IgE, down-regulating high-affinity IgE receptors and limiting mast cell degranulation, Xolair minimises the release of mediators throughout the allergic inflammatory cascade. About Roche in Immunology The Roche Group’s immunology medicines include: Actemra®/RoActemra® (tocilizumab) for rheumatoid arthritis, polyarticular juvenile idiopathic arthritis (pJIA), systemic juvenile idiopathic arthritis (sJIA) and giant cell arteritis (GCA) and for the treatment of severe or life-threatening chimeric antigen receptor (CAR) T cell-induced cytokine release syndrome (CRS); Rituxan®/MabThera® (rituximab) for rheumatoid arthritis granulomatosis with polyangiitis and microscopic polyangiitis and for pemphigus vulgaris (PV); Xolair® (omalizumab) for allergic asthma and chronic idiopathic urticaria (CIU); Pulmozyme® (dornase alfa) for cystic fibrosis; and Esbriet® (pirfenidone) for idiopathic pulmonary fibrosis (IPF). Roche has more than 15 investigational medicines in clinical development for immunological diseases that include asthma, autoimmune diseases, rheumatoid arthritis, ulcerative colitis and Crohn's disease. About Roche Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world’s largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice. In recognising our endeavour to pursue a long-term perspective in all we do, Roche has been named one of the most sustainable companies in the pharmaceuticals industry by the Dow Jones Sustainability Indices for the fifteenth consecutive year. This distinction also reflects our efforts to improve access to healthcare together with local partners in every country we work. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit www.roche.com. All trademarks used or mentioned in this release are protected by law. References[1] Gupta RS, Warren CM, Smith BM, et al. Prevalence and Severity of Food Allergies Among US Adults. JAMA Netw Open. 2019;2(1):e185630. doi:10.1001/jamanetworkopen.2018.5630.[2] Gupta RS, Warren CM, Smith BM, et al. The Public Health Impact of Parent-Reported Childhood Food Allergies in the United States. Pediatrics. 2018;142(6):e20181235. doi:10.1542/peds.2018-1235.[3] USDA Food Safety and Inspection Service. Food Allergies. https://www.fsis.usda.gov/food-safety/safe-food-handling-and-preparation/food-safety-basics/food-allergies. Accessed November 2023.[4] Xolair (omalizumab) Prescribing Information. Genentech, Inc. and Novartis Pharmaceuticals Corporation 2024.[5] Groetch M, Mudd K, Woch M, et al. Retail Food Equivalents for Post-Oral Immunotherapy Dosing in the Omalizumab as Monotherapy and as Adjunct Therapy to Multi-Allergen Oral Immunotherapy in Food-Allergic Children and Adults (OUtMATCH) Clinical Trial. J Allergy Clin Immunol Pract. 2023;11(2):572-580.e2. doi:10.1016/j.jaip.2022.10.022.[6] Benedé S, Blázquez AB, Chiang D, Tordesillas L, Berin MC. The Rise of Food Allergy: Environmental Factors and Emerging Treatments. EBioMedicine. 2016;7:27-34.[7] US FDA. Food Allergies. https://www.fda.gov/food/food-labeling-nutrition/food-allergies. Accessed February 2024. Roche Global Media RelationsPhone: +41 61 688 8888 / e-mail: media.relations@roche.com Hans Trees, PhDPhone: +41 79 407 72 58 Nathalie AltermattPhone: +41 79 771 05 25 Simon GoldsboroughPhone: +44 797 32 72 915 Karsten KleinePhone: +41 79 461 86 83 Nina MählitzPhone: +41 79 327 54 74 Kirti PandeyPhone: +49 172 6367262 Dr. Rebekka SchnellPhone: +41 79 205 27 03 Sileia UrechPhone: +41 79 935 81 48 Roche Investor Relations Dr. Bruno Eschli Phone: +41 61 68-75284 e-mail: bruno.eschli@roche.com Dr. Sabine BorngräberPhone: +41 61 68-88027 e-mail: sabine.borngraeber@roche.com Dr. Birgit MasjostPhone: +41 61 68-84814e-mail: birgit.masjost@roche.com Investor Relations North America Loren Kalm Phone: +1 650 225 3217 e-mail: kalm.loren@gene.com Attachment 25022024_MR_Xolair_NEJM_en

The Biologics License Application for CT-P47 was based on Phase III data comparing CT-P47 to the reference product ACTEMRA® (tocilizumab) JERSEY CITY, N.J., Jan. 28, 2024 /PRNewswire/ -- Celltrion USA today announced the submission of Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for CT-P47, a biosimilar candidate of the reference product ACTEMRA® (tocilizumab)[1]. The BLA submission was based on data from the global Phase III clinical trial designed to evaluate the efficacy, pharmacokinetics, safety, and immunogenicity of CT-P47 compared to the reference product ACTEMRA® in patients with moderate to severe active rheumatoid arthritis with inadequate response to methotrexate up to Week 52. "The submission of CT-P47 for review is an important step toward providing patients with rheumatoid arthritis a more accessible avenue to treatment for conditions that present such a significant disease burden," said Thomas Nusbickel, Chief Commercial Officer at Celltrion USA. "We plan to lead the market by establishing a diverse product lineup in the autoimmune disease market in the U.S. We will continue to actively cooperate with the FDA's review in an effort to bring this new treatment option to people living with rheumatoid arthritis as soon as possible." ACTEMRA® is indicated for several indications, including moderate to severe rheumatoid arthritis in adults as well as juvenile idiopathic polyarthritis and systemic juvenile idiopathic arthritis. CT-P47, containing the active ingredient tocilizumab, is a recombinant humanized monoclonal antibody that acts as an interleukin 6 (IL-6) receptor antagonist. Celltrion is seeking approval for CT-P47 in both intravenous and subcutaneous routes of administration. About Celltrion USA Celltrion USA is Celltrion's U.S. subsidiary established in 2018. Headquartered in New Jersey, Celltrion USA is committed to expanding access to innovative biologics to improve care for U.S. patients. Celltrion USA will continue to leverage Celltrion's unique heritage in biotechnology, supply chain excellence, and best-in-class sales capabilities to improve access to high-quality biopharmaceuticals for U.S. patients. Celltrion endeavors to offer high-quality, cost-effective solutions through an extensive global network that spans more than 110 different countries. For more information, please visit: . 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Although forward-looking statements contained in this presentation are based upon what management of Celltrion believes are reasonable assumptions, there can be no assurance that forward-looking statements will prove to be accurate, as actual results and future events could differ materially from those anticipated in such statements. Celltrion undertakes no obligation to update forward-looking statements if circumstances or management's estimates or opinions should change except as required by applicable securities laws. The reader is cautioned not to place undue reliance on forward-looking statements. References SOURCE Celltrion USA