Kiniksa Pharmaceuticals’ Post

Have you read 'The impact of RARE disease on siblings experience' impact report? Supported by Alexion Pharmaceuticals, Inc. and created by RARE Revolution Magazine. The report shares the personal insights of young people living with rare disease, how it's embedded into the fabric of their family life, its impact on their education and decision-making. Read the full report at https://lnkd.in/eGTfXCyk

The rarest day of the year! #rarediseaseday2024 Orphan diseases are rare – yet, when their numbers are combined, we see that one in 12 persons in the world is a sufferer. That is a big job for pharmaceutical companies to take on. In this video, we explore the challenges and the available support – and mark World Rare Disease Day. With Ellinor Lindholm and Lovisa Rosenquist. #raredisease #rarediseaseday #arexadvisor #regulatoryaffairs

#Bronchiectasis is a chronic condition affecting approx. 370,000 adults in the US, where the lung’s bronchi become permanently damaged. On #WorldBronchiectasisDay, we are raising awareness for non-cystic fibrosis bronchiectasis (NCFB), a disease with no FDA-approved drugs specifically for NCFB. Verona’s investigational drug #ensifentrine is currently in Phase 2 clinical studies for the treatment of NCFB. Learn more about this focus area: https://bit.ly/3z3gVvX

Sobi - Swedish Orphan Biovitrum AB (publ) gets EU okay for long-acting haemophilia A drug: The European Commission has approved Sobi’s long-acting factor VIII replacement therapy #Altuvoct for the treatment of #haemophilia A in the #EU, with a broad label that spans all ages and any disease severity. Altuvoct (efanesoctocog alfa) is dosed by injection once a week and is a successor to Sobi’s long-established haemophilia A therapy Eloctate (efmoroctocog alfa) – sold by partner Sanofi – which has to be dosed every four days and sometimes even more frequently. The new drug was also developed in partnership with Sanofi and was approved in the US last year as Altuviio. https://lnkd.in/eQAB_hG6

What pharma news hit the headlines in July? Find out in our latest Spotlight, where we highlight a landmark approval in chronic obstructive pulmonary disease for Sanofi and Regeneron, and more. Read it today: https://hubs.la/Q02KcZ1m0

Global #IrritableBowelDisease Market  The market was valued at USD 21.45 Billion in 2022 which is expected to reach USD 27.16 Billion in 2029. The increasing incidence and prevalence of #crohnsdisease, and #ulcerativecolitis are major drivers for the growth of the Market. Inflammatory Bowel Disease can occur at any age, although the disease is most common in #adolescents and #adults between the ages of 15 and 35. #Diet and #Stress do not cause #inflammatoryboweldisease but they can worsen the conditions. #northamericamarket holds the largest Inflammatory Bowel Disease Treatment market. In 2021, North America contributed to around 40% of the global revenue. Reistone Biopharma Co. Ltd., a Chinese biopharmaceutical company in the clinical stages, reported positive findings from a phase II clinical trial that assessed the safety and effectiveness of its drug candidate, SHR0302, an investigational selective Janus kinase type 1 (JAK1) inhibitor, for the treatment of moderate-to-severe ulcerative colitis. The key companies operating in the Inflammatory Bowel Disease Treatment Market are AbbVie Inc., Biogen, Johnson & Johnson Services, Inc., Amgen Inc., UCB S.A., Novartis AG, Takeda Pharmaceutical Company Limited, Merck Group & Co., Inc., Pfizer Inc., and Eli Lilly and Company. Inflammatory Bowel Disease Treatment Market by Type: Crohn’s Disease Ulcerative Colitis Inflammatory Bowel Disease Treatment Market by Drug Case: #Amino #salicylates #Corticosteroids TNF inhibitors IL inhibitors #Antiintegrin JAK #inhibitors The Global Irritable Bowel Disease market showcased growth at a CAGR of 2.60% during 2019-2022. Request a FREE sample of this report here; https://lnkd.in/dauZahQM

#Sandoz receives European Commission approval for #Wyost® and #Jubbonti®, the first and only #biosimilars of #denosumab in #Europe 🚀Sandoz, the global leader in generic and biosimilar medicines, announced that the European Commission (EC) has granted marketing authorization for Wyost® (denosumab) and Jubbonti® (denosumab), the first and only biosimilar versions of reference medicines Xgeva® and Prolia® in Europe. 🎯Wyost® (denosumab) and Jubbonti® (denosumab) approved by EC for all indications of denosumab reference medicines Xgeva® and Prolia®. EC approval based on robust development program confirming that biosimilar matches reference medicine in terms of safety, efficacy and quality. Approved for treatment of #cancerrelatedbone disease and #osteoporosis respectively.

🔍 Biogen's Bold Move: Aduhelm Out, Leqembi In! Unlocking the Future of Alzheimer's Therapies Biogen unveils a game-changing strategy, bidding farewell to Aduhelm and placing the spotlight on Leqembi and a dynamic pipeline. This strategic pivot marks a pivotal moment, emphasizing agility and innovation in addressing Alzheimer's challenges. 🚀 Key Moves: Strategic Reprioritization: Biogen's strategic shift waves goodbye to the Aduhelm era, embracing a forward-looking vision. CEO Chris Viehbacher's leadership underscores a commitment to adaptability and sustainable growth. Leqembi Takes Center Stage: With Leqembi securing full FDA approval, Biogen capitalizes on its potential, offering a beacon of regulatory clarity compared to Aduhelm. The move signals a strategic leap toward a more promising Alzheimer's treatment landscape. Accelerating Pipeline Excellence: Biogen propels its vision by fast-tracking the development of key pipeline medications—BIIB080 and BIIB113. The renewed focus on neuroscience innovation echoes a commitment to advancing the frontier of neurological therapeutics. 🌐 The Big Picture: Biogen's strategic shake-up sets the stage for a transformative chapter in Alzheimer's research. This bold move aligns with the industry's evolution, embracing change and fostering resilience for the next era of pharmaceutical breakthroughs. #Biogen #Leqembi #AlzheimersInnovation #PharmaRevolution 🚀💡 1. Sagonowsky E. Biogen abandons Aduhelm efforts, focuses on Eisai-partnered Leqembi and pipeline drugs. Fierce Pharma. 

Incorporating Rare Disease Patient Outcomes into Value Assessment The health outcomes that matter most to rare disease patients can be difficult to capture in a clinical endpoint, and even more challenging to quantify as part of a health technology assessment (HTA). But just because those patient inputs are difficult to measure doesn’t mean we shouldn’t try – it just means we need to bring smart stakeholders together to figure out a workable approach. That was the key point of the Innovation and Value Initiative (IVI) 5th Annual Methods Summit in Washington, DC, where I was pleased to be part of a panel discussion, “Rare Disease, Unique Needs: Adapting for True Patient Impact.” During the panel as well as throughout the Summit, we focused on the need to better incorporate patient and societal perspectives and health equity factors into HTA, which is frequently used by health care payers to determine the value of a treatment. Our panel, which included Annie Kennedy of the EveryLife Foundation, Dionne L. Stalling of Rare and Black, and Ilisa (Halpern) Paul of Venable, who moderated the conversation, agreed about the importance of evaluating rare disease treatments differently than those for broader populations and we need to ensure othat the views of the rare disease community are considered and better integrated into HTA. Jason Spangler Richard Chapman Adrian Kielhorn Andrea Hofelich Tamar Thompson

#RareDiseaseDay is coming up fast, so we are sharing our latest whitepaper/guide --> From Rare to Aware: Pharma's Role in Rare Disease Patient Activation. Grab a copy below :) #LucidQuest