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The International Pandemic Preparedness Secretariat recently released the 100 Days Mission Therapeutics Roadmap, an important new tool to help the world prepare for future catastrophic viral outbreaks. Working to achieve the Roadmap’s three strategic visions is critical: 1️⃣ Ensure sustained funding throughout the drug development lifecycle. 2️⃣ Develop at least two Phase-2 ready therapeutic candidates against each of the identified viral pathogen families. 3️⃣ Develop programmable platforms or technologies capable of speeding up the availability of new or enhanced therapeutics in case of a pandemic — and be able to rapidly and safely apply them to treat “Disease X.” READDI is proud to have helped shape the direction of the Roadmap. READDI CEO James Rosen and READDI Co-founder and Scientific Adviser Dr. Nat Moorman provided virology expertise, timelines and budget projections from their experience planning READDI’s own way forward. READDI is a nonprofit biotechnology company working in the public interest with a singular focus: to make antiviral drugs available to everyone who needs them, when they need them, where they need them in the event of a viral outbreak. Learn more about how the Therapeutic Roadmap’s goals mirror READDI's goals: https://lnkd.in/eCGktp2X #pandemicpreparedness #antivirals #equitablehealthcare #virology

Medicines Australia and its members take a lead role in Australia’s pharmaceutical horizon scanning to keep policy makers informed of innovative new classes of medicines that have the potential to significantly impact the way conditions are treated or the way that medicines are assessed and funded.    In Australia, horizon scanning is maturing with concerted efforts by industry and the Commonwealth to strengthen frameworks and processes so that our system is ready for new technologies ahead of their assessment and approval for use in Australia by the TGA. Last week, many of Australia's leading scientists, researchers, policy makers, regulators and patient groups attended our horizon scanning forum to learn more about cell and gene therapies, new antibiotics, vaccine advances and diagnostics.   The HTA Review has highlighted the importance of robust horizon scanning as an indicator of a world-class health system and the importance of advanced notice for policy makers to be able to plan and prepare the health system for disruptive medical advances. Over the last decade, the arrival of cell and gene therapies and genetic testing has revolutionised the diagnostics and treatment practices of many life-threatening conditions.    But our system to assess and fund these medicines was not prepared, and today patients are still waiting to access these and other new medicines far longer than in other countries.    This cannot continue.     Medicines Australia and its members support the recommendation for a stronger horizon scanning process.     We look forward to co-designing with the Commonwealth, patient groups and other stakeholders, a world-class horizon scanning system that enables greater flexibility and informed decision-making to bring breakthrough medical technologies to Australia faster. Find out more about >> https://lnkd.in/gcczfeey #StrongerPBS

🚀 Exciting Developments in the Pharma Industry! 🌟 🔬 The FDA has given the green light to several innovative treatments, including: - Ohtuvayre (ensifentrine) for COPD maintenance. - Tepylute (thiotepa) targeting breast and ovarian cancers. - Capvaxive (pneumococcal 21-valent conjugate vaccine) for pneumococcal disease prevention. - Rytelo (imetelstat) for myelodysplastic syndrome. - Iqirvo (elafibranor for primary biliary cholangitis. 💊 "Sun Pharma" has achieved FDA approval for its new alopecia areata drug, Leqselvi, promising new hope for those affected by this condition. 🌍 Pfizer's gene therapy for hemophilia B has received approval from the European Commission, marking a significant milestone in treatment options available in the EU. 🏥 Despite strong sales reports from major companies like AstraZeneca, Roche, and Sanofi, the EU has controversially rejected Eisai and Biogen's Alzheimer's drug, Leqembi, after approvals in the US and other regions. 🤖 Strategic industry moves: - Caribou Biosciences and Roche have announced strategic shifts, including workforce adjustments. - Vertex Pharmaceuticals has initiated legal action over limits on fertility services for its Casgevy treatment patients. Stay tuned for more updates as the pharma industry continues to innovate and evolve! #Pharma #FDA #Biotech #HealthcareInnovation #MedicalResearch #DrugDevelopment #niper #pharmacy #mpharma #bpharma #Gpat #Niperhyderabad #nipermohali

📌 Vertex's recent acquisition of Alpine has raised our conviction on the potential of the immunoglobulin A Nephropathy (IgAN) market. With ~130K biopsy-confirmed IgAN patients in the USA, ~140K in the EU, ~130K in Japan, and ~5 million in China, it is the most common primary glomerulonephritis globally. 📌 Up to 50% of these patients will develop end-stage renal disease (ESRD) within 10-20 years from diagnosis, requiring dialysis or transplant. ESRD remains a major health economic burden and contributes to morbidity and mortality. 📌 Currently, Calliditas' TARPEYO and Travere Therapeutics' FILSPARI are FDA approved with disease-modifying potential. Several other drugs with distinct mechanisms of action are in late-stage development, with pivotal readouts expected in 2024. These programs include Chinook Therapeutics' atrasentan, Novartis' iptacopan, and Omeros' narsoplimab, among others. 📌 Despite rising competitive intensity, we hold an optimistic outlook on the IgAN market and are closely watching how the safety and efficacy stack up across programs. #kidneydisease hashtag #nephrology hashtag #ckd hashtag #strategy hashtag #marketintelligence hashtag #competitiveintelligence hashtag #eosintelligence hashtag #perspectives hashtag #management hashtag #consulting hashtag #pharmaceuticals hashtag #marketresearch hashtag #marketing hashtag #leadership hashtag #lifesciences hashtag #healthcare hashtag #pipeline hashtag #pharmainnovation hashtag #marketanalysis hashtag #biotech ----------------------------------------------------------------------------------- Ready to leverage the power of intelligence to drive Nephrology/Kidney Disease product development and commercialization strategy? At EOS Intelligence , we offer competitive intelligence monitoring services in key therapeutic areas to make you stay ahead of the curve. Let us know your perspectives & challenges and we will get you covered. Write us at bhabesh.panigrahi@eos-intelligence.com

Everything you missed in #Pharma & #Biotech 🦠🔬 ✅ Approvals AstraZeneca's Fasenra (benralizumab) receives expanded approval for use in children with severe asthma 💸 Deals Vertex Pharmaceuticals acquires Alpine Immune Sciences, Inc. for $4.9 billion, expanding its portfolio of renal disease treatments with the addition of povetacicept Abceutics Inc. acquired by Merck for $208M, enhancing its antibody-drug conjugates with selectivity enhancers Johnson & Johnson to invest $6.6 million in collaboration with Rallybio to develop treatments for fetal and neonatal alloimmune thrombocytopenia Nvelop Therapeutics launches with $100M to advance gene editing delivery work from renowned scientists David Liu and Keith Joung, with a focus on developing novel delivery technologies for gene editing therapies 🎯Late-stage developments MaaT Pharma presents positive 18-month data for MAAT013, showing overall survival advantage in acute graft-versus-host disease (aGVHD) from Early Access Program Marinus Pharma's Phase III trial for ganaxolone in status epilepticus fails to meet early stopping criteria Roche's tafasitamab-cxix (Columvi) demonstrates positive Phase 3 results in diffuse large B-cell lymphoma Roche and Eli Lilly and Company receive FDA breakthrough status for early Alzheimer's disease detection blood test 📈 Access Regeneron faces lawsuit from US Department of Justice over alleged inflated costs due to undisclosed reimbursements for Eylea (aflibercept) CMS proposes new policies to improve access to innovative treatments and address health disparities, including increased reimbursement for new sickle cell disease gene therapies ✳️ Other: The European Parliament adopts a stance on pharmaceutical legislation, emphasizing the need to address drug shortages and antimicrobial resistance 👉 What’s your favorite industry news? Hannah Kurth #pharma #biotech #pulsenews

#CureSickleCellNow #TransfusionDependentBetaThalassemia Today, exa-cel (exagamglogene autotemcel) was approved by the US Food and Drug Administration (FDA) for the treatment of transfusion-dependent β-thalassemia (TDT) in patients 12 years and older. This was an expansion of the original announcement on December 8, 2023 for sickle cell disease. Exa-cel is a CRISPR/Cas9 edit of the patient's stem cells that results in high fetal hemoglobin (HbF or baby hemoglobin). This is a great move forward for this very rare disease (TDT) - I expect that it might be possible to deploy this therapy to all with this disease soon given it's small numbers in the US. However, gene editing for TDT and sickle cell disease have important requirements so it is very important that these patients have access to disease specific expert physicians. Access to care and to expert physicians remains the single most important issue for the new FDA approved therapies for both diseases. https://lnkd.in/eZPhaxr6

Yesterday was #RareDiseaseDay. The annual observance highlights how far the #biopharma industry has come and serves as a wake-up call for how far it must still go to meet the needs of #raredisease patients. Despite recent investment and breakthroughs for #rarediseases, experts told BioSpace that helping a much larger patient population will require cross-sector support and desperately needed funding. We looked at some of the investments to date, as well as the biopharma industry’s promising pipelines and what is required to bring more groundbreaking therapies to patients with unmet medical needs. #Patientadvocacy groups have been critical to the development of the first marketed drugs for certain rare diseases, including progeria and Friedreich's ataxia. We spoke with leaders of these groups formed by families coping with rare diseases, providing case studies on their partnerships with biopharma companies. https://lnkd.in/g7R6hU23 #RareDiseaseDay2024 #genetherapy #genetherapies #duchenne #duchennemusculardystrophy #biopharmaceuticals

🌟 𝐄𝐱𝐜𝐢𝐭𝐢𝐧𝐠 𝐃𝐞𝐯𝐞𝐥𝐨𝐩𝐦𝐞𝐧𝐭𝐬 𝐢𝐧 𝐭𝐡𝐞 𝐃𝐫𝐮𝐠𝐬 𝐟𝐨𝐫 𝐁𝐥𝐚𝐮 𝐒𝐲𝐧𝐝𝐫𝐨𝐦𝐞 𝐌𝐚𝐫𝐤𝐞𝐭! 🚀 𝐂𝐥𝐢𝐜𝐤 𝐇𝐞𝐫𝐞, 𝐓𝐨 𝐆𝐞𝐭 𝐅𝐫𝐞𝐞 𝐒𝐚𝐦𝐩𝐥𝐞 𝐑𝐞𝐩𝐨𝐫𝐭 https://lnkd.in/dqE84zhG 🌟 Key Aspects: Rare Disease Focus: Blau syndrome falls under the category of rare diseases, making the market relatively niche compared to more prevalent conditions. Consequently, drug development efforts may face unique challenges such as limited patient populations for clinical trials and regulatory pathways tailored to rare diseases. Treatment Goals: The primary aim of drug therapies for Blau syndrome is to alleviate symptoms and inflammation associated with the condition. This may involve targeting specific inflammatory pathways or modulating the immune response to reduce organ damage and improve quality of life for affected individuals. Limited Treatment Options: Given the rarity of Blau syndrome, there may be a limited number of approved medications specifically indicated for this condition. Healthcare providers may resort to off-label use of certain drugs or adopt treatment strategies from related inflammatory disorders to manage symptoms in affected individuals. 💡 Innovation and Research: Targeted Therapies: Ongoing research efforts aim to identify novel therapeutic targets and develop targeted therapies that address the underlying inflammatory mechanisms implicated in Blau syndrome. These may include biologic agents, small molecule inhibitors, or immunomodulatory drugs designed to suppress aberrant immune responses and mitigate tissue damage. Precision Medicine: Advances in genetic testing and molecular diagnostics may facilitate the development of personalized treatment approaches tailored to the specific genetic mutations and immunological profiles of individuals with Blau syndrome. Precision medicine strategies could optimize treatment outcomes and minimize adverse effects. 🌍 Global Landscape: The Drugs for Blau Syndrome Market is part of the broader landscape of rare disease therapeutics, with stakeholders including pharmaceutical companies, academic research institutions, patient advocacy groups, and regulatory agencies collaborating to advance understanding and treatment options for this condition. Market dynamics may vary across regions due to differences in healthcare infrastructure, regulatory frameworks, and access to specialized care and medications for rare diseases like Blau syndrome. #Company AbbVie Novartis Teva Pharmaceuticals Pfizer Amgen Janssen Global Services LLC MYLAN N.V. Fulgent Genetics Invitae Alkem Labs Limited Accord Healthcare Zydus Group #Type • Oral • Parenteral #Application • Hospital • Specialty Clinic • Other #BlauSyndrome #RareDisease #DrugDevelopment #PrecisionMedicine

PharmaCircle has published our Special Report: Eyeing Up the Ophthalmic Disease Market 2024. This is a 27-page special report that provides an in-depth view into the eye disease market, including top product sales, key approvals, drug delivery technologies, formulation/excipients, epidemiology and much more. The special report references hand-curated content from our subscription database. This is PharmaCircle’s first special report designed to cover an important niche area of pharmaceutical development and specifically tailored to companies active in this space.   Special Report’s Table of Contents:   •      Introduction •      Key Product Sales •      Epidemiology •      NME Pipeline •      Cell And Gene Therapy •      Formulation And Excipients •      Recent Key Approvals •      Delivery Beyond Topicals •      Pestel •      Strategic Deals, Partnerships and Acquisitions •      CMO/CDMO Landscape •      The Future   PharmaCircle has been a leading information provider to the life sciences industry for more than 20 years, providing hand-curated data on drug, biologics and combination product development that is comprehensive, reliable and analysis-ready.  Many life sciences innovators and manufacturers are PharmaCircle clients and rely on our full complement of research, development, clinical, business intelligence, supply chain and regulatory content and analytics for mission-critical projects.   To learn more about how to order this Special Report, please contact us at info@pharmacircle.com or go to https://lnkd.in/e2xWQhzT. 

A Priority Review Voucher (PRV) is a program established by the U.S. Food and Drug Administration (FDA) to incentivize the development of treatments for certain neglected diseases or pediatric rare diseases. The purpose of a PRV is to encourage pharmaceutical companies to invest in research and development for these diseases, which may not have significant commercial markets. By offering a PRV as a reward, the FDA aims to stimulate the development of new drugs and therapies for these underserved patient populations. When a company receives FDA approval for a qualifying drug, they are awarded a PRV. This voucher can be used to expedite the review process for any future drug application submitted by the company. In addition to using the PRV for their own drug applications, companies also have the option to sell or transfer the voucher to another company. This creates a market for PRVs, where companies can monetize the voucher by selling it to another pharmaceutical company that wants to accelerate the review of their own drug application. Overall, the PRV program serves as a valuable incentive for companies to invest in the development of treatments for neglected diseases or pediatric rare diseases.