Evidentic GmbH’s Post

FDA's Office of New Drugs (OND) Research Outcomes Report for FY2022-2023! This comprehensive report highlights the significant strides made in regulatory science, aimed at improving new drug approval processes and addressing unmet medical needs. Key Highlights: 1.Research Contributions: Over 80 external presentations and 94 peer-reviewed publications showcasing groundbreaking work in various therapeutic areas. 2.Innovative Projects: a.Development of clinical outcome assessments for rare diseases. b.Physiologically-based pharmacokinetic modeling for predicting fetal exposure to maternal drugs. c.Advancements in pediatric oncology and acute pain treatment strategies. 3.Regulatory Impacts: 33 outcomes that directly facilitated regulatory decision-making, including new drug development tools and internal policies. Read the full report #FDA #DrugDevelopment #ClinicalResearch #RegulatoryScience #Innovation #Healthcare

The Alliance for Regenerative Medicine has expressed concerns about the potential impact the European Union #HTA Coordination Group’s recent guidance may have on #ATMPs and #rarediseases. Read at The Evidence Base® #rwd #realworlddata #marketaccess #pharma #biopharma #hta #healthtecnologyassessment #healthdata #healthequity #JCA #jointclinicalassessment #singlearmtrials #externalcontrolarms

AB Science Secures European Patent for Masitinib, Strengthening Leadership in Mastocytosis Treatment AB Science SA announced a significant milestone with the European Patent Office granting a patent (EP 3359195A1) for masitinib in the treatment of severe Mastocytosis, providing protection until October 2036. Masitinib, a leading compound by AB Science, is now protected in Europe for use in severely symptomatic systemic mastocytosis patients, including those unresponsive to current treatments. This patent also covers patients with severe symptoms like pruritus, flushes, or depression. This European patent complements existing IP protection in the USA and Japan, reinforcing AB Science's global strategy. Additionally, masitinib holds orphan drug designations from the EMA and FDA, ensuring market exclusivity upon approval, and emphasizing the need for effective treatments in rare diseases. AB Science’s broader vision includes securing patents for masitinib in amyotrophic lateral sclerosis, multiple sclerosis, Alzheimer's disease, and prostate cancer, demonstrating their commitment to addressing challenging conditions and improving patient outcomes. Why it matters? • Market Dynamics: The exclusivity provided by the patent means that masitinib will likely become a leading treatment option for severe mastocytosis, potentially reducing the market share for alternative treatments. • Research and Development: Competing pharmaceutical companies might redirect their R&D efforts towards different indications or alternative therapies to avoid direct competition with masitinib. • Pricing and Accessibility: The exclusivity and potential effectiveness of masitinib could influence pricing strategies, making it a benchmark for cost-effectiveness in mastocytosis treatment. • Regulatory Strategies: Companies might need to reassess their regulatory strategies, considering masitinib’s strong IP position and orphan drug status. 📊 GlobalData analysis from Patent Analytics tool shows the key players with publications related to treating systemic mastocytosis disease:   ⚡ Connect with us today to explore the endless possibilities and propel your business forward! 📧 patents@globaldata.com or ☎ explore more at our contact page: https://lnkd.in/ggfMtshS #ABScience #Masitinib #Mastocytosis #EuropeanPatent #OrphanDrug #EMA #FDA #MedicalInnovation #RareDisease #IPProtection #ClinicalResearch #HealthcareInnovation #PatientCare #MedicalBreakthrough #PharmaNews

**Breaking news for the medical community and those living with primary biliary cholangitis (PBC)! 🎉** Gilead Sciences Sciences has received FDA accelerated approval for **Livdelzi (seladelpar)**, a groundbreaking oral therapy that offers new hope as a second-line treatment for PBC. Livdelzi, a peroxisome proliferator-activated receptor (PPAR) delta agonist, has demonstrated sustained efficacy and safety in clinical trials, significantly improving the lives of many with PBC. Key highlights include: - **62% of patients** achieved the primary endpoint in the Phase 3 RESPONSE study, compared to 20% with placebo. - **25% of patients** experienced normalization of alkaline phosphatase (ALP), a critical biomarker for liver health. - Livdelzi also showed significant improvements in pruritus (chronic itching), a debilitating symptom for PBC patients. This approval is a major milestone for Gilead and the PBC community, offering a new option for those who have had an inadequate response to existing treatments. Congratulations to everyone involved in bringing this innovative treatment to market, and here's to a brighter future for those affected by PBC! 🌟 #HealthcareInnovation #FDAApproval #PBC #LiverDisease #GileadSciences #Pharmaceuticals #Biotechnology

🗺️ To Attend an Upcoming Symposium on Advanced Therapy Medicinal Products (ATMPs) I am thrilled to be part of a special symposium that marks a significant milestone-15 years of groundbreaking advancements in Advanced Therapy Medicinal Products (ATMPs). This event will not only celebrate this remarkable journey but also highlight how the work of the Committee for Advanced Therapies (CAT) has been instrumental in shaping the regulatory framework in Europe. Their efforts have led to the approval of 27 innovative gene- and cell-based therapies, offering new hope for patients with unmet medical needs. This symposium will give a unique opportunity to gain valuable insights into the evolution of CAT, discuss regulatory developments, and explore upcoming innovations that could revolutionize patient care in the next 15 years. - Learn about the evolution of CAT and how it has adapted to meet the challenges of emerging ATMP technologies. - Discuss with experts the regulatory developments that continue to shape the future of ATMPs. - Explore the upcoming innovations that have the potential to revolutionize patient care in the next 15 years. I’m looking forward to engaging with Frontier to develop drugs and other industry leaders, scientists, and regulatory experts. These conversations are essential as we work together to ensure that high-quality, safe, and effective therapies continue transforming the healthcare landscape. I will share insights from the symposium with my network and continue building collaborations that will help advance these revolutionary treatments. I encourage you to do the same and look forward to staying connected as we collectively shape the future of medicine! #ATMPs #GeneTherapy #CellTherapy #HealthcareInnovation #ClinicalTrials #RegulatoryAffairs #CAT #EMA #MedicalInnovation #CNRResearch

Competitive Dynamics in Metabolic Disorder: Interesting Times to Witness The metabolic disorder landscape is undergoing a seismic shift, with recent FDA approvals igniting an intriguing competitive dynamic. In the last two weeks, Novo Nordisk's Wegovy received approval as a preventative for cardiovascular events in obese patients, while Madrigal's Rezdiffra secured the world's first approval for NASH with liver fibrosis. Although these approvals seem independent, a deeper analysis reveals the potential for intense competition between their underlying mechanisms. While Rezdiffra addresses a long-standing unmet need in NASH, it may face fierce competition from liver disease therapies and the increasingly prominent GLP-1 drug class. A Nature Reviews on Drug Discovery article highlights nearly 10 late-stage assets, including three GLP-1 analogs from Novo Nordisk, Lilly, and Boehringer Ingelheim/Zealand Pharma. Growing evidence suggests GLP-1 agonists exhibit anti-inflammatory effects on the liver, improving liver enzymes and potentially impacting fibrosis. A positive trial result could elevate GLP-1 agonists' competitive positioning, potentially relegating Rezdiffra to treating only advanced-stage patients. In this evolving landscape, Madrigal must invest in clinical trials to study Rezdiffra's preventive effect on NASH progression and associated fibrosis. Demonstrating efficacy in prevention could carve out a competitive advantage, solidifying Rezdiffra's position as a first-line therapy. As the battle for market share intensifies, the metabolic disorder arena will witness a captivating interplay of clinical data, strategic positioning, and commercial maneuvering. Pharmaceutical companies must stay agile, adapt to evolving dynamics, and leverage scientific expertise to differentiate offerings and secure a foothold in this rapidly evolving landscape. #MetabolicDisorders #NASH #LiverFibrosis #GLP1Agonists #CardiovascularRisk #ObesityTreatment #WeightLoss #DrugDevelopment #ClinicalTrials #CompetitiveStrategy #PharmaceuticalInnovation #MedicalBreakthroughs #HealthcareLandscape #FDAApprovals #BlockbusterDrugs #MarketCompetition #CommercialStrategy #BusinessIntelligence #TherapeuticAreaStrategy #ProductDifferentiation #StrategicPositioning #RegulatoryApprovals #PipelineAssets #LateStageAssets #PhaseIIITrials

Exciting News in the Field of Pharmaceutical Science! As a student deeply immersed in the world of pharmaceutical sciences, I am thrilled to share that the Medicines and Healthcare Products Regulatory Agency (MHRA) in the UK has recently given the green light to Zilucoplan, an innovative treatment for generalized myasthenia gravis (gMG). What is gMG? It's a rare autoimmune disorder where the immune system erroneously attacks components of the neuromuscular junction, leading to muscle weakness and fatigue. This can severely impact daily activities and even be life-threatening due to respiratory muscle weakness. Zilucoplan's Role: This pioneering medication is the first of its kind—a once-daily, subcutaneous, self-administered therapy specifically targeting patients with anti-acetylcholine receptor (AChR) antibody-positive gMG. Why this matters: The ability for patients to self-administer Zilucoplan daily could revolutionize treatment by empowering individuals with more control over their management plan and potentially enhancing their quality of life. The approval of Zilucoplan is more than just a scientific advancement because gMG affects 15 in every million people in the UK; it is a ray of hope for those who live with this difficult condition. As I continue my journey in the pharmaceutical sciences, it's breakthroughs like these that inspire me. They remind us of the profound impact that dedicated research and development can have on lives around the globe. #PharmaceuticalSciences #Innovation #RareDiseases #gMG #Zilucoplan #Healthcare #Neurology #PatientCare #futurescientists https://lnkd.in/e42v_rUE

In her recent Forbes article, Sally Pipes, president, CEO, and the Thomas W. Smith fellow in healthcare policy at the Pacific Research Institute, discusses the FDA's approval of donanemab, a new treatment for Alzheimer's disease developed by Eli Lilly and Company under the brand name Kisunla. This medication targets amyloid, a protein that accumulates in the brains of Alzheimer's patients, and has been shown to slow disease progression by 35% over 18 months compared to a placebo. This approval follows closely behind another similar drug called lecanemab, marketed as Leqembi by Eisai and Biogen, which also demonstrated efficacy in slowing Alzheimer's progression. The significance of these approvals cannot be overstated. They offer hope to millions of Alzheimer's patients and their families, potentially providing more quality time together and reducing the strain of caregiving on society. However, the future development of such life-changing treatments may be jeopardized by political pressures advocating for drug price controls, which could disincentivize pharmaceutical companies from investing in research and development for diseases like Alzheimer's. #SallyPipes #Alzheimers #DrugApproval #HealthcarePolicy

Proposed Model to Build Capacity for Emergency Use Authorization for Therapeutics: Guidance for National Medicine Regulatory Authorities The new Emergency Use Authorizations for Therapeutics: Guidance for National Medicine Regulatory Authorities, provides practical guidance to MRAs on adopting, implementing, and managing expedited approval pathways for therapeutics (drugs and non-vaccine biological products). The guidance provides recommendations for therapeutics emergency use authorization (EUA) and examples of different approaches Medicine Regulatory Authorities (MRAs) in low, middle, and high-income countries have taken to address these recommendations. The goal of this resource is to facilitate rapid access to safe, effective, and quality therapeutics in response to public health emergencies and to facilitate greater international collaboration, harmonization, and data sharing between MRAs. https://lnkd.in/eWbXeuyh

We will focus our operating and financial resources towards several initiatives, including establishing global partnerships for NTI164, our broad-spectrum cannabinoid drug therapy, and focusing on securing its registration in Australia, initially through the provisional registration pathway unique to Australia’s Therapeutic Drug Administration (TGA). Dr Thomas Duthy, Executive Director said, “The strategy announced today reflects our core focus to date on generating conclusive clinical evidence pertaining to the efficacy and safety of NTI164 for paediatric neurological patients with persistent or progressive neuroinflammation where standard therapies are urgently needed or lacking altogether. We believe Neurotech is now in a strong position to leverage our robust clinical data, accompanying intellectual property, manufacturing expertise and ongoing regulatory initiatives to deliver significant value to our shareholders moving forward through a combination of global corporate development through partnering, while in parallel leveraging our domestic expertise here in Australia with timely regulatory submissions initially for our orphan disease franchise.” Dr Duthy continued “We believe now is the right time to pursue an active strategy for our unique paediatric neurological franchise which consists of two rare (orphan) diseases with limited or no treatment options and autism with many patients where approved therapies are restrictive (nonenabling), have negative side-effects and are therefore not generally considered as effective front line treatments. We note that during 2023, at least 49 deals were announced involving rare neurological diseases, with disclosed values totalling US$13.2 billion. As a Board, we are fully committed to this strategy and are positioning the business to achieve these stated goals as quickly as possible and within our balance sheet capability with $11.6 million in cash as at 30 June 2024.” Read the full ASX Announcement here: https://lnkd.in/g5PmAftx #NTI #Neurotech #biotech #ASX #ASXnews #cannabinoid #GlobalPartnerships #NTI164 #CannabinoidTherapy #TGARegistration #Biopharma #DrugDevelopment #Australia #NeurologicalDisorders #HealthcareInnovation