At #DIA2024, a panel of experts, including Dr. Peter Marks, Director of the FDA’s Center for Biologics Evaluation and Research, expressed strong support for the accelerated approvals program. It has expedited drugs that fulfill unmet medical needs and provided patients with life-extending treatments for rare cancers. Joanne Eglovitch recaps the session via Regulatory Affairs Professionals Society (RAPS): https://lnkd.in/gkkD-AYw
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𝐂𝐞𝐥𝐥𝐭𝐫𝐢𝐨𝐧 𝐔𝐒𝐀 𝐒𝐮𝐛𝐦𝐢𝐭𝐬 𝐁𝐋𝐀 𝐟𝐨𝐫 𝐂𝐓-𝐏𝟒𝟕: 𝐀 𝐍𝐞𝐰 𝐄𝐫𝐚 𝐢𝐧 𝐑𝐡𝐞𝐮𝐦𝐚𝐭𝐨𝐢𝐝 𝐀𝐫𝐭𝐡𝐫𝐢𝐭𝐢𝐬 𝐓𝐫𝐞𝐚𝐭𝐦𝐞𝐧𝐭 📄 Celltrion USA Celltrion USA has submitted the Biologics License Application (BLA) for CT-P47, a biosimilar candidate of ACTEMRA® (tocilizumab), to the U.S. FDA. 💊 CT-P47, containing tocilizumab, aims to provide a more accessible treatment option for rheumatoid arthritis. The submission is based on positive Phase III trial results, evaluating efficacy, safety, and more. 👥 Thomas Nusbickel Tom Nusbickel, Chief Commercial Officer at Celltrion USA, emphasizes the importance of this step in offering patients a diverse product lineup in the autoimmune disease market. 📆 Stay tuned as Celltrion actively cooperates with the FDA for a potential new treatment option for rheumatoid arthritis. #HealthcareInnovation #Biotech #FDASubmission #RheumatoidArthritis #BioPharma #MedicalAdvancements #CTP47 #ACTEMRA #CelltrionUSA #ClinicalTrials
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The FDA has issued a draft guidance providing recommendations to sponsors of allogeneic cell-based medical products on appropriate cell safety testing to support an Investigational New Drug Application (IND) or Biologics License Application (BLA). Rather than presenting a prescriptive checklist, the guidance encourages a dynamic, risk-based assessment throughout the development process. By highlighting factors like cell expansion potential, culture reagent impact, and the scope of potential patient exposure, the guidance prompts a nuanced evaluation of safety considerations beyond traditional paradigms. The draft guidance reinforces the interconnected nature of drug development, where decisions made at one stage can have broader implications throughout the entire process. This interconnectedness underscores the need for early, cross-functional communication and proactive risk assessment strategies. As the science and technology behind allogeneic cell therapies continue to progress, this draft guidance offers valuable guidance for navigating the complexities of this evolving landscape and advancing the development of safe and effective therapies. Link to the draft guidance: https://lnkd.in/df7nF8x5 #FDA #CellTherapy #RegenerativeMedicine #AdvancedTherapeutics #AllogeneicCells #RiskBasedApproach #IND #BLA #CMC
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Amylyx Pharmaceuticals' ALS drug, Relyvrio, didn't meet its goals in the crucial Phase III trial, casting uncertainty on its future. Despite this, the drug's safety profile remained consistent. Considerations - 🔹 The impact on ALS treatment advancements and patient hope. 🔹 Potential regulatory discussions and the commitment to patient well-being may guide Amylyx's next steps. This setback in ALS research emphasizes the challenges in neurodegenerative disease treatment but also the unwavering quest for solutions. #ALS #HealthcareNews #ClinicalTrials
Future of Amylyx’s ALS Drug in Question After Phase III Failure | BioSpace
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Head of International Affairs Department - Italian Medicines Agency / Hospital Pharmacist specializing in Regulatory Affairs
Choosing predictive and clinically relevant endpoints is a key aspect of drug development in the regulatory space. By assessing orphan drug development for sickle cell disease (SCD) over the past two decades, we observed that failures in clinical development were mainly found in phase 3 due to lack of efficacy on vaso-occlusive crises as the primary study endpoint. This was likely related to variable definitions and heterogeneity of pain scoring and treatment. If you are interested in improving the debate on how fostering pharmaceutical R&D on SCD (and other rare diseases), you may wish to read our last work on this. https://lnkd.in/difgpBep #sicklecell #orphandrugs
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Small drops, big impact! Please, do learn more about the possibilities of remote sampling! My team members Suzanne Lanooij and Anne Martens (in collaboration with Theo Rispens), share their thoughts in a short review article for the International Association of Therapeutic Drug Monitoring and Clinical Toxicology (IATDMCT) on how a small finger prick sample, taken at home, can be used for Therapeutic drug monitoring (TDM) of Biologics. https://lnkd.in/erCPUYmY (paid content) Home-sampling for TDM of biologics is as reliable as normal venipuncture, and therefore serves as a great alternative since it is less painful, while saving time and costs for all patients. This diagnostic service can be requested by hospitals. Are you interested in how small drops of blood at home can have a great impact on patient’s lives? Visit https://lnkd.in/e8rqxRUR or contact us to get started. #Fingerprick #Homesampling #Microsampling #TDM #IATDMCT
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Despite a challenging prior year, 2023 saw a remarkable surge in FDA approvals for groundbreaking medicines, marking a turning point in healthcare. From novel oncology treatments to innovative therapies for rare diseases, this bounty of approvals signifies hope and progress for patients globally. Read more about these game-changing approvals below! #HealthcareAdvancements #pharma #InnovativeMedicines
2023 drug approvals: After a down year, FDA signs off on a bounty of new meds, including 7 from Pfizer
fiercepharma.com
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Small drops, big impact! Please, do learn more about the possibilities of remote sampling! Our Sanquin colleagues Suzanne Lanooij and Anne Martens (of the groups of Annick de Vries and Theo Rispens), share their thoughts in a short review article for the International Association of Therapeutic Drug Monitoring and Clinical Toxicology (IATDMCT) on how a small finger prick sample, taken at home, can beused for Therapeutic drug monitoring (TDM) of Biologics. https://lnkd.in/erCPUYmY (paid content) Home-sampling for TDM of biologics is as reliable as normal venipuncture, and therefore serves as a great alternative since it is less painful, while saving time and costs for all patients. This available diagnostic service can be requested by hospitals. Are you interested in how small drops of blood at home can have a great impact on patient’s lives? Visit Fingerpricks (https://lnkd.in/esZE2sJJ) or contact us to get started. #Fingerprick #Homesampling #Microsampling #TDM #IATDMCT
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Introduced in the 1980s, biologic medications have since become important tools in modern medicine. However, biologics are expensive, greatly affecting the healthcare budgets of both underdeveloped and developed countries. Fortunately, biosimilars, which are highly similar, reverse-engineered versions of existing biological medicines and their active ingredients, are now available as more affordable options for patients treated with biologics. This book discusses biosimilars with chapters on clinical trials, regulation, pharmacovigilance, and the interchangeability of biosimilars with biologics. It also addresses future trends in the biosimilars market. https://lnkd.in/dwZGv6SR
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Enhancing the accuracy of patient selection in clinical trials is crucial for the success of new treatments. The FDA’s recent guidelines highlight the importance of enrichment strategies, including improved patient selection, to optimize trial outcomes. Patient misselection remains a significant challenge in clinical trials, often leading to ineffective results and increased costs. Delphi-MD by QuantalX offers advanced, objective tools to refine patient selection, ensuring that only the most appropriate candidates are enrolled, thereby improving trial efficiency and effectiveness: https://www.quantalx.com Learn more about the guidelines at https://vist.ly/3mgsb7x #ClinicalTrials #Pharma #PatientSelection #FDA #DelphiMD #QuantalX
Brain Health Diagnosis
fda.gov
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Continued Emphasis on the Brain Disease Model: Despite its limitations, the brain disease model continues to dominate largely because it supports the development and funding of medical treatments and pharmaceutical interventions, which are concrete, scalable, and economically beneficial for the medical and pharmaceutical industries. Additionally, this model has played a significant role in shaping public policies and research agendas. #Addiction #Recovery
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