Tagged News
Aflibercept 8 mg Shows Extended Dosing Intervals in Real-World nAMD Treatment
• Real-world data from nearly 40,000 patients reveals aflibercept 8 mg allows for extended dosing intervals of 2+ weeks in neovascular AMD patients compared to previous anti-VEGF treatments.
• Treatment-experienced patients switching to aflibercept 8 mg demonstrated dosing interval extensions of 16-21 days, with treatment-naive patients achieving approximately 10-week intervals between injections.
• Early efficacy data shows vision improvements in patients with baseline vision of 20/50 or worse, suggesting potential benefits in both treatment-naive and treatment-experienced populations.
FDA Initiates PDUFA VIII Reauthorization Process with Public Meeting and Assessment Report
• FDA has announced a hybrid public meeting on July 14, 2025, to begin the reauthorization process for PDUFA VIII, as the current authorization expires in September 2027.
• The agency has completed an independent assessment of product quality information request communications between FDA and sponsors, with findings now available for public comment.
• PDUFA VII introduced several enhancements including new meeting types, pilot programs for rare diseases and real-world evidence, and strengthened support for cell and gene therapy product development.
Dawn Health Secures €11.5 Million to Expand Pharma-Focused Digital Health Platform
• Copenhagen-based Dawn Health has raised €11.5 million from existing investors to scale its regulatory-grade digital health platform designed specifically for pharmaceutical companies.
• The platform, already utilized by industry leaders like Merck and Novartis, supports patients with conditions including oncology, multiple sclerosis, and rare pediatric diseases through AI-driven symptom tracking and clinical integration.
• This funding will accelerate Dawn Health's SaaS delivery model, product development, and international expansion as the company aims to become the global leader in pharmaceutical digital health solutions.
Study Confirms Clinical Trial Results Accurately Predict Real-World Benefits of Prostate Cancer Therapies
• New research comparing randomized controlled trials and real-world outcomes for prostate cancer treatments shows clinical trials may slightly underestimate therapeutic benefits in actual practice.
• The study examined multiple drug classes including androgen-targeted therapies, radiopharmaceuticals, and immunotherapy approved between 2010-2019, finding consistent or better real-world effectiveness.
• These findings provide reassurance that clinical trial results can reliably predict treatment outcomes across diverse patient populations despite the typically more selective trial enrollment criteria.
Real-World Data Emerges as Key Solution to Oncology Treatment Challenges
• Oncologists face significant challenges with treatment costs and financial toxicity, as novel cancer therapies often come with prohibitively high price tags passed on to patients.
• The rapid proliferation of new cancer treatments has created uncertainty about optimal sequencing, as clinical trials typically evaluate therapies in specific settings that may not reflect real-world practice.
• Real-world data (RWD) and real-world evidence (RWE) are becoming increasingly important tools to address these challenges and improve oncology reporting and treatment decisions.
FDA-Approved Lenire Device Shows 91.5% Success Rate in Real-World Tinnitus Treatment Study
• A new study published in Nature Communications Medicine reveals that 91.5% of tinnitus patients experienced clinically meaningful improvement after 12 weeks of treatment with the Lenire bimodal neuromodulation device.
• The retrospective analysis of 220 patients represents one of the largest real-world studies of tinnitus treatment, confirming results from previous clinical trials that led to FDA approval in March 2023.
• Lenire works by simultaneously delivering audio tones through headphones and mild electrical pulses to the tongue, offering a promising treatment option for the estimated 25 million Americans suffering from tinnitus.
Long-Term Follow-Up of TRANSFORM Trial Shows Sustained Benefits of Liso-Cel CAR T-Cell Therapy in Relapsed LBCL
• Three-year follow-up data from the phase 3 TRANSFORM trial demonstrates lisocabtagene maraleucel (liso-cel) significantly improved event-free survival with a median of 29.5 months versus 2.4 months with standard of care in relapsed large B-cell lymphoma.
• Liso-cel showed impressive efficacy with an 87% overall response rate and 74% complete response rate, while maintaining a favorable safety profile with lower rates of cytokine release syndrome and neurotoxicity compared to axicabtagene ciloleucel.
• The study revealed that patients who received liso-cel as second-line therapy had substantially better outcomes than those who crossed over after standard chemotherapy, emphasizing the importance of early CAR T-cell intervention.
Related Clinical Trials:
Kite, A Gilead Company
Posted 1/25/2018
The Lymphoma Academic Research Organisation
Posted 3/10/2021
Juno Therapeutics, a Subsidiary of Celgene
Posted 7/27/2018
Mallinckrodt to Present Five Clinical Studies on TERLIVAZ for Hepatorenal Syndrome at DDW 2025
• Mallinckrodt will present five clinical analyses of TERLIVAZ (terlipressin) for hepatorenal syndrome with rapid reduction in kidney function (HRS-AKI) at Digestive Disease Week 2025 in San Diego.
• TERLIVAZ is the first and only FDA-approved treatment for improving kidney function in adults with HRS-AKI, a rare and life-threatening condition affecting approximately 42,000 Americans annually.
• The presentations will include real-world evidence on treatment outcomes in various patient populations, including those on transplant waitlists and with alcohol-associated liver disease.
Decade-Long UK Registry Reveals Key Insights into Idiopathic Pulmonary Fibrosis Management and Future Directions
• The British Thoracic Society's analysis of the UK Idiopathic Pulmonary Fibrosis Registry provides the first comprehensive long-term data on over 5,000 IPF cases across 64 UK hospitals.
• Key findings include significant diagnostic delays with 60% of patients experiencing symptoms for over a year before their first clinic visit, and a shift in antifibrotic treatment preferences from pirfenidone to nintedanib after 2017.
• Experts are now advocating for a shift from consensus-based criteria toward personalized, data-driven approaches for early diagnosis and treatment, with initiatives like the global PRIME-PPF study aiming to refine risk stratification.
CVAC System Demonstrates Superior Stone Clearance in Multiple Real-World Studies at AUA 2025
• Real-world data presented at AUA 2025 shows the CVAC System achieves significant kidney stone volume reduction of up to 99% in most patients, with one study reporting a median clearance rate of 96.4%.
• The CVAC System demonstrated superior performance compared to flexible and navigable ureteric access sheath (FANS), removing significantly more absolute stone volume while maintaining consistently low intrarenal pressure.
• Multiple studies using Quantitative Stone Analysis Software (QSAS) validate stone volume as a more precise and clinically meaningful measure for assessing kidney stone treatment outcomes.