Clinical Trial News
NICE Provisionally Approves BioMarin's Vimizim for Ultra-Rare Morquio Disease with Real-World Data Requirements
- NICE has issued a provisional approval for BioMarin's Vimizim (elosulfase alfa), priced at £395,000 per patient annually, for treating the ultra-rare Morquio disease affecting 88 patients in England.
- The approval is contingent on implementing real-world data collection and establishing a price cap, marking NICE's innovative approach to managing high-cost rare disease treatments.
- The MPS Society welcomes the decision while expressing concerns about further delays in the approval process, as patients urgently await access to this life-changing therapy.
FDA Approves Two New Ustekinumab Biosimilars, Bringing Total US Biosimilar Count to 65
- The FDA has approved Biocon's Yesintek (ustekinumab-kfce) and Celltrion's Steqeyma (ustekinumab-stba) in November and December 2024, expanding treatment options for autoimmune conditions including Crohn's disease and psoriasis.
- Of the 65 FDA-approved biosimilars, 14 have received interchangeable designation, allowing pharmacists to substitute them for reference products without prescriber intervention, enhancing patient access to more affordable treatments.
- The biosimilar landscape continues to evolve with products spanning five major classes: insulin, granulocyte colony-stimulating factors, monoclonal antibodies, TNF-alpha inhibitors, and VEGF inhibitors.
Ropeginterferon Alfa-2b Shows Promise in Polycythemia Vera Treatment
- Ropeginterferon alfa-2b demonstrates a high overall response rate of 90% in polycythemia vera (PV) patients, including complete and partial hematologic responses.
- The study found no dose-limiting toxicities with ropeginterferon alfa-2b, supporting its favorable safety profile and potential for long-term use.
- Treatment with ropeginterferon alfa-2b led to significant reductions in JAK2 V617F allelic burden, indicating molecular responses in PV patients.
- The research suggests that ropeginterferon alfa-2b could effectively normalize blood cell counts and prevent thromboembolic complications in PV patients.
Highlighted Clinical Trials:
AOP Orphan Pharmaceuticals AG
Posted 8/1/2010
AstraZeneca Expands Immuno-Oncology Portfolio with Three Strategic Partnerships
- AstraZeneca secures rights to Inovio's INO-3112, a DNA vaccine targeting HPV-related cancers, in a deal worth up to $727.5 million, planning combination studies with durvalumab.
- MedImmune partners with Heptares on adenosine A2A receptor antagonist HTL-2071 for $510 million, aiming to enhance T-cell anti-cancer activity.
- A collaboration with Mirati Therapeutics will explore combining durvalumab with HDAC inhibitor mocetinostat in non-small cell lung cancer trials starting next year.
Europe Emerges as Leading Hub for Precision Medicine Innovation and Implementation
- Europe's healthcare systems are rapidly adopting genomic medicine and personalized therapies, driven by collaboration among providers, payers, and pharmaceutical companies across the continent.
- The convergence of advanced diagnostics, genetic analytics, and companion diagnostics has created a mature ecosystem for precision medicine delivery in everyday clinical practice.
- European institutions' willingness to share platforms and data while maintaining security standards positions the continent to potentially surpass the US in precision medicine commercialization.
India Revamps Clinical Trial Regulations to Align with Global Standards
- India has significantly reformed its clinical trial regulations, extending SAE reporting timelines from 10 to 14 days to match international standards and streamlining the approval process.
- The regulatory framework now requires trials to be evaluated based on risk-benefit assessment, innovation value, and unmet medical needs in the country, ensuring greater transparency.
- New draft standards for accreditation of sites, investigators, and ethics committees have been introduced by CDSCO, aiming to enhance operational standards across clinical research.
Palbociclib Plus Letrozole Significantly Extends Progression-Free Survival in ER+ Breast Cancer
- Palbociclib, a CDK4/6 inhibitor, combined with letrozole, an aromatase inhibitor, significantly improved progression-free survival (PFS) in postmenopausal women with ER+/HER2- metastatic breast cancer.
- The PALOMA-1/TRIO-18 phase II trial demonstrated a median PFS of 20.2 months with the palbociclib-letrozole combination, compared to 10.2 months with letrozole alone.
- Palbociclib received accelerated FDA approval based on these results, pending confirmatory phase III trial outcomes, marking a significant advancement in targeted therapy for hormone receptor-positive breast cancer.
- Common adverse events associated with the combination therapy included neutropenia and leukopenia, manageable through dose modification and blood count monitoring.
Highlighted Clinical Trials:
Eli Lilly and Company
Posted 4/20/2015
University of Texas Southwestern Medical Center
Posted 1/24/2014
Pfizer
Posted 10/19/2012
German Breast Group
Posted 10/30/2013
Royal Marsden NHS Foundation Trust
Posted 3/25/2015
University of California, San Francisco
Posted 10/19/2015
Pfizer
Posted 3/23/2015
Spanish Breast Cancer Research Group
Posted 3/13/2014
Dana-Farber Cancer Institute
Posted 1/1/2014
Washington University School of Medicine
Posted 4/10/2013
Pancreatic Cancer Action Network and Perthera Launch "Know Your Tumor" Initiative for Precision Treatment
- The Pancreatic Cancer Action Network has partnered with Perthera to launch "Know Your Tumor" initiative, offering molecular profiling services to guide personalized treatment decisions for pancreatic cancer patients.
- The program aims to streamline molecular testing processes, providing comprehensive tumor analysis reports within one month of biopsy, while building a database of approximately 3000 patient molecular profiles over three years.
- With pancreatic cancer's dire 6% five-year survival rate, this initiative represents a significant step toward the goal of doubling survival rates by 2020 through personalized treatment approaches.
Serum Free Light Chain Levels Predict Need for Future PCI in STEMI Patients
- Combined free immunoglobulin light chain (cFLC) levels were evaluated in ST-elevation myocardial infarction (STEMI) patients, showing a peak on day 7 post-MI.
- cFLC levels in STEMI patients correlated with cystatin C and negatively correlated with CD14++CD16- monocytes, indicating associations with renal function and inflammation.
- Elevated cFLC concentrations were associated with an increased need for future percutaneous coronary intervention (PCI), suggesting a predictive value.
- The study highlights the potential of cFLC levels as a biomarker for risk stratification in STEMI patients regarding future interventional needs.
Novartis Revolutionizes Clinical Trial Recruitment Through Digital Innovation and Social Media
- Novartis has launched a dedicated Twitter channel @NovartisCancrUS reaching nearly 10,000 followers to promote cancer clinical trials and facilitate patient recruitment in the United States.
- The company has developed an interactive global clinical trials database with GPS functionality, allowing patients worldwide to locate and connect with nearby trial opportunities.
- Novartis's innovative Signature trial program enables personalized cancer trials across the US, matching patients' genetic alterations to relevant drugs while allowing them to receive treatment closer to home.
Highlighted Clinical Trials:
Novartis Pharmaceuticals
Posted 5/1/2014