Clinical Trial News
FDA Grants Breakthrough Status to Devices Targeting Alzheimer's, Heart Failure, and More
• The FDA granted breakthrough device designation to Boston Scientific's Vercise deep brain stimulation (DBS) system for mild probable Alzheimer's in patients 65 and older.
• Cognito Therapeutics received breakthrough designation for its neurostimulation device, designed to reactivate the brain's immune system by stimulating it at a specific frequency.
• Puzzle Medical Devices' ModulHeart, a minimally invasive transcatheter heart pump, received breakthrough status, offering a potential alternative to open-heart surgery for heart failure patients.
FDA Approves Prucalopride for Chronic Idiopathic Constipation
The FDA has approved prucalopride (Motegrity) for treating chronic idiopathic constipation (CIC), offering a new treatment option that enhances colonic peristalsis to increase bowel motility. This approval is based on clinical studies showing significant improvements in patients.
Jazz Pharmaceuticals Acquires GW Pharma in $7.2B Cannabis Medicine Deal
• Jazz Pharmaceuticals has agreed to acquire GW Pharmaceuticals for $7.2 billion, marking a significant expansion into cannabis-derived medicines and creating a leader in neuroscience therapeutics.
• GW's flagship product Epidiolex, the first FDA-approved cannabis-based medicine, achieved $510 million in sales in 2020 and holds patent protection until at least 2035.
• The acquisition diversifies Jazz's portfolio beyond its narcolepsy drug Xyrem and includes GW's late-stage pipeline candidate nabiximols, currently in Phase 3 trials for MS spasticity.
Disease Rarity and Clinical Benefits Drive Rare Disease Drug Pricing in Germany's AMNOG System
• Germany's AMNOG system evaluates rare disease drugs based on multiple factors, with drugs treating ultra-rare conditions generally commanding higher prices while staying below €50 million annual sales threshold.
• Analysis of over 20 rare disease drugs reveals that G-BA benefit ratings significantly influence pricing, with treatments like Spinraza and Orkambi securing higher reimbursements due to demonstrated clinical benefits.
• Clinical trial design and comparator choice show less impact on pricing decisions, with German payers focusing more on data robustness and incremental benefits regardless of trial methodology.
BMS Seeks FDA Approval for Zeposia in Ulcerative Colitis with May Decision Date
• Bristol-Myers Squibb has submitted Zeposia (ozanimod) for FDA review in treating moderate to severe ulcerative colitis, with a priority review decision expected by May 30.
• In clinical trials, Zeposia demonstrated significant efficacy with 18.4% of patients achieving clinical remission at 10 weeks compared to 6.8% on placebo, and 37% maintaining remission at one year.
• If approved, Zeposia would become the first S1P modulator for ulcerative colitis, potentially expanding BMS's market presence beyond its current multiple sclerosis indication.
Promising Results for Ziresovir in Treating Respiratory Syncytial Virus in Infants
A recent phase 3 trial has shown promising results for the approval of ziresovir as a treatment for respiratory syncytial virus in infants.
Medtronic's DiamondTemp Ablation System Receives FDA Approval for Atrial Fibrillation Treatment
• Medtronic's DiamondTemp Ablation (DTA) system has gained FDA approval for treating recurrent, symptomatic paroxysmal atrial fibrillation (AF) in patients unresponsive to drug therapy.
• The DTA system is the first FDA-approved, temperature-controlled, irrigated radiofrequency (RF) ablation system with diamonds, offering real-time feedback for precise lesion creation.
• Clinical trials demonstrated the DTA system's safety and effectiveness, with improved procedural efficiencies like shorter RF ablation times and reduced saline infusion volumes.
• The DIAMOND-AF trial showed a 96.7% freedom from patient complication rate in the DTA group, compared to 93.4% in the control group (P<0.0001).
Sangamo Therapeutics Charts Pragmatic Course in Gene Editing Evolution, Balancing Innovation with Viability
• Sangamo Therapeutics has strategically diversified beyond pure gene editing, building a three-pillar approach across gene therapy, ex vivo editing, and in vivo genome engineering to ensure financial sustainability.
• Under CEO Sandy Macrae's leadership, Sangamo has secured $1.6 billion in funding, established six big pharma partnerships, and developed proprietary zinc finger technology for precise genetic manipulation.
• The company emphasizes cautious progression in genome editing, focusing initially on rare diseases while building evidence for broader applications, with manufacturing capability seen as critical for long-term success.
US Biosimilar Market Set for $100 Billion Cost Reduction as Adoption Accelerates
• The US biosimilar market has shown significant growth with 20 approved products, projected to reduce drug costs by $100 billion over the next five years through increased competition and adoption.
• Recent oncology biosimilars in bevacizumab, trastuzumab, and rituximab markets are achieving record market share, expected to reach 60% combined volume share within two years of launch.
• Regulatory changes have expanded biosimilar categories to include 90 new molecules, with insulin biosimilars poised to enter the market and potentially transform diabetes care costs.
Rising Liver Cancer Cases in the US Linked to Alcohol-Associated Liver Disease and MASLD
From 2000 to 2021, the US saw a significant increase in primary liver cancer cases and deaths, primarily due to Alcohol-Associated Liver Disease (ALD) and Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD).