Betibeglogene autotemcel (beti-cel; Zynteglo; formerly Lentiviral Autologous Beta-Globin Gene Therapy, LentiGlobin) is an autologous hematopoietic stem cell-based gene therapy for the treatment of adult and pediatric patients with β-thalassemia who require regular red blood cell (RBC) transfusions.
Emerging Technology Reports
The Emerging Technology Report provides regular updates about emerging and potentially disruptive technologies that are likely to have a significant impact in healthcare. This “early warning” report monitors new health technologies from 6 months to 2 years within a regulatory submission and tracks those as they go through clinical trial development and the regulatory approval process.
For each emerging technology, our analysts describe the technology and intended use, its prospects relative to the regulatory process and FDA information, competing technologies, relevant clinical guidelines, high-level look at the evidence such as published abstracts in the medical literature and available meeting abstracts, and available clinical trials.
iStent infinite Trabecular Micro-Bypass System for Open-Angle Glaucoma
The iStent infinite is a trabecular micro-bypass stent system intended to reduce intraocular pressure (IOP) as a stand-alone procedure for uncontrolled primary open-angle glaucoma (OAG). The device includes 3 heparin-coated titanium stents measuring one-third millimeter (mm) in length and width. The stents are preloaded into an auto-injection system that allows the surgeon to…
Pegzilarginase for Arginase 1 Deficiency
Pegzilarginase is an investigational recombinant form of the human enzyme arginase 1 (ARG1) proposed for the treatment of pediatric and adult patients with ARG1 deficiency. It is administered by intravenous infusion or subcutaneous injection.
Fexapotide Triflutate for Benign Prostatic Hyperplasia
Fexapotide triflutate is an investigational injectable apoptotic agent proposed for the treatment of benign prostatic hypertrophy (BPH).
Quell Wearable Neurostimulation Device for Fibromyalgia
Quell is a wearable transcutaneous electrical nerve stimulator (TENS) device indicated as an aid for reducing the symptoms of fibromyalgia in adults with high pain sensitivity, including use during sleep.
GammaSense Stimulation System for Alzheimer Disease
The GammaSense Stimulation System is an investigational, home-use, wearable device that delivers auditory and visual stimulation to entrain gamma frequency neural activity in the brain. It is proposed for the treatment of mild to moderate Alzheimer disease (AD). Brainwave entrainment is a technique to prompt the brain’s neural activity to synchronize to the frequency of exter…
Organ Care System (OCS) Heart
The OCS Heart is a portable extracorporeal heart perfusion and monitoring system indicated for the preservation of donation-after-brain-death (DBD) donor hearts deemed unsuitable for procurement and transplantation at initial evaluation due to limitations of prolonged cold static cardioplegic preservation (e.g., > 4 hours of cross-clamp time); and for the ex vivo reanimation,…
Mavacamten (Camzyos) for Obstructive Hypertrophic Cardiomyopathy
Mavacamten (Camzyos) is an oral cardiac myosin inhibitor indicated for the treatment of adults with symptomatic New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy (HCM) to improve functional capacity and symptoms.
BlueLeaf Endovenous Valve Formation (EVF) System for Chronic Venous Insufficiency
The BlueLeaf Endovenous Valve Formation (EVF) System is an endovascular device intended to form autogenous tissue leaflets from femoral and popliteal vein walls that mimic a functional valve. It is under investigation for the treatment of deep vein reflux in chronic venous insufficiency.
Narsoplimab for Stem Cell Transplant-Associated Thrombotic Microangiopathy
Narsoplimab is an investigational, intravenous monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2) for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA).