Fidanacogene elaparvovec-dzkt (Beqvez; Pfizer Inc.) is a single-infusion gene therapy for the treatment of adults with moderate to severe hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated vi…
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.
Ventura Interatrial Shunt (V-Wave Ltd.) for Heart Failure
The Ventura Interatrial Shunt (V-Wave Ltd.) is an investigational transcatheter interatrial shunt proposed for treatment of chronic heart failure.
Rusfertide (Protagonist Therapeutics Inc./Takeda Pharmaceutical Co. Ltd.) for Polycythemia Vera
Rusfertide (formerly PTG-300; Protagonist Therapeutics Inc./Takeda Pharmaceutical Co. Ltd.) is a subcutaneously injected hepcidin mimetic for the treatment of erythrocytosis associated with polycythemia vera.
Varipulse Pulsed Field Ablation (Biosense Webster Inc.) for Atrial Fibrillation
Varipulse is an investigational pulsed-field ablation system in development for treatment of paroxysmal atrial fibrillation.
Letetresgene Autoleucel (Adaptimmune Therapeutics PLC) for Advanced Synovial Sarcoma
Letetresgene autoleucel is an investigational autologous engineered T-cell therapy for the treatment of HLA-A*02 positive patients with advanced synovial sarcoma expressing the NY-ESO-1 antigen.
ReCET (Endogenex Inc.) Duodenal Mucosal Regeneration for Type 2 Diabetes
ReCET (Endogenex Inc.) is an investigational endoscopic procedure that uses pulsed electric fields to promote duodenal mucosal regeneration for treatment of type 2 diabetes that is poorly controlled with oral medications and/or insulin.
Relyvrio (Sodium Phenylbutyrate/Taurursodiol; Amylyx Pharmaceuticals Inc.) for Amyotrophic Lateral Sclerosis (ALS)
Relyvrio (sodium phenylbutyrate/taurursodiol, formerly AMX0035; Amylyx Pharmaceuticals Inc.) is an oral fixed-dose combination drug indicated for the treatment of amyotrophic lateral sclerosis (ALS) in adults. As of April 4, 2024, Relyvrio has been withdrawn from the market.
TriClip G4 System (Abbott Medical) Transcatheter Tricuspid Valve Repair for Tricuspid Regurgitation
The TriClip G4 System (Abbott Medical) is a transcatheter tricuspid valve repair system indicated for improving quality of life and functional status in patients with symptomatic severe tricuspid regurgitation despite optimal medical therapy, who are at intermediate or greater risk for surgery and in whom transcatheter edge-to-edge valve repair is clinically appropriate and i…
Eko Low Ejection Fraction Tool (ELEFT; Eko Health Inc.) for Artificial Intelligence (AI)-based Stethoscope Detection of Low Ejection Fraction
The Eko Low Ejection Fraction Tool (ELEFT; Eko Health Inc.) is an artificial intelligence (AI)-based algorithm that analyzes single-lead electrocardiogram (ECG) data collected via a compatible Eko digital stethoscope as an aid in the detection of low left ventricular ejection fraction in adult patients during a routine physical examination. It is not intended as a sole means …
Remestemcel-L (Mesoblast Ltd.) for Acute Graft-Versus-Host Disease
Remestemcel-L is an investigational allogeneic liquid cell suspension of ex-vivo cultured adult human mesenchymal stem cells derived from healthy donor bone marrow proposed for the treatment of pediatric patients with acute graft-versus-host-disease (aGVHD) refractory to standard first-line steroid therapy.