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.
Relyvrio (formerly AMX0035) is an oral fixed-dose combination of sodium phenylbutyrate (PB) and taurursodiol (TURSO). PB is a prescription drug used to treat urea cycle disorders and TURSO (also known as tauroursodeoxycholic acid [TUDCA]) is a widely available non-prescription supplement. Relyvrio is indicated for the treatment of amyotrophic lateral sclerosis (ALS) in adults…
AuroLase is a focal ablation therapy that uses gold nanoparticles and a near-infrared laser to thermally ablate localized prostate cancer.
Beremagene geperpavec is an investigational topical gene therapy proposed for the treatment of open wounds in patients with dystrophic epidermolysis bullosa.
Tofersen is an investigational antisense oligonucleotide proposed for the treatment of amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene.
A radiolabeled monoclonal antibody targeting tumor antigen B7-H3 in development for intraventricular radioimmunotherapy for treatment of neuroblastoma central nervous system/leptomeningeal metastases.
RBX2660 is an investigational microbiota-based ready-to-use enema product for fecal microbiota transplantation proposed to treat recurrent Clostridoides difficile infection.
Etranacogene dezaparvovec (formerly AMT-061) is an investigational recombinant gene transfer therapy product designed to help patients with hemophilia B produce sufficient plasma levels of endogenous coagulation factor IX (FIX). The main therapeutic goal is long-term expression of FIX at levels steady enough to eliminate the need for routine prophylactic replacement therapy w…
Tofersen is an investigational antisense oligonucleotide proposed for the treatment of amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene.
Skysona (elivaldogene autotemcel; eli-cel) is lentiviral-based gene therapy indicated to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD).
The Vibrant vibrating capsule is an investigational device designed to alleviate symptoms of chronic idiopathic constipation (CIC) by inducing a bowel movement through mechanical vibration of the colon.