Amvuttra (vutrisiran) is an injectable RNA interference (RNAi) therapeutic agent indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
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.
TissueGene-C for Knee Osteoarthritis
TissueGene-C is an allogeneic cell and gene therapy delivered via intra-articular injection in development for the treatment of knee osteoarthritis.
Radiolabeled Omburtamab for Metastatic Neuroblastoma
Omburtamab is a radiolabeled monoclonal antibody targeting tumor antigen B7-H3 in development for intraventricular radioimmunotherapy for treatment of neuroblastoma central nervous system/leptomeningeal metastases.
Rebyota (Fecal Microbiota, Live-jslm) for Prevention of Recurrent Clostridioides Difficile Infection
Rebyota (fecal microbiota, live-jslm) is a ready-to-use enema product for fecal microbiota transplantation indicated for the prevention of recurrence of Clostridioides difficile infection (CDI) in individuals 18 years of age and older, following antibiotic treatment for recurrent CDI.
FlowSense Device for Evaluation of Ventricular Shunt Function
The Flowsense device is a wireless, noninvasive thermal flow sensor in development for the detection of flow in implanted cerebrospinal fluid shunts in patients with hydrocephalus.
Teplizumab-mzwv (Tzield) for the Delay of Symptomatic Type 1 Diabetes
Teplizumab is an intravenously administered anti-CD3 monoclonal antibody indicated to delay the onset of stage 3 type 1 diabetes (T1D) in adult and pediatric patients aged 8 years and older with stage 2 T1D.
Revita Duodenal Mucosal Resurfacing for Type 2 Diabetes Mellitus
Revita DMR is an investigational endoscopic hydrothermal ablation device for duodenal mucosal resurfacing proposed for treatment of type 2 diabetes that is poorly controlled with oral medications and/or insulin.
CureSight for Amblyopia in Children
CureSight is a software-based prescription digital therapeutic for treatment of amblyopia that utilizes eye tracking technology to improve visual acuity and stereo acuity by training the visual system to use both eyes simultaneously.
AuroLase Therapy for Localized Prostate Cancer
AuroLase is a focal ablation therapy that uses gold nanoparticles and a near-infrared laser to thermally ablate localized prostate cancer.
Skysona (Elivaldogene Autotemcel) for Cerebral Adrenoleukodystrophy
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).