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.
Embozene Microspheres are biocompatible, nonresorbable hydrogel particles under investigation for genicular artery embolization (GAE) to treat symptomatic knee osteoarthritis (OA).
Oleogel-S10 is a semisolid gel containing 10% betulin-rich extract from birch bark and 90% refined sunflower oil. Betulin is a naturally occurring triterpene with medicinal properties believed to help modulate the inflammation response and encourage formation of new skin during wound healing. It is in development for the treatment of skin wounds associated with epidermolysis …
Aduhelm (aducanumab) is an intravenously administered amyloid beta-directed antibody for the treatment of Alzheimer disease.
Mavacamten is an investigational oral myosin inhibitor that targets the underlying molecular mechanisms of hypertrophic cardiomyopathy (HCM). It is proposed for the treatment of patients with symptomatic obstructive HCM.
Beta thalassemia is blood disorder caused by sequent variants in the beta-globin (HBB) gene that threaten normal production of hemoglobin. Lentiviral Autologous Beta-Globin Gene Therapy (Proposed Name: Zynteglo, formerly LentiGlobin) aims to reduce or eliminate the need for regular blood transfusions in patients with transfusion-dependent beta-t…
Vosoritide is a longer-acting C-type natriuretic peptide (CNP) analog that modulates activity of the fibroblast growth factor receptor 3 (FGFR3) by inhibiting a signaling pathway that mediates its effects, thereby stimulating endochondrial growth, resulting in enhanced growth velocity. It is indicated to increase linear growth in pediatric patients aged ≥ 5 years with achondr…
Omaveloxolone is an investigational oral drug that activates nuclear factor erythroid 2–related factor 2 (Nrf2). It is proposed for the treatment of Friedreich ataxia (FA), a rare, hereditary, neurodegenerative disorder that is driven by mitochondrial dysfunction and impaired Nrf2 activity. FA typically presents between the ages of 10 and 15 years.
Vutrisiran is an injectable RNA interference (RNAi) therapeutic agent under investigation for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
The iStent infinite is an investigational device designed for use in a stand-alone procedure to reduce elevated intraocular pressure (IOP) in patients with open-angle glaucoma uncontrolled by prior therapy. The device includes 3 heparin-coated titanium stents measuring 1/3 mm in length and width. The stents are preloaded into an auto-injection system that allows the surgeon t…
Livmarli (maralixibat) is an ileal bile acid transporter (IBAT) inhibitor indicated for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 1 year of age and older.