Etranacogene dezaparvovec (Hemgenix; formerly AMT-061) is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with hemophilia B (congenital factor IX deficiency) who currently use factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.
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.
Zavegepant (Zavzpret) for Acute Treatment of Migraine
Zavegepant is an intranasal novel small-molecule, calcitonin gene-related peptide (CGRP) receptor antagonist indicated for the acute treatment of migraine with or without aura in adults.
Omecamtiv Mecarbil for Heart Failure With Reduced Ejection Fraction (HFrEF)
Omecamtiv mecarbil is a first-in-class oral cardiac myosin activator under investigation for the treatment of heart failure with reduced ejection fraction (HFrEF).
Trofinetide (Daybue) for Rett Syndrome
Trofinetide (Daybue) is an oral synthetic glypromate analog indicated for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older.
Omaveloxolone (Skyclarys) for Friedreich Ataxia
Omaveloxolone is an oral drug that activates nuclear factor erythroid 2-related factor 2 (Nrf2). It is intended for 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.
Pegcetacoplan (Syfovre) for Geographic Atrophy
Pegcetacoplan (Syfovre; Apellis Pharmaceuticals Inc.) is a complement inhibitor indicated for the treatment of geographic atrophy secondary to age-related macular degeneration.
AGN1 Local Osteo-Enhancement Procedure (LOEP) Kit
The AGN1 Local Osteo-Enhancement Procedure (LOEP) Kit is a resorbable calcium-based implant to improve femoral bone density and strength in postmenopausal patients with osteoporosis.
TIVUS System (SoniVie) for Pulmonary Hypertension
The Therapeutic Intravascular Ultrasound System (TIVUS) is an intravascular ultrasound system under investigation for catheter-based pulmonary artery denervation to treat adult patients with pulmonary arterial hypertension.
Tofersen for Amyotrophic Lateral Sclerosis
Tofersen is an investigational antisense oligonucleotide proposed for the treatment of amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene.
Botaretigene Sparoparvovec for X-Linked Retinitis Pigmentosa
Botaretigene sparoparvovec is an investigational gene replacement therapy for the treatment of X-linked retinitis pigmentosa due to mutations in the RPGR gene. It is administered by subretinal injection.