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.
Horizon Scanning
It is a challenge for health plans and clinicians to know what emerging technologies are in the preapproval phase and what their clinical, financial, and risk impacts might be. This is where we come in.The Hayes Horizon Scanning Solution identifies innovative and potentially disruptive health technologies that are in various stages of the regulatory approval process. Our Research Analysts continually investigate new technologies that are on the horizon or in the early stages of clinical investigation, as well as the testing of existing technologies that may be seeking approval for new clinical applications. Hayes Horizon Scanning helps you track emerging technologies before you need to make decisions. Accessed through the Hayes Knowledge Center, Hayes Horizon Scanning addresses the following fundamental questions to help our clients forecast emerging technologies:
- Is this emerging technology new or replacing an existing technology?
- Where is this technology in the FDA regulatory process?
- When will this technology be commercially available in the market?
- What is the projected use, cost, and financial impact of this technology?
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.