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.

Etranacogene Dezaparvovec (Hemgenix) for Hemophilia B

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.

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.

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.