Etravirine is an anti-viral medication approved by the U.S. Food and Drug Administration (FDA) to treat human immunodeficiency virus (HIV) infections.

A recent screening study of 853 FDA-approved medications found that etravirine can significantly enhance frataxin (FXN) levels and confer resistance to oxidative stress in cells derived from Friedreich’s ataxia (FA) patients.

Fratagene Therapeutics currently holds a U.S. patent on the use of etravirine as a therapeutic option for FA.

How does etravirine work?

FA is a rare, genetic, and progressive neuromuscular disorder, and is caused by mutations in the FXN gene.

The FXN gene encodes for a protein called frataxin found in energy-producing structures within cells called mitochondria. Researchers do not fully understand the role of frataxin in the mitochondria. However, they think that it helps assemble clusters of iron and sulfur molecules, which are critical for the function of many proteins, including proteins that cells need for energy production. Mutated forms of the FXN gene greatly reduce the production of the frataxin protein and therefore lead to mitochondrial dysfunction.

Researchers believe that etravirine increases the production of frataxin in the cells by increasing the “translation” of the FXN messenger RNA (mRNA), an intermediary molecule between DNA and protein. Because etravirine does not increase the production of the FXN mRNA itself, any potential issues that could arise from the over-activation of the FXN gene are avoided.

Etravirine in clinical trials

A Phase 2 open-label pilot trial (NCT04273165) will evaluate the safety and effectiveness of etravirine tablets in 30 FA patients, ages 10 to 40. Participants will receive a dose of 200 mg or 400 mg of etravirine daily for four months. Apart from assessing the safety and tolerability of the treatment, the study will also determine the severity of ataxia using the scale for the assessment and rating of ataxia (SARA), and measure frataxin protein levels in immune cells and heart wall thickness using echocardiography. It will assess the patients’ quality of life using the SF-36 health survey.

The IRCCS Eugenio Medea Scientific Institute will conduct the trial in collaboration with the University of Rome Tor Vergata in Italy. The trial is expected to start in March 2020, and be completed by March 2022.

Other information

According to researchers, etravirine shows an excellent safety profile but no information is available about its potential efficacy. Although etravirine showed good outcomes in patient-derived FA cells, researchers have not been yet tested its efficacy in nerve and heart cells. There is also no experimental data for etravirine activity from animal models of the disease.


Last updated: March 8, 2020


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