The UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved Omaveloxolone (Skyclarys) as the first-ever treatment for Friedreich’s ataxia in patients aged 16 and older, a major breakthrough for those affected by this rare and debilitating condition.

Friedreich’s ataxia is the most common form of inherited ataxia, estimated to affect at least 1 in 50,000 people. It causes progressive damage to the nervous system, leading to worsening balance, coordination, and mobility over time.

Taken as an oral capsule, Omaveloxolone works by activating Nrf2, a protein that helps the body defend against oxidative stress—an imbalance that contributes to the nerve damage seen in this condition. People with Friedreich’s ataxia typically have reduced levels of this protein.

The approval follows a 48-week clinical trial involving 103 patients aged 16–40. Those treated with Omaveloxolone showed statistically significant improvements in neurological function compared to those who received a placebo.

For the full list of all side effects reported with this medicine, see Section 4 of the Patient Information Leaflet or the Summary of Product Characteristics (SmPC) available on the MHRA’s website. Anyone who suspects they are having a side effect from this medicine should talk to their doctor, pharmacist or nurse and report it directly to the MHRA Yellow Card scheme, either through the website (https://yellowcard.mhra.gov.uk/) or by searching the Google Play or Apple App stores for MHRA Yellow Card.   

Learn more: MHRA approves first UK treatment for Friedreich’s ataxia, omaveloxolone - GOV.UK