Omaveloxolone at a glance
Medicine class
Nrf2 pathway activator
Licensed use
Friedreich’s ataxia from age 16 years
Form
Oral hard capsules
Food
Take on an empty stomach; avoid grapefruit
What is Omaveloxolone used for?
Its UK-licensed use is:
Treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older.
How does Omaveloxolone work?
Omaveloxolone binds to Keap1 and activates Nrf2, increasing the expression of genes involved in cellular defence against oxidative stress. Nrf2 activity is reduced in cells from people with Friedreich’s ataxia, although the precise way activation produces clinical benefit is not fully understood.
Omaveloxolone preparations and strengths
Capsule
Route: Oral
Strengths: 50 mg
How to take Omaveloxolone
Follow the specialist’s instructions and the pack leaflet.
Take on an empty stomach, at least one hour before or two hours after food.
Swallow capsules whole.
If swallowing is difficult, open the capsules and sprinkle all their contents onto two tablespoons of apple puree. Consume immediately and do not store the mixture.
Avoid grapefruit and grapefruit juice.
If vomiting occurs after taking it, do not take replacement capsules.
Omaveloxolone side effects
Common or expected effects
- Headache
- Nausea, vomiting, diarrhoea or abdominal discomfort
- Reduced appetite or weight loss
- Tiredness
- Sore mouth or throat
- Back pain or muscle spasms
- Rash, itching or hives
- Raised liver enzymes
- Changes in cholesterol or triglycerides
Get urgent medical advice
- Sudden weight gain, peripheral swelling or shortness of breath requires immediate clinical assessment.
- A severe allergic reaction with airway swelling, breathing difficulty, faintness or collapse requires emergency help.
- Marked liver-test abnormalities detected during monitoring require prompt prescriber review.
Omaveloxolone in pregnancy
Omaveloxolone should not be used during pregnancy because human data are unavailable and animal studies found reproductive toxicity. Effective contraception is required before and during treatment. Omaveloxolone can make hormonal contraception less effective, so specialist advice about non-hormonal or additional barrier contraception is important.
Omaveloxolone while breastfeeding
Omaveloxolone should not be used while breastfeeding. It is unknown whether it enters human milk, and a risk to the infant cannot be excluded.
Omaveloxolone interactions
Check all prescribed, over-the-counter and herbal products before treatment.
Strong or moderate CYP3A4 inhibitors, including itraconazole, ketoconazole, clarithromycin, fluconazole, ciprofloxacin, ciclosporin and fluvoxamine
These can increase omaveloxolone exposure and adverse effects; concurrent use should generally be avoided or managed by the specialist.
CYP3A4 inducers, including carbamazepine, phenytoin, phenobarbital, primidone, rifampicin, efavirenz and St John’s wort
These can lower omaveloxolone exposure and reduce its effectiveness, so alternatives should be considered.
Hormonal contraceptives
Omaveloxolone may reduce contraceptive effectiveness; use an alternative or additional non-hormonal method as advised.
Common questions about Omaveloxolone
Answers are fully visible for fast scanning and source review.
What is omaveloxolone used for?
It is licensed in the UK to treat Friedreich’s ataxia in adults and adolescents aged 16 years and older.
How does omaveloxolone work?
It activates the Nrf2 pathway, supporting cellular responses to oxidative stress. Its precise therapeutic mechanism in Friedreich’s ataxia remains uncertain.
Can children take omaveloxolone?
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.