Omaveloxolone

Pronunciation: oh-ma-vel-OX-oh-lone
Generic name: omaveloxolone
Brand name: Skyclarys
Dosage form: oral capsule (50 mg)
Drug class: Miscellaneous central nervous system agents

Medically reviewed by Carmen Pope, BPharm. Last updated on Dec 4, 2024.

What is omaveloxolone?

Omaveloxolone (brand name Skyclarys) is used for Friedreich's ataxia, to help slow the loss of strength and coordination and slow Friedreich's ataxia (FA) disease progression.

Omaveloxolone's mechanism of action involves activating the Nrf2 pathway to reduce inflammation, improve neurological function, and slow disease progression, although the exact way it works is unknown. In Friedreich's ataxia, a pathway called the Nrf2 pathway is impaired, and Nrf2 activity is lower, resulting in nerve damage. 

Omaveloxolone gained FDA approval on February 28, 2023, under the brand name Skyclarys for Friedreich's ataxia in adults and adolescents 16 years and older. There is no omaveloxolone generic.

Omaveloxolone capsules are taken once a day.

What is Friedreich's ataxia?

Friedreich's ataxia is a rare, inherited disease that causes progressive damage to the nervous system which causes poor muscle control and movement problems over time. This can affect walking and balance, hand coordination, speech, swallowing, eye movements, and eventually may result in patients requiring a wheelchair. FA may also affect the heart muscle and cause a type of heart disease called cardiomyopathy.

FA is caused by a faulty gene, called the frataxin gene (or FXN) which results in damage to nerve fibers in the spinal cord and peripheral nerves.

Omaveloxolone side effects

The most common omaveloxolone side effects are nausea, stomach pain, diarrhea, abnormal lab tests including liver enzymes, headache, tiredness, or muscle pain.

Serious omaveloxolone side effects

Omaveloxolone may cause the following serious side effects. 

Increase in blood liver enzymes. Some people taking omaveloxolone have had an increase in the level of liver enzymes in their blood. Your healthcare provider will do liver function tests before you start taking omaveloxolone every month for the first 3 months after starting your treatment with omaveloxolone during certain times as needed while taking omaveloxolone.

If your liver enzymes increase, your healthcare provider may change your dose during treatment, stop treatment for some time, or completely stop treatment with omaveloxolone.

Increase in a blood protein called B-Type Natriuretic Peptide (BNP). BNP tells how well your heart is working. Your healthcare provider will check your BNP levels before your treatment with omaveloxolone. Tell your healthcare provider if you have signs and symptoms of your heart not working well such as too much fluid in your body (fluid overload). Signs and symptoms may include:

• sudden weight gain (3 pounds or more of weight gain in 1 day, or 5 pounds or more of weight gain in 1 week)
• swelling in your arms, hands, legs, or feet (peripheral edema)
• fast heartbeat (palpitations)
• shortness of breath.

If you have symptoms of fluid overload that are considered side effects of omaveloxolone, your healthcare provider may stop treatment with omaveloxolone.

Changes in cholesterol levels. Increases in low-density lipoprotein cholesterol (LDL-C) or bad cholesterol and decreases in high-density lipoprotein cholesterol (HDL-C) or good cholesterol have happened during treatment with omaveloxolone. Your healthcare provider will check your cholesterol levels before and during your treatment with omaveloxolone.

Allergic reactions. Omaveloxolone may cause an allergic reaction. Get emergency medical help if you have signs of an allergic reaction, such as hives, difficulty breathing, or swelling of your face, lips, tongue, or throat.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.

Before taking omaveloxolone

Tell your doctor if you have or have ever had:

• heart failure or other heart problems;
• high cholesterol; or
• liver disease.

Tell your doctor if you are pregnant, planning to become pregnant, or breastfeeding.

Pregnancy

It is not known if omaveloxolone will harm an unborn baby.

Omaveloxolone can make hormonal contraceptives less effective. If you use hormonal birth control, ask your doctor about alternative birth control options such as a condom or non-hormonal intrauterine device while using omaveloxolone and for at least 28 days after your last dose.

Breastfeeding

It is not known if omaveloxolone passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby if you take omaveloxolone.

How should I take omaveloxolone?

Take omaveloxolone exactly as your healthcare provider tells you to take it.

Omaveloxolone is taken 1 time a day.

• Take omaveloxolone on an empty stomach, at least 1 hour before a meal.
• Swallow the capsules whole and do not crush or chew.

If you or the person you are caring for is unable to swallow an omaveloxolone capsule whole, the capsules may be opened and the entire contents of both halves sprinkled onto 2 tablespoonfuls (30 mL) of applesauce.

• Stir the mixture.
• Swallow all the mixture of medicine and applesauce right away. Do not store the mixture of medicine and applesauce to use at a later time.
• The contents of the omaveloxolone capsules should not be mixed with milk or orange juice.
• Do not administer omaveloxolone by an enteral feeding tube.

Omaveloxolone dosing information

Usual Adult and Adolescent (aged 16 and older) Omaveloxolone Dose for Friedreich's Ataxia: omaveloxolone 150 mg (3 capsules) 1 time daily on an empty stomach. 

Available as: omaveloxolone 50mg capsules 

Related/similar drugs

Skyclarys, omaveloxolone

What happens if I miss a dose?

Skip the missed dose and use your next dose at the regular time. Do not use two doses at one time.

What happens if I overdose?

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

What should I avoid while taking this medicine?

Do not drink grapefruit juice or eat grapefruit. These may change the amount of omaveloxolone in your blood.

What other drugs will affect omaveloxolone?

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements such as St. John’s Wort.

Taking omaveloxolone with other medicines can cause serious side effects.

Omaveloxolone may affect the way other medicines work, and other medicines may affect how omaveloxolone works.

Omaveloxolone is known to interact with moderate or strong CYP3A4 inhibitors including:

• antifungal medicines (itraconazole, ketoconazole, posaconazole, voriconazole)
• macrolide antibiotics (erythromycin, clarithromycin)
• antiretrovirals (atazanavir, darunavir, indinavir, nelfinavir, ritonavir, saquinavir, cobicistat)
• heart or blood pressure medicines (diltiazem, verapamil)
• nefazodone

Know the medicines you take. Keep a list of them to show to your healthcare provider and pharmacist when you get a new medicine.

Omaveloxolone Prescribing Information 

Review the Skyclarys (omaveloxolone) Prescribing Information for more detailed information about this medicine. Discuss any medical questions you have with your doctor or other health care provider. This is not all the information you need to know about this medicine for safe and effective use, and it does not take the place of talking to your doctor about your treatment.

Storage

• Store at room temperature between 68°F to 77°F (20°C to 25°C)
• Keep all medicines out of the reach of children and pets.

Ingredients 

Omaveloxolone is only available under the brand name Skyclarys.

Active ingredient: omaveloxolone
Inactive ingredients: croscarmellose sodium, magnesium stearate, pregelatinized starch, and silicified microcrystalline cellulose. The hard capsule shells contain FD&C Blue #1, ferric oxide yellow, hypromellose, titanium dioxide, and white edible ink.

Who makes omaveloxolone

Reata Pharmaceuticals, Inc., manufactures omaveloxolone.

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

Medical Disclaimer