Friedreich ataxia (FA) is a rare inherited disease that affects the nervous system. It is usually diagnosed in childhood and affects about 5,000 people in the U.S. It causes a loss of muscle control. Symptoms get worse as the disease progresses. People with FA may have the following symptoms:
- Hearing loss
- Vision loss
- Trouble walking
- Tiredness
- Slow or slurred speech
- Weakness, numbness, and pain in arms and legs
- Chest pain or fast heartbeat
- Shortness of breath
Skyclarys was approved in 2023 as the first treatment for FA. Skyclarys contains the active ingredient omaveloxolone. Skyclarys does not cure FA but may help to reduce symptoms and delay the worsening of the disease.
According to studies, Skyclarys can improve neurological functioning and slow disease progression. Below are common questions and answers about Skyclarys and whether it may be a good treatment option for you.
How Does It Work?
Skyclarys is a nuclear factor (erythroid-derived 2) like 2 (Nrf2) activator. Nrf2 is a protein that helps protect cells. The Nrf2 pathway, a defense system for your body, does not work as well in FA, which causes stress, inflammation, and cell damage. Skyclarys activates the Nrf2 pathway, which helps to restore and improve mitochondrial cell function. It also works to reduce stress and inflammation.
How Do I Take It?
Skyclarys is a capsule that is taken by mouth. You will take three 50-milligram capsules at the same time every day. It should be taken on an empty stomach, at least 1 hour before eating.
Skyclarys should be swallowed whole. It should not be crushed or chewed. If you have trouble swallowing Skyclarys, the capsules can be opened and the contents sprinkled on 2 tablespoons of applesauce. The mixture should be stirred and swallowed right away.
The capsule contents should not be mixed with milk or orange juice. Opened capsules cannot be given by an enteral feeding tube.
If you miss a dose, you should skip that dose and take your next dose at the regular time. Do not take extra or double doses.
How Was It Studied?
The safety and efficacy of Skyclarys in treating FA was studied. Eighty-two people completed the 48-week study.
People in the study received either 150 milligrams of Skyclarys or a placebo, which contains no medicine. The study included people ages 16 to 40 years, with an average age of 24. About 97% of people in the study were White, and 3% were unknown races. Forty-six percent of the people studied were female.
Treatment success was measured as a change from baseline in the modified Friedreich’s Ataxia Rating Scale (mFARS) score. The mFARS is an assessment of neurological function and tests coordination and stability. The mFARS assesses the following criteria.
- Speech
- Upper limb (arm) coordination
- Lower limb (leg) coordination
- Upright stability (sitting posture, standing posture, and walking)
Scores range from 0 to 99, with a lower score meaning less physical impairment. At the beginning of the study, scores ranged from 20 to 80, with an average mFARS score of 40.
A long-term extension study was completed after the initial 48 weeks. All the people in this part of the study took Skyclarys.
What Are the Benefits Based on Studies?
- Improved neurological function. After 48 weeks, Skyclarys improved neurological function, compared to the placebo. People taking the placebo had their mFARS scores worsen by 0.85 points. Those taking Skyclarys had their mFARS score improve by 1.56 points.
- Persistent benefit. In the 3-year extension study, those who took Skyclarys had a continual slowing of disease progression. People who initially took the placebo and then started on Skyclarys showed a benefit in disease progression. But they could not match the results of those who started treatment earlier.
A natural history study for FA was started in 2003. It followed over 1,250 people in 14 locations. It measured the symptoms of the disease as well as the disease progression. It also led to the development of the mFARS.
An analysis compared the long-term use of Skyclarys to the natural history study, in which people did not receive medicine. This was just an exploratory analysis and was not a formal clinical study.
After 3 years of treatment with Skyclarys, mFARS scores were lower than those from the natural history study, which did not give treatment. Research has shown that mFARS scores usually increase by about 2 points per year. Those treated with Skyclarys had their score increase by 3 points over 3 years. Those in the natural history study, who took no medicine, had their scores increase by 6.61 points in 3 years. Skyclarys slowed the mFARS progression by 55% in this analysis.
Is It Right for Me?
Skyclarys may be right for you if you have Friedreich ataxia and are over the age of 16. Talk with your health care provider for more information.
Skyclarys was not studied in people with certain health conditions because it can increase the risk of serious side effects. You should not take Skyclarys if you have severe liver disease.
How Long Does It Take to Work?
In studies, some people saw an improvement in functioning at 12 weeks. For some people, an improvement in symptoms may take longer. Talk with your health care provider if you do not notice an improvement in 12 weeks.
How Can I Prevent and Manage Side Effects?
The most common side effects of Skyclarys include the following.
- Increased liver enzymes
- Headache
- Nausea
- Belly pain
- Fatigue
- Diarrhea
Increased liver enzymes were the most common side effect seen in studies. This could mean that your liver is injured or inflamed. Your health care provider will regularly check your liver enzymes with bloodwork. Tell your health care provider if you develop any of the following symptoms of liver damage:
- Nausea
- Vomiting
- Yellowing of the skin or eyes
- Pain in your right upper stomach
Maintaining healthy lifestyle habits can help prevent headaches. Be sure you are eating a healthy diet, staying hydrated, and getting enough sleep. If you develop headaches, you can try resting in a cool, dark room, applying cool compresses, meditating, or focusing on breathing exercises.
Nausea, belly pain, and diarrhea frequently happen with Skyclarys. Eating smaller, more frequent meals may help. Also, try eating slowly and chewing your food well. Avoid caffeine, alcohol, and carbonated beverages.
If you develop diarrhea, make sure you are staying hydrated. Water, broths, coconut water, sports drinks, and rehydration solutions are good options to replenish lost fluids. You should avoid dairy, spicy, fatty, and sugary foods until you are feeling better. Simplify your diet to include bland foods, such as rice, potatoes, toast, and bananas. Your pharmacist can recommend an OTC anti-diarrheal medicine such as Imodium. Talk with your health care provider if your diarrhea becomes severe.
Skyclarys commonly causes fatigue. You should not drive or do other activities that require alertness or coordination until you know how Skyclarys affects you.
These are not all of the possible side effects. Talk with your health care provider if you are having symptoms that bother you. In the U.S., you can report side effects to the FDA at www.fda.gov/medwatch or by calling 800-FDA-1088 (800-332-1088). In Canada, you can report side effects to Health Canada at www.health.gc.ca/medeffect or by calling 866-234-2345.
What Type of Drug Interactions Can Happen?
Many medicines can interact with Skyclarys. These medicines can affect the way Skyclarys works and vice versa. This can also increase your risk of side effects. Some medicines are metabolized by an enzyme called CYP3A4. If you take these together, your health care provider may have to change your dosage or change your medicine. Ask your pharmacist or other health care provider if any of your medicines can cause this interaction.
Skyclarys can reduce the effect of hormonal birth control. This can increase your chances of getting pregnant. Non-hormonal birth control, such as condoms and diaphragms, should be used instead of, or along with, hormonal birth control while taking Skyclarys and for 28 days after stopping.
Grapefruit and grapefruit juice should be avoided while taking Skyclarys. Grapefruit can increase the effect of Skyclarys and cause an increase in side effects.
Tell your pharmacist and other health care providers about all prescription drugs, over-the-counter (OTC) medications, vitamins/minerals, herbal products, and supplements you take or have recently taken. This will help them find out if there are any interactions with Skyclarys.
What Do I Need to Do to Get It?
Skyclarys requires a prescription from a health care provider. Your neurologist can prescribe Skyclarys, and it may require a prior authorization from your insurance. Your health care provider will work with your insurance to get the medicine approved.
Skyclarys is a specialty medicine that is only available from specialty pharmacies. The specialty pharmacy will coordinate the delivery of your prescription. It is not available at retail pharmacies.
Is There Any Cost Assistance Available?
Assistance may be available to help you afford your medicine. Eligibility depends on your current prescription and medical coverage. Visit https://www.biogenreach.com/patient/#affordabilityOptions or call 844-987-3224 for more information.
