Skyclarys (Omaveloxolone): FDA approves first therapy for Friedreich’s Ataxia in patients aged 16 years and older. 

For individuals living with the rare and devastating disease Friedreich’s ataxia (FA), Skyclarys (omaveloxolone) represents a ray of hope. This groundbreaking medication received FDA approval on February 28, 2023, marking a significant milestone in the fight against this debilitating condition.

Manufacturer Detail

Manufactured by Reata Pharmaceuticals, Skyclarys is the first and only drug approved for this indication. It is an oral, once-daily medication that has received orphan drugs, fast track, priority review, and rare pediatric disease designations from the FDA.

Indication Of Skyclarys 

Skyclarys (omaveloxolone) are miscellaneous central nervous system agents that belong to the class known as Nrf2 Activators. Skyclarys is an FDA-approved prescription medicine indicated for the treatment of Friedreich’s ataxia (FA) in patients aged 16 years and older. 

What is Friedreich’s ataxia? 

Friedreich’s ataxia is a rare genetic disorder that affects coordination and movement due to mutations in the frataxin gene. This autosomal recessive condition leads to mitochondrial dysfunction, causing symptoms like balance and coordination difficulties, muscle control issues, fatigue, and, in some cases, heart problems. Symptoms generally emerge in childhood or adolescence and progress over time.

Mechanism

The mechanism of omaveloxolone is not fully understood, but it is known to activate the nuclear factor (erythroid-derived 2)-like 2 (Nrf2) pathway, alleviating the oxidative stress that is thought to interfere with proper mitochondrial function in FA.

Dosage Information 

The recommended dosage for Skyclarys capsule is 150 mg, equivalent to three capsules, administered orally once daily. It is imperative to administer Skyclarys on an empty stomach, allowing at least 1 hour before eating. The capsules should be swallowed whole, and any attempt to open, crush, or chew them should be avoided.

Patients with moderate hepatic impairment are recommended a reduced dosage of 100 mg once daily. In adverse reactions, further dosage reduction to 50 mg once daily may be considered. Skyclarys is contraindicated in patients with severe hepatic impairment.

How To Consume Skyclarys? 

Before starting the treatment with Skyclarys, it is essential to conduct baseline assessments, including alanine aminotransferase (ALT), aspartate aminotransferase (AST), bilirubin, B-type natriuretic peptide (BNP), and lipid parameters. Regular monitoring of these parameters is advised throughout the treatment course.

Follow your healthcare provider’s instructions precisely when taking Skyclarys. Ingest Skyclarys capsules on an empty stomach, ensuring a minimum of 1 hour before eating. Consume the capsules whole do not open, crush, or chew them. If you happen to miss a dose, skip the missed dose and resume your regular schedule the next day. Avoid doubling the next dose or exceeding the prescribed dosage.

Drug Interactions

When considering drug interactions, it is advised to avoid the concurrent use of Skyclarys with moderate or strong CYP3A4 inhibitors. In situations where unavoidable, a cautious approach involves considering a reduction in Skyclarys dosage while closely monitoring its effects. Additionally, it is recommended to steer clear of simultaneous use with moderate or strong CYP3A4 inducers. However, it is important to consult a healthcare professional for information on potential drug interactions.

Usage in Specific Populations

• Pregnancy: Limited data exist on developmental risks with Skyclarys use in pregnant women. Animal studies suggest omaveloxolone administration during pregnancy or pregnancy with lactation may lead to developmental toxicity.
• Lactation: It is unknown whether Skyclarys is present in human milk or its effects on the breastfed infant. Women are advised not to breastfeed during treatment with Skyclarys and for three months after the last dose.
• Pediatric: Skyclarys is indicated for the treatment of FA in patients aged 16 years and older.
• Geriatric: The safety and effectiveness of Skyclarys in geriatric patients have not been established.

Side Effects

The most common side effects of Skyclarys include elevated liver enzymes (AST/ALT), headache, nausea, abdominal pain, fatigue, diarrhea, and musculoskeletal pain.

Other Information

Before starting the Skyclarys treatment, inform your healthcare provider about your medical history, specifically mentioning any liver problems, a history of heart issues (including heart failure), high blood cholesterol levels, and if you are pregnant or planning to become pregnant. 

The impact of Skyclarys on unborn babies is unknown, and if you use hormonal birth control, consider using an additional non-hormonal form while on Skyclarys and for 28 days post-treatment. If you are breastfeeding or planning to breastfeed, it’s unclear whether Skyclarys passes into breast milk, so discuss the optimal feeding approach for your baby with your healthcare provider.

How can I buy Skyclarys Online?

Nowadays, purchasing Skyclarys is a simple process done online, especially for people in the US and Europe. You can contact the SANSFRO team or other respectable companies that specialize in importing drugs from these areas to start this procedure.

To obtain comprehensive details about Skyclarys price in the Indian market and procurement procedures, please contact our committed Patient Support Team at (91) 9315705373 or by email at help@sansfro.com. Our group is committed to offering precise and up-to-date Skyclarys pricing information in the Indian market. Make sure that getting this necessary medication is a smooth process by getting in touch with our reliable services.

Conclusion

Skyclarys (omaveloxolone) is a significant advancement in the treatment of Friedreich’s ataxia, providing a new therapeutic option for patients aged 16 years and older. It is important to note that the use of Skyclarys may be associated with certain side effects, and patients should always consult their healthcare provider for guidance on its use and potential interactions with other medications.

FAQs

1. What is Skyclarys?

Skyclarys is the first and only FDA-approved medication for the treatment of Friedreich’s ataxia (FA). It works by increasing the production of frataxin, a protein crucial for healthy nerve function.

2. Who can take Skyclarys?

Skyclarys is currently approved for adults (16 years and older) with genetically confirmed FA.

3. What are the benefits of taking Skyclarys?

Clinical trials have shown that Skyclarys can slow the progression of FA and improve symptoms such as muscle weakness, coordination problems, and fatigue.

4. What is the storage condition of Skyclarys? 

Store Skyclarys at temperatures between 20°C to 25°C (68°F to 77°F), with excursions allowed between 15°C and 30°C (59°F to 86°F). Keep the Skyclarys away from the pets and children. 

5. Is Skyclarys safe for children under 16 years of age?

The safety and effectiveness of Skyclarys in children younger than 16 years of age have not been established.

6. What should I avoid while taking Skyclarys?

While taking Skyclarys, avoid consuming grapefruit or grapefruit juice as they can alter the levels of Skyclarys in your bloodstream.

Reference 

1. https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/216718Orig1s000lbl.pdf 
2. https://everyone.org/Skyclarys-omaveloxolone 
3. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-treatment-friedreichs-ataxia 
4. https://www.reatapharma.com/investors/news/news-details/2023/Reata-Pharmaceuticals-Announces-FDA-Approval-of-Skyclarys-Omaveloxolone-the-First-and-Only-Drug-Indicated-for-Patients-with-Friedreichs-Ataxia/