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FDA Approves Oral Tx for Rare Condition – Skyclarys

This week the FDA approved a new ORAL specialty drug, Skyclarys (omaveloxolone) from Reata Pharmaceutical, indicated for the treatment of Friedreich’s Ataxia in adults and adolescents aged 16 years and older. Friedreich’s Ataxia is a rare condition that affects about 5,000 patients in the United States. This oral med is administered once daily.

The company was granted orphan drug, fast track and rare pediatric disease designations from the FDA. With this approval, the agency granted a rare pediatric disease priority review voucher.

Friedreich’s ataxia causes progressive damage to the spinal cord, peripheral nerves, and the brain, resulting in uncoordinated muscle movement, poor balance, difficulty walking, changes in speech and swallowing, and a shortened lifespan. The condition can also cause heart disease. This disease tends to develop in children and teenagers and gradually worsens over time.

Reata confirmed that he annual WAC cost for Skyclarys will be $370,000.

Given its cost and the relatively small patient base, it is likely that Skyclarys will launch through limited distribution.

CLICK HERE to access prescribing information 


FDA approves first treatment for Friedreich’s ataxia

FDA has approved Skyclarys (omaveloxolone) as the first treatment for Friedreich’s ataxia, a rare, inherited, degenerative disease that damages the nervous system, characterized by impaired coordination and walking.

Patients take Skyclarys capsules orally without food once a day at a recommended dosage of 150 mg.

The efficacy and safety of Skyclarys to treat Friedreich’s ataxia was evaluated in a 48-week randomized, placebo-controlled, and double-blind study [Study 1 (NCT02255435)] and an open-label extension.

Study 1 enrolled 103 individuals with Friedreich’s ataxia who received placebo (52 individuals) or Skyclarys 150 mg (51 individuals) for 48 weeks. Of the research participants, 53% were male, 97% were white, and the mean age was 24 years at study entry. Nine (18%) patients were younger than age 18.

The primary objective was to evaluate the change in the modified Friedreich’s Ataxia Rating Scale (mFARS) score compared to placebo at week 48. The mFARS is a clinical assessment that measures disease progression, namely swallowing and speech (bulbar), upper limb coordination, lower limb coordination, and upright stability. Individuals receiving Skyclarys performed better on the mFARS than people receiving placebo.

In a post hoc analysis, individuals who continued treatment with Skyclarys in an open-label extension for up to three years performed better on the mFARS compared to a matched set of untreated patients from a natural history study.

Safety Information

The most common side effects of Skyclarys were an increase in alanine transaminase and an increase of aspartate aminotransferase, which can be signs of liver damage, headache, nausea, abdominal pain, fatigue, diarrhea and musculoskeletal pain.

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