FDA Approves Skyclarys for Friedreich's Ataxia in Adults and Adolescents Aged 16 Years and Older

Not reported in label.

Skyclarys is indicated for the treatment of Friedreich's ataxia in adults and adolescents aged 16 years and older.

The recommended dosage is 150 mg (3 capsules) taken orally once daily on an empty stomach, at least 1 hour before or 2 hours after eating. Capsules should be swallowed whole or opened and sprinkled onto applesauce; do not crush, chew, or mix with milk or orange juice. For moderate hepatic impairment (Child-Pugh Class B), reduce to 100 mg once daily, with possible further reduction to 50 mg if adverse reactions occur; avoid use in severe hepatic impairment (Child-Pugh Class C). With strong CYP3A4 inhibitors, avoid concomitant use or reduce to 50 mg once daily; with moderate CYP3A4 inhibitors, avoid or reduce to 100 mg, then 50 mg if adverse reactions occur; avoid with strong or moderate CYP3A4 inducers. Obtain ALT, AST, bilirubin, BNP, and lipid parameters before initiating and during treatment.

The efficacy was evaluated in a 48-week, randomized, double-blind, placebo-controlled study (Study 1; NCT02255435) in patients aged 16 to 40 years with Friedreich's Ataxia. A total of 103 patients were randomized to Skyclarys 150 mg once daily (n=51) or placebo (n=52). The primary analysis in the Full Analysis Population without pes cavus (n=82) showed a statistically significant treatment difference in mFARS score change from baseline at Week 48: Skyclarys LS mean change -1.56 vs. placebo 0.85, treatment difference -2.41 (95% CI -4.32, -0.51), p=0.0138. Baseline mean mFARS scores were 40.95 (SD 10.39) for Skyclarys and 38.78 (SD 11.03) for placebo.

Not reported in label.

Not reported in label.