Plano-based Reata Pharmaceuticals announced Tuesday that Skyclarys, the first Food and Drug Administration-approved treatment for the ultra-rare neuromuscular disease Friedreich’s ataxia, is now available to patients.
The final FDA go-ahead follows a brief stall due to a production process-related impurity after its initial spring approval. Despite the hiccup, the drug is launching more than a month ahead of schedule.
Freidreich’s ataxia is an inherited neurodegenerative disorder that affects approximately 5,000 patients in the U.S. It is usually diagnosed in adolescence and causes progressive loss of muscle strength and coordination that typically results in wheelchair confinement and can shorten life.