FDA finally approves Reata's rare genetic disease drug
After raising serious efficacy questions, the FDA this evening approved Reata Pharmaceuticals’ drug for a rare genetic disease known as Friedreich’s ataxia, which affects the body’s nerves, for those 16 years old and older.
While not a cure, the drug known as Skyclarys (omaveloxolone), which will cost $370,000 annually, may slow the progression of this debilitating disease that begins in late childhood and affects about 6,000 Americans and 22,000 individuals globally. The FDA’s approval is based on a positive pivotal trial, showing omaveloxolone beat placebo in terms of a change in what’s known as an mFARS score — a physician-assessed neurological rating scale used to measure FA disease progression.
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