Reata stock soars on FDA approval for ultra-rare disease drug after 15 years
The Skyclarys medication is the first disease-specific treatment for adults with the neuromuscular condition Friedreich’s ataxia.
is bringing a drug to the commercial market after receiving Food and Drug Administration approval for the first disease-specific treatment for an ultra-rare neuromuscular disease. It’s a big win for the company that’s been inching closer to transitioning from clinical stage to one that markets and sells drugs worldwide. Besides Skyclarys, it has also been working onAnalysts at Citi estimate Skyclarys will create a $1 billion-plus revenue stream for Reata. They expect the company’s valuation to rise now that it’s proven it can generate a commercial product.
“In drug development, this is one of those events that’s very rare,” he said. “Drug approvals are fairly rare on their own, but this is a completely novel drug with a novel mechanism.”Huff said the drug works by improving mitochondrial function — or the body’s ability to convert energy from food into a form that cells can use — and may be able to help with a number of neurodegenerative diseases, like Parkinson’s, Huntington’s and potentially Lou Gehrig’s and Alzheimer’s, Huff said.
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