FDA rejects Akcea rare-disease drug that uses gene-blocking technology

The Food and Drug Administration on Monday rejected Akcea Therapeutics’ application to market Waylivra, its drug for familial chylomicronemia syndrome, in the United States.

Akcea didn’t give a reason for the rejection. Waylivra, also known as volanesorsen, would have been Akcea’s first drug in the U.S. market. The condition it treats, FCS, leads to a buildup of fat in organs.

The company sells Tegsedi, a drug based on the same technology, in Europe to treat hereditary transthyretin amyloidosis, and is still seeking FDA approval for that drug.

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