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UK regulator grants early patient access to Akcea's rare disease drug


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UK regulator grants early patient access to Akcea's rare disease drug

Akcea Therapeutics Inc. said a U.K. regulator granted early patient access to its drug volanesorsen for familial chylomicronemia syndrome, a rare genetic disease.

The medicine received a positive scientific opinion from the U.K's Medicines and Healthcare Products Regulatory Agency for treating familial chylomicronemia syndrome.

The U.K. regulator granted the positive opinion through the early access to medicines scheme, a program designed to give patients with life threatening or seriously debilitating conditions access to treatments that have not yet received marketing authorization from the European Commission.

Familial chylomicronemia syndrome is a rare genetic condition that prevents the body from breaking down fats, causing abdominal pain with a high risk of recurrent, potentially fatal, acute pancreatitis.

The positive opinion was granted to Akcea unit Akcea Therapeutics U.K. Ltd.

Akcea Therapeutics, a unit of Ionis Pharmaceuticals Inc., developed volanesorsen using Ionis' proprietary antisense technology for treating rare metabolic disorders. Akcea recently acquired the rights to Ionis' inotersen and AKCEA-TTR-L drugs indicated to treat forms of transthyretin amyloidosis, a progressive, sometimes fatal, genetic disease.