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UK price watchdog rejects Akcea's rare disease drug in draft guidance

The U.K. National Institute for Health and Care Excellence said Akcea Therapeutics Inc.'s volanesorsen was not a cost-effective treatment for patients with familial chylomicronemia syndrome.

Familial chylomicronemia syndrome, or FCS, is a rare genetic disease that prevents the body from breaking down fats, causing abdominal pain and unpredictable and recurrent episodes of acute pancreatitis. The condition is estimated to affect between 55 and 110 people in England.

In a draft guidance, the health cost watchdog said that while the medicine showed some benefit in the short term, there was a lack of evidence in whether it continued to work over a longer period. Furthermore, there was uncertainty about the drug because the company's application described a different dose than the one used in its main clinical trials.

The agency also raised pricing concerns, saying the drug's cost-effectiveness estimates were higher than what it normally considers to be an acceptable use of the country's National Health Service resources.

Volanesorsen, a self-administered injection designed to treat FCS, is also called Waylivra. It was approved across Europe in 2019.

Akcea Therapeutics is a unit of Ionis Pharmaceuticals Inc.