NICE Shoots Down Akcea’s Waylivra

NICE has recommended against NHS use of Akcea Therapeutics’ Waylivra (volanesorsen) for treating the ultra-rare disease familial chylomicronaemia syndrome (FCS).

The agency cited concerns over the clinical evidence and the drug’s high cost. Waylivra has a list price of £11,394 ($14,907) for a single-use syringe.

Waylivra received EU approval in May 2019 as an adjunct to diet in adults with genetically confirmed FCS that are at high risk for pancreatitis and have had inadequate responses to diet and triglyceride lowering therapy.