NICE rejects Akcea's Waylivra due to concerns over clinical evidence, high cost

The National Institute for Health and Care Excellence published an evaluation consultation document on Friday recommending against NHS use of Akcea Therapeutics' Waylivra (volanesorsen) for the treatment of familial chylomicronaemia syndrome (FCS). In making its decision, the agency cited "concerns around the clinical evidence and high cost" of the drug, which carries a list price of £11,394 ($14,907) for a single-use syringe.

Commenting on the recommendation, Andy Caldwell, Akcea's manager in the UK and Republic of Ireland, said "we are disappointed with the draft guidance," adding "we will continue to work with NICE" to understand its concerns around Waylivra. In 2018, the FDA rejected approval of the drug, despite an advisory committee previously voting 12-8 in favour of clearance.

Waylivra is an antisense oligonucleotide drug co-developed by Akcea and Ionis Pharmaceuticals to reduce the production of ApoC-III, a protein that regulates plasma triglycerides. The drug is administered once weekly for three months, before being given as a maintenance dose once every two weeks. Waylivra was approved in Europe in May last year as an adjunct to diet in adults with genetically confirmed FCS and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate.

Clearance was based on findings from the Phase III APPROACH and COMPASS trials, with results from the former showing that Waylivra was associated with statistically significant and clinically meaningful reductions in triglycerides over the study period. In its appraisal, NICE said that while the therapy showed "some short-term benefits… it is uncertain whether this is maintained in the longer term." The agency also raised concerns as the licensed dose was not used in clinical trials.

The recommendation by NICE is open for public consultation until January 27.

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