The FDA quashed Akcea Therapeutics' hopes for Waylivra in the U.S. in 2018, at least temporarily, but the rare disease med did win a European nod last year. Now, though, it's running into reimbursement barriers in one key trans-Atlantic market—England.
In draft guidance, the country's cost watchdogs rejected the drug for familial chylomicronaemia syndrome, citing a too-high price, uncertainty about its approved dose and worries about long-term effectiveness.
The National Institute for Health and Care Excellence (NICE) said it found cost-effectiveness estimates for Waylivra “much higher than what NICE considers acceptable for highly specialized technologies.” It’s asking Akcea to shed some light on its data and clarify other points in its reimbursement application.
The drug costs £11,394 per 285 mg syringe before any discounts. Akcea did offer up a price break, but apparently it wasn't significant enough to sway the assessors at NICE.
Familial chylomicronaemia syndrome, or FCS, affects about one or two people per million, or about 55 to 110 people in all of England. It causes very high triglyceride levels in patients’ blood and can trigger abdominal pain, liver and spleen enlargement, fatigue and other symptoms.
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The company is “disappointed” with the draft guidance, said Andy Caldwell, country manager for the U.K. and Ireland. Akcea will “continue to work with NICE so this much-needed treatment can be provided to the patients that need it the most," Caldwell said in a statement.
Patients in England and Wales taking the med through Akcea's early access program can continue to receive treatment, he added.
Akcea won European approval for the drug in May 2019 after an August 2018 rejection at the U.S. FDA. In the wake of the European nod, the drugmaker promoted a commercial chief from within to focus on the Waylivra rollout and other opportunities. The company has since published data on the drug and says it's working with the FDA on a path forward.