Clinical uncertainty, price pushes UK's NICE to spurn Akcea's Waylivra
Echoing some of the concerns the FDA underscored before handing its rejection, the UK’s cost-effectiveness watchdog NICE has also forsaken Akcea’s drug, volanesorsen.
On Friday, NICE issued a draft recommendation rejecting the familial chylomicronaemia syndrome (FCS) drug, citing a myriad of reasons including uncertainty underpinning its clinical evidence as well as price. The drug is engineered to work by diminishing the production of ApoC-III, a protein that regulates plasma triglycerides. FCS is caused by insufficient or impaired function of an enzyme responsible for breaking down triglycerides. It affects between 55 and 110 people in England.
The drug, branded Waylivra, was granted conditional approval in Europe last May. The FDA rejected the drug in August 2018, despite a positive 12-8 recommendation by an expert advisory panel. Experts within and outside the agency did, however, spotlight the drug’s link with sudden and unexpected drops in platelet counts as well as Akcea’s decision to switch the dosing regimen and platelet monitoring strategy, which were never tested in clinical trials.
NICE, in its rebuff, also took issue with the different dose. In addition, the institute highlighted the uncertainty of the drug’s long term effect, acknowledging that it does show a short term benefit. NICE also said aspects of the company’s economic modeling were “highly uncertain,” and that the drug’s cost-effectiveness estimates are much higher than what it considers acceptable.
The list price of volanesorsen is £11,394 per single-use 285 mg syringe. It is given as a once-weekly self-administered injection (decreasing to once-fortnightly after 3-months).
The NICE decision is open for public consultation until 24 January 2020.
Akcea expressed its disappointment at NICE’s decision but pledged to keep working with the institute to ensure Waylivra is endorsed.
The company, along with sister drugmaker Ionis, develops antisense drugs that are engineered to interrupt the production of disease-causing proteins by targeting the specific corresponding messenger RNA (mRNA) that encodes that protein, thereby manipulating protein production.
In an unexpected twist in September, a trifecta of senior executives running Akcea — CEO Paula Soteropoulos, president Sarah Boyce and COO Jeffrey Goldberg — left the Boston drugmaker in one fell swoop.
The board of the Ionis majority-owned spinoff swooped in to place Damien McDevitt — the chief business officer of the California company — in the interim chief spot. He will now be joined by former GSK executive Alex Howarth who will take on the role of COO.
When the unceremonious departures were announced earlier this year, no explanation was initially provided by the company. Eventually, it emerged that the exits were unrelated to the launches of Akcea’s two drugs, Tegsedi and Waylivra, but were in fact driven by the need to go shopping for deals to expand its arsenal of drugs-in-development as the next big trial readouts are expected by 2021.