The company has been seeking approval for use of the therapy in adults with severe or moderately severe haemophilia B without a history of factor IX inhibitors.
In its guidance, NICE’s evaluation committee said that while there is clear evidence that the therapy reduces the number of bleeding episodes haemophilia B patients have each year, there is not enough evidence on how well it works in the long term, meaning cost-effectiveness estimates are “uncertain”.
It also cited problems with the indirect comparison between the gene therapy and factor IX replacement therapies…
