The National Institute for Health and Care Excellence (NICE) has recommended CSL Behring’s haemophilia B gene therapy Hemgenix (etranacogene dezaparvovec) for immediate reimbursement by NHS England.
The agency’s positive opinion, which specifically applies to adults with severe or moderately severe haemophilia B without a history of factor IX (FIX) inhibitors, reverses its previous decision to reject the therapy.
Affecting more than 2,000 people in the UK, haemophilia B is a genetic bleeding disorder resulting from missing or insufficient levels of clotting FIX.
Patients with severe cases of the disease currently require lifelong treatment with intravenous FIX, which can still leave them vulnerable to breakthrough bleeds and pain in the days before infusions.
CSL’s Hemgenix addresses the underlying genetic cause of haemophilia B by enabling the body to continuously produce FIX.
NICE’s updated decision is supported by results from the late-stage HOPE-B trial, in which a single infusion of Hemgenix demonstrated significant increases in mean FIX activity levels of 36.9% at 18 months.
These increases were sustained at 36.7% at 24 months and 38.6% at 36 months and led to an adjusted annualised bleed rate reduction of 64% at 24 and 36 months post-dose compared to the six-month lead-in period.
Additionally, 96% of Hemgenix-treated patients discontinued routine FIX prophylaxis, meaning they no longer required regular infusions for up to 36 months.
In its draft guidance in August last year, NICE’s evaluation committee said that while there was clear evidence that the therapy reduces the number of bleeding episodes haemophilia B patients have each year, there was not enough on how well it works in the long term, meaning cost-effectiveness estimates were “uncertain”.
CSL has now agreed to a managed access programme with NICE under the Innovative Medicines Fund and said it will “work closely and collaboratively with the NHS England selected therapeutic centres to ensure that eligible patients can access treatment as soon as possible”.
Eduardo Cabas, general manager, CSL Behring UK and Ireland, said: “We are thrilled that NICE has honoured its commitment to a more progressive approach when assessing highly innovative medicines such as [Hemgenix]…”