European News

CSL, a biotech company, has announced that it has received conditional marketing authorisation (CMA) from the European Commission (EC) for its haemophilia B drug Hemgenix.

Hemgenix (etranacogene dezaparvovec) is an adenoassociated virus five (AAV5)-based gene therapy. It is indicated for the treatment of moderately severe to severe haemophilia B without a Factor IX (FIX) inhibitors history ‒ antibodies that can develop in patients treated with FIX replacement therapies.

The CMA was granted after a positive recommendation from the EMA’s Human Medicines Committee in December, and approval by the FDA the previous month. Currently, Hemgenix is now under review in the UK.

These approvals were based on the HOPE-B trial, the results of which showed 96% of haemophilia B patients dosed with a single infusion of Hemgenix had a sustained increase in FIX levels, accompanied by a 64% reduction in bleed rates. Follow up showed these effects were sustained for at least two years.

However, Hemgenix is currently priced at $3.5m per dose in the US, making it the most expensive medicine in the world and above the $2.9m that the Institute for Clinical and Economic Review (ICER) claims is a fair maximum price.

CSL states that the benefits of Hemgenix, such as the reduction of FIX replacement therapies and the preventing of other healthcare-related costs associated with uncontrolled bleeds, justifies the one-off cost.

“We now need to work to ensure that as many eligible patients across Europe can access this innovative treatment as possible,” said Lutz Bonacker, general manager of CSL’s European commercial operations, “We are fully committed to working together with payers and other stakeholders to achieve this.”