NETHERLANDS — The European Commission has approved CSL and uniQure’s gene therapy Hemgenix, making it the first one-time treatment in the EU for adults with severe and moderately severe hemophilia B.
Hemophilia B is a rare, lifelong bleeding disorder caused by a single gene defect that results in insufficient production of factor IX, a protein produced primarily by the liver that aids in the formation of blood clots.
As a result of the disease, hemophilia B patients may still experience spontaneous bleeding episodes, limited mobility, joint damage, or severe pain.
Prophylactic infusions of factor IX replacement therapy to temporarily replace or supplement low levels of blood-clotting factor are used to treat moderate to severe hemophilia B.
While these treatments are effective, people with hemophilia B must adhere to infusion schedules for the rest of their lives.
Hemgenix allows people with hemophilia B to produce their own factor IX, potentially lowering the risk of bleeding.
Hemgenix was approved in the United States late last year, and it is currently being reviewed by UK regulators.
CSL paid US$450 million upfront to the gene therapy from its developer uniQure in 2020, in a deal that could end up being worth more than US$2 billion, factoring in potential milestone payments.
The pivotal HOPE-B trial findings supported approval in Europe, which followed a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use in December.
The ongoing study found that hemophilia B patients treated with Hemgenix experienced stable and durable increases in mean factor IX activity levels of 36.9%, resulting in a 64% reduction in adjusted annualized bleed rate.
Furthermore, 96% of patients were able to discontinue routine factor IX prophylaxis after a single infusion of Hemgenix.
CSL launched the therapy in the United States with a list price of US$3.5 million, making it the most expensive medicine in the world.
This price was higher than the US$2.9 million suggested by the Institute for Clinical and Economic Review (ICER) in an earlier report.
Pricing in Europe will likely be lower than in the US, depending on the outcome of negotiations with EU member states and their health technology assessment (HTA) authorities.
CSL and uniQure claim that the benefits of Hemgenix in reducing the use of Factor IX replacement therapies and preventing other healthcare-related costs associated with uncontrolled hemophilia B outweigh the one-time cost.
Another gene therapy developer, Bluebird Bio, pulled out of the EU market entirely last year, claiming that pricing policies made it impossible to build a viable business there.
The CHMP recommendation keeps Hemgenix ahead of closest competitors Pfizer/Roche, whose fidanacogene elaparvovec candidate is in the BENEGENE-2 trial, with results expected in 2023.
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