Reimbursement urged in Spain for Hemgenix hemophilia B treatment
Spanish commission issues positive recommendation on gene therapy
The Spanish Interministerial Commission on the Pricing of Medicines, known as CIPM, has issued a positive recommendation for national reimbursement of Hemgenix (etranacogene dezaparvovec) for eligible people with hemophilia B, according to CSL Behring, the company that markets the gene therapy.
The recommendation means that patients in Spain should soon have access to the gene therapy through the national healthcare system. Hemgenix is approved in the U.S., and conditionally approved in the European Union for adults with severe or moderately severe hemophilia B without a history of inhibitors, or neutralizing antibodies against factor IX (FIX).
CSL Behring is working to expand access to Hemgenix across Europe, according to a company press release. The company said it is “building on recent milestone access decisions” for the treatment’s use and reimbursement for people with hemophilia B in the U.K., Scotland, France, Denmark, and Austria.
“We are proud that the Spanish CIPM has recognized Hemgenix … as an innovative one-time treatment option, marking a pivotal moment for Spanish haemophilia B patients and acknowledging the remaining unmet needs in haemophilia B treatment,” said Maria Jose Sanchez Losada, CSL Behring Iberia’s general manager. “Following this positive recommendation, our priority will now be to work with healthcare authorities to conclude the agreement and ensure Hemgenix can reach patients and healthcare professionals throughout the country.”
CSL Behring working with Spanish commission on Hemgenix reimbursement
Hemophilia B is caused by mutations in the F9 gene that impair the production or function of FIX, a protein needed for the blood to clot and stop bleeding. Treatment mainly involves supplying patients with a functional version of FIX to help prevent and control bleeding episodes, but such therapy is required throughout a person’s lifetime.
Given as a one-time infusion into the bloodstream, Hemgenix is a gene therapy that works to provide patients with a functional version of the F9 gene. The therapy is packaged aboard a harmless adeno-associated virus 5 targeting the liver. Once there, the working version of F9 is taken up by liver cells, which use their machinery to produce functional FIX. This is expected to increase FIX levels in the body and thereby help to prevent and control bleeds.
The agreement with the CIPM for Hemgenix is an important step forward for Spanish patients, the local healthcare system and regulatory authorities. … We are fully committed to continuing our close collaboration with stakeholders and local authorities to bring [this treatment] to all eligible people living with haemophilia B.
The therapy’s approval was mainly supported by data from an ongoing Phase 3 clinical trial called HOPE-B (NCT03569891), which is assessing Hemgenix’s safety and efficacy in 54 men, ages 18-75, with moderately severe to severe hemophilia B, over the course of five years.
Data after two years indicated the therapy was safe and effective, leading to sustained increases in FIX and lowering bleeding rates. That has allowed patients to stop using standard preventive factor replacement therapies. Long-term three-year results released late last year continued reinforcing the long-lasting efficacy and safety of Hemgenix in people with hemophilia B.
“The agreement with the CIPM for Hemgenix is an important step forward for Spanish patients, the local healthcare system and regulatory authorities,” said Diego Sacristan, CSL Behring’s head of international. “We are fully committed to continuing our close collaboration with stakeholders and local authorities to bring [this treatment] to all eligible people living with haemophilia B, where the unmet need still remains.”
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