HEM B Therapy Etranacogene Dezaparvovec Wins Thumbs-up
EMA's CHMP recommended conditional marketing authorization for gene therapy
A committee of the European Medicines Agency (EMA) has issued a positive opinion recommending the one-time gene therapy etranacogene dezaparvovec be granted conditional marketing authorization to treat appropriate adults with hemophilia B.
The recommendation from the EMA’s Committee for Medicinal Products for Human Use (CHMP) will now be reviewed by the European Commission, which will issue a final decision that applies to all EU member states, as well as Iceland, Liechtenstein, and Norway.
If etranacogene dezaparvovec is approved, it would become the first gene therapy for hemophilia B available in the European economic area, according to CSL Behring, which acquired the its global rights from its original developer, uniQure, in 2021.
The gene therapy was recently approved in the U.S., under the brand name Hemgenix.
“The CHMP’s positive opinion moves us one step closer to bringing this groundbreaking innovation to hemophilia B patients in Europe,” said Emmanuelle Lecomte Brisset, senior vice president and head of global regulatory affairs at CSL, in a press release. “Getting a new medicine to this stage of the regulatory process takes the support of many, including clinical trial participants, the hemophilia community in general, investigators, clinicians, regulatory agencies, our people, and our partners at uniQure, to name a few.”
Hemophilia B is caused by mutations in the gene that provides instructions for making the clotting protein factor IX (FIX). Etranacogene dezaparvovec uses a harmless viral vector to deliver a highly functional version of the gene to cells in the liver, promoting healthy FIX production.
“We are very pleased with this positive opinion recommending conditional marketing authorization for etranacogene dezaparvovec,” said Matt Kapusta, CEO of uniQure, said in a separate press release. “We look forward to continuing our partnership with CSL to help bring this potentially life-changing treatment option to people with hemophilia B.”
CHMP’s positive opinion was supported by data from the Phase 3 HOPE-B trial (NCT03569891), which tested the experimental gene therapy in 54 men with moderate to severe hemophilia B.
Results showed, bleeding rates decreased by 64% over up to two years after treatment compared with rates with standard of care FIX replacement therapy.
Over this time, FIX activity levels were maintained at about 37% of normal values and the use of FIX replacement therapies decreased dramatically, with all but three patients stopping preventive treatment entirely.
The gene therapy was generally well tolerated, with no treatment-related side effects that were deemed serious. The HOPE-B trial is expected to conclude in 2025.
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