FIRST TWO HEMOPHILIA B PATIENTS RECEIVE CSL BEHRING’S GENE THERAPY HEMGENIX IN FRANCE

KUALA LUMPUR, July 4 (Bernama) -- Global biotechnology leader CSL Behring announced that two hemophilia B patients were treated with the gene therapy HEMGENIX (etranacogene dezaparvovec) at Hemophilia Treatment Centers in France.

According to a statement, this milestone achievement makes HEMGENIX the first gene therapy administered as a treatment in a real-world setting for hemophilia B in Europe.

“This milestone has been made possible by the innovative Direct Access scheme adopted in France, allowing patients to benefit from early access to pioneering treatments.

“We are encouraged to see increasing access to gene therapies in European countries and are fully committed to ensuring that access to potentially life-changing treatment continues,” said CSL Behring Senior Vice President and General Manager of Commercial Operations Europe, Dr Lutz Bonacker.

HEMGENIX is the first one-time gene therapy approved in Europe for the treatment of adults with severe and moderately severe hemophilia B, an inherited bleeding disorder caused by the lack of Factor IX (a protein needed to produce blood clots to stop bleeding).

Following European Commission approval, HEMGENIX was the first ever therapy to be granted Direct Access in France, thus enabling the first patients to be treated in Europe outside of the clinical programme.

Though effective, current therapies can be time intensive and require regular treatment that can have a substantial impact on a patient’s daily life, while HEMGENIX offers a one-time treatment, allowing people living with hemophilia B to produce their own Factor IX, which can lower the risk of bleeding.

HEMGENIX was granted conditional marketing authorisation by the European Commission for the European Union and European Economic Area in February 2023, following approval from the United States Food and Drug Administration in November 2022.

It has also been approved by Health Canada, the United Kingdom's Medicines and Healthcare products Regulatory Agency, Switzerland’s Swissmedic and Australia’s Therapeutic Goods Administration.

-- BERNAMA