CSL Behring confirms long-term efficacy of HEMGENIX in hemophilia B treatment

Investing.com -- Global biotechnology leader CSL Behring (ASX:CSL; USOTC: CSLLY (OTC:CSLLY)) announced the four-year results from the HOPE-B study, confirming the long-term efficacy and safety of HEMGENIX® (etranacogene dezaparvovec-drlb), a one-time infusion treatment for adults with hemophilia B. The results were presented at the 18th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD).

Data from the study showed that HEMGENIX continues to deliver sustained factor IX activity levels for up to four years post-treatment. The treatment also provides long-term bleed protection, eliminates the need for routine factor IX prophylaxis, and has a favorable safety profile. HEMGENIX, approved by the U.S. Food and Drug Administration (FDA) in 2022, is the first gene therapy for the treatment of adults with hemophilia B who use factor IX prophylaxis therapy or have life-threatening bleeding or repeated serious spontaneous bleeding episodes. The therapy can treat adult patients both with and without AAV5 neutralizing antibodies.

The HOPE-B trial involved 54 adult male participants with severe or moderately severe hemophilia B, with or without preexisting AAV5 neutralizing antibodies. They were infused with a single dose of HEMGENIX. Out of the 54 participants, 51 completed four years of follow-up. The treatment resulted in near-normal mean factor IX activity levels sustained through four years post-treatment, with a reduction in the annualized bleeding rate (ABR) by approximately 90% from the lead-in to the fourth year. In the fourth year, 94% of patients remained free of continuous prophylaxis treatment. No patients returned to continuous prophylaxis between the third and fourth years.

No serious adverse events related to HEMGENIX treatment were reported. The treatment was generally well-tolerated, with most adverse events occurring in the first six months post-treatment. The most common adverse event was an increase in alanine transaminase (ALT), for which 16.7% of participants received supportive care with reactive corticosteroids.

The clinical development of HEMGENIX was led by uniQure (Nasdaq: NASDAQ:QURE) and the sponsorship of the clinical trials transitioned to CSL after it licensed global rights to commercialize the treatment. CSL has established a post-marketing registry to generate additional evidence on the long-term safety, efficacy, and durability of gene therapy. HEMGENIX has been granted conditional marketing authorization by the European Commission (EC) for the European Union and European Economic Area, the UK’s Medicines and Healthcare products Regulatory Agency (MHRA), Health Canada, Switzerland’s Swissmedic, and provisional approval by Australia’s Therapeutic Goods Administration (TGA).

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.