CSL's HEMGENIX® is a one-time, single dose treatment for adults with hemophilia B who require routine prophylaxis
The approval is based on results from the ongoing Phase III, open label, single-dose, single-arm, multi-center HOPE-B trial of 54 participants, the largest gene therapy trial in hemophilia B to date. Data from the pivotal trial show that people with hemophilia B treated with a single infusion of HEMGENIX demonstrated significant increases in mean FIX activity levels (as measured by one-stage assay) of 36.9% at 18-months, that were sustained at 36.7% at 24-months post-treatment, compared to the six-month lead in period. Seven to 18 months post-infusion, the mean ABR was reduced by 58% compared to the six-month lead-in period (4.13 to 1.73). Following infusion of HEMGENIX, 96% of patients (52 out of 54) discontinued use of prophylaxis and remained free of previous continuous routine prophylaxis therapy. Of the adverse events reported based on phase 2b and phase 3 trial (Hope-B), most frequently reported adverse drug reactions were ALT elevations, headache, influenza-like illness and AST elevations.
"This approval continues to demonstrate CSL's promise to pursue, develop and deliver new innovative treatment options that meet the needs of the rare disease community," said Dr.
Hemophilia B is a rare, lifelong bleeding disorder caused by a single gene defect, resulting in insufficient production of factor IX, a protein primarily produced by the liver that helps the blood to properly clot. Treatments for hemophilia B include prophylactic infusions of factor IX replacement therapy to temporarily replace or supplement low levels of blood-clotting factor and, while these therapies are effective, those with hemophilia B must adhere to strict, lifelong infusion schedules. Those with hemophilia B may also still experience spontaneous bleeding episodes as well as limited mobility, joint damage, or severe pain as a result of the disease. HEMGENIX allows people living with hemophilia B to produce their own factor IX, which can lower the risk of bleeding.
"The approval of HEMGENIX in
HEMGENIX® was also approved by the
About Hemophilia B
Hemophilia B is a life-threatening rare disease caused by a mutation on the F9 gene, resulting in low levels of functional clotting factor IX. People with the condition are particularly vulnerable to bleeds in their joints, muscles, and internal organs, leading to pain, swelling, and joint damage. Current treatments for moderate to severe hemophilia B include life-long prophylactic infusions of factor IX to temporarily replace or supplement low levels of the blood-clotting factor.
About HEMGENIX®
HEMGENIX® is a gene therapy that reduces the rate of abnormal bleeding in eligible people with hemophilia B by enabling the body to continuously produce factor IX, the deficient protein in hemophilia B. It uses AAV5, a non-infectious viral vector, called an adeno-associated virus (AAV). The AAV5 vector carries the Padua gene variant of Factor IX (FIX-Padua) to the target cells in the liver, generating factor IX proteins that are 5x-8x more active than normal. These genetic instructions remain in the target cells, but generally do not become a part of a person's own DNA. Once delivered, the new genetic instructions allow the cellular machinery to produce stable levels of factor IX.
About the Pivotal HOPE-B Trial
The pivotal Phase III HOPE-B trial is an ongoing, multinational, open-label, single-arm study to evaluate the safety and efficacy of HEMGENIX®. Fifty-four adult hemophilia B patients classified as having moderately severe to severe hemophilia B and requiring prophylactic factor IX replacement therapy were enrolled in a prospective, six-month or longer observational period during which time they continued to use their current standard of care therapy to establish a baseline Annual Bleeding Rate (ABR). After the six-month lead-in period, patients received a single intravenous administration of HEMGENIX® at the 2x10^13 gc/kg dose. Patients were not excluded from the trial based on pre-existing neutralizing antibodies (NAbs) to AAV5.
A total of 54 patients received a single dose of HEMGENIX® in the pivotal trial, with 53 patients completing at least 18 months of follow-up. The primary endpoint in the pivotal HOPE-B study was ABR 52 weeks after achievement of stable factor IX expression (months 7 to 18) compared with the six-month lead-in period. For this endpoint, ABR was measured from month seven to month 18 after infusion, ensuring the observation period represented a steady-state factor IX transgene expression. Secondary endpoints included assessment of factor IX activity.
No serious adverse reactions were reported. One death resulting from urosepsis and cardiogenic shock in a 77-year-old patient at 65 weeks following dosing was considered unrelated to treatment by investigators and the company sponsor. A serious adverse event of hepatocellular carcinoma was determined to be unrelated to treatment with HEMGENIX by independent molecular tumor characterization and vector integration analysis. No inhibitors to factor IX were reported.
Long-term 24-month data presented at the 54th
Important Information for
For more information and a complete risk/benefit profile, please refer to the Product Monograph available here.
About CSL
CSL (ASX:CSL; USOTC:CSLLY) is a leading global biotechnology company with a dynamic portfolio of lifesaving medicines, including those that treat hemophilia and immune deficiencies, vaccines to prevent influenza, and therapies in iron deficiency, dialysis and nephrology. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL – including our three businesses,
For more information about CSL, visit www.CSL.com.
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