U.S. Food and Drug Administration approves CSL's HEMGENIX® (etranacogene dezaparvovec-drlb), the first gene therapy for hemophilia B
- This historic approval provides a new treatment option that reduces the rate of annual bleeds, reduces or eliminates the need for prophylactic therapy and generates elevated and sustained factor IX levels for years after a one-time infusion
- With the approval of HEMGENIX, CSL now offers an even more comprehensive portfolio of treatments for people living with hemophilia B, ushering in a new era of treatment options
KING OF PRUSSIA, Pa., Nov. 22, 2022 /PRNewswire/ -- Global biotechnology leader CSL (ASX: CSL) today announced that the U.S. Food and Drug Administration (FDA) approved HEMGENIX® (etranacogene dezaparvovec-drlb), the first and only one-time gene therapy for appropriate adults with hemophilia B. HEMGENIX is approved for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage or have repeated, serious spontaneous bleeding episodes. In the ongoing clinical trial, HEMGENIX reduced the rate of annual bleeds and 94 percent of patients discontinued factor IX prophylaxis and remained prophylaxis-free.
Experience the full interactive Multichannel News Release here: https://www.multivu.com/players/English/9088951-csl-announces-fda-approval-of-hemgenix/
"As part of our promise to patients, CSL is committed to delivering innovative and groundbreaking solutions to address unmet medical needs, and we are proud to introduce the next wave of breakthrough medicines for people living with hemophilia B," said Paul Perreault, CSL's Chief Executive Officer and Managing Director. "We recognize and thank all trial participants, scientists and investigators—without whom this important achievement would not have been possible—and look forward to seeing the positive impact of HEMGENIX on the hemophilia B community."
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 blood clots form. Treatments for moderate to severe 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. They may also still experience spontaneous bleeding episodes as well as limited mobility, joint damage or severe pain as a result of the disease. For appropriate patients, HEMGENIX allows people living with hemophilia B to produce their own factor IX, which can lower the risk of bleeding.
"We are thrilled to witness this milestone in hemophilia B treatment," shared Kim Phelan, Chief Operating Officer of The Coalition for Hemophilia B. "Over the years we have seen a variety of advancements for the hemophilia community, but gene therapy is the first treatment option to offer those living with hemophilia B--and caregivers--the possibility of freedom from the need for regular, ongoing infusions."
The FDA approval is supported by results from the ongoing HOPE-B trial, the largest gene therapy trial in hemophilia B to date. Results from the study demonstrated that HEMGENIX allowed patients to produce mean factor IX activity of 39 percent at six months and 36.7 percent at 24 months post infusion. Seven to 18 months post-infusion, the mean adjusted annualized bleeding rate (ABR) for all bleeds was reduced by 54 percent compared to the six-month lead-in period on factor IX prophylactic replacement therapy (4.1 to 1.9). In addition, 94 percent (51 out of 54) of patients treated with HEMGENIX discontinued use of prophylaxis and remained free of previous continuous routine prophylaxis therapy. The most common side effects (incidence ≥5%) were liver enzyme elevations, headache, elevated levels of a certain blood enzyme, flu-like symptoms, infusion-related reactions, fatigue, nausea and feeling unwell.
"HEMGENIX is unique in its approach to increasing mean factor IX activity and hemostatic protection in those with hemophilia B, and today's approval could fundamentally transform the treatment paradigm for this life-long condition," said Dr. Steven Pipe, Professor and the Laurence A. Boxer Research Professor of Pediatrics and Professor of Pathology at the University of Michigan and a lead investigator in the HOPE-B study. "As a clinician, I look forward to being able to provide a new treatment option that may help patients treated with HEMGENIX become free from the regular infusion schedule that many people living with hemophilia B rely on to protect them from the debilitating effects of the condition."
The multi-year clinical development program for HEMGENIX was led by uniQure (Nasdaq: QURE), and sponsorship of the clinical trials transitioned to CSL after it acquired global rights to commercialize the treatment.
"Today's approval of the world's first gene therapy for hemophilia B is an historic achievement based on more than a decade of research and clinical development," said Matt Kapusta, Chief Executive Officer, uniQure. "We have always believed that gene therapy had the potential to provide transformative benefits to people living with hemophilia B and are excited that the hemophilia community will have a new, safe and effective treatment option available to them."
CSL Behring, a CSL business, will make HEMGENIX available for eligible people with hemophilia B as soon as possible.
"CSL is proud to have been at the forefront of providing life-changing medicines for rare diseases for over a century. Today's historic approval builds on our promise to put patients first in all that we do to discover, develop and deliver biotherapeutics and vaccines that meet their needs," said Bill Mezzanotte, Head of Research & Development and Chief Medical Officer of CSL. "With HEMGENIX, we now offer a comprehensive portfolio of innovative medicines for hemophilia B, giving people living with the condition more choice in treatments and better and more durable control over their disease."
HEMGENIX is still currently under assessment by other regulatory agencies.
About Hemophilia B
About the Pivotal HOPE-B Trial
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.
Important Safety Information (ISI)
What is HEMGENIX?
HEMGENIX is administered as a single intravenous infusion and can be administered only once.
What medical testing can I expect to be given before and after administration of HEMGENIX?
HEMGENIX may lead to elevations of liver enzymes in the blood; therefore, ultrasound and other testing will be performed to check on liver health before HEMGENIX can be administered. Following administration of HEMGENIX, your doctor will monitor your liver enzyme levels weekly for at least 3 months. If you have preexisting risk factors for liver cancer, regular liver health testing will continue for 5 years post-administration. Treatment for elevated liver enzymes could include corticosteroids.
What were the most common side effects of HEMGENIX in clinical trials?
What should I watch for during infusion with HEMGENIX?
What should I avoid after receiving HEMGENIX?
Please see full prescribing information for HEMGENIX.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
You can also report side effects to CSL Behring's Pharmacovigilance Department at 1-866-915-6958.
For more information about CSL, visit CSL.com.
View original content:https://www.prnewswire.com/news-releases/us-food-and-drug-administration-approves-csls-hemgenix-etranacogene-dezaparvovec-drlb-the-first-gene-therapy-for-hemophilia-b-301685654.html
Company Codes: Australia:CSL, OTC-PINK:CSLLY, NASDAQ-NMS:QURE, OTC-BB:CSLLY