KING OF PRUSSIA, PA, USA – DECEMBER 15, 2021 – Global biotherapeutics leader CSL Behring today announced that the Committee for Medicinal Products for Human Use (CHMP), the chief scientific body of the European Medicines Agency (EMA) accepted its request for an accelerated assessment of the etranacogene dezaparvovec Marketing Authorisation Application (MAA). Etranacogene dezaparvovec (also known as EtranaDez), currently being studied in the Phase 3 HOPE-B clinical trial, is an investigational gene therapy for people living with hemophilia B, a life-threatening bleeding disorder.
The CHMP grants accelerated assessment when a medicinal product is expected to be of major public health interest, particularly pertaining to therapeutic innovation. Accelerated assessment potentially reduces the review timeline from 210 days to 150 days once the MAA is filed and validated.
“CSL Behring has been dedicated to improving the lives of patients with Hemophilia B for decades and the acceptance of an accelerated regulatory review underscores the high unmet need for a long-term, innovative treatment option for these patients,” said Emmanuelle Lecomte Brisset, Head of Global Regulatory Affairs at CSL Behring. “We look forward to submitting our Marketing Authorization Application in the first half of 2022 so that we can make this novel therapy option available to patients as soon as possible.”
Brahm Goldstein, MD, MCR, Vice President, Research and Development, Hematology added, “Accelerated regulatory review supports our commitment to advanced research and providing pioneering treatment options. Gene therapy, and its associated near-normal blood clotting potential, could be life changing for people living with hemophilia B who are vulnerable to spontaneous bleeding in their muscles, internal organs and joints. This milestone underscores our excitement about the promise of gene therapy for people living with hemophilia B.”
Along with global partner, uniQure (Nasdaq: QURE), the company recently announced positive topline results from the HOPE-B pivotal trial of etranacogene dezaparvovec. The study achieved its primary endpoint of non-inferiority in annualized bleeding rate after stable Factor IX (FIX) expression, assessed at 18 months following a single dose. It also achieved a secondary endpoint demonstrating statistical superiority in reduction of annualized bleeding rate compared to baseline FIX prophylactic therapy. uniQure led the multi-year clinical development of etranacogene dezaparvovec prior to entering into a Commercialization and License Agreement with CSL Behring in June 2020 for exclusive global rights to etranacogene dezaparvovec.
About Hemophilia B
Hemophilia B is a life-threatening degenerative disease. People with the condition are particularly vulnerable to bleeds in their muscles, internal organs, and joints, leading to pain, swelling, and joint damage. Current treatment includes life-long prophylactic infusions of FIX to temporarily replace or supplement low levels of the blood-clotting factor. This can reduce joint bleeding events, prevent life-threatening bleeds, and preserve joint function. However, infusions can be cumbersome, painful and veins can fibrose over time, making ongoing treatment difficult. A person’s immune system may also generate inhibitors against the replacement factor, negating its benefit. In addition, many people receiving prophylaxis are forced to plan their lives around the highs and lows of their FIX levels, which rise immediately after an infusion but drop over time -- leaving them especially vulnerable to bleeds and pain in the days before their next infusion. Most troubling, prophylactic FIX replacement therapy sometimes fails to control unobservable micro-bleeds in the joints, meaning that the degeneration can continue despite regular infusions. Missing an infusion may also the increase their likelihood of a life-threatening bleed or even premature death.
About Gene Therapy in Hemophilia B
Gene therapy has the potential to make a functional cure possible in hemophilia B. Gene therapy achieves this with modified non-infectious viruses called “vectors” that can enter certain cells. Vectors act as delivery trucks, carrying a package of genetic instructions to specific cells. Once delivered, the package acts like a generator that plugs into the cellular machinery, allowing a person to produce their own stable levels of FIX. A certain type of vector, called an adeno-associated virus, or AAV, dissolves after delivering its package. The genetic instructions remain, but never actually become a part of a person’s own DNA.
About Etranacogene Dezaparvovec
Etranacogene dezaparvovec (also known as CSL222 and EtranaDez, previously known as AMT-061) uses a specific type of AAV, called AAV5, as its delivery vehicle. The AAV5 vector carries the patent-protected Padua gene variant of Factor IX (FIX-Padua), which generates FIX proteins that are 5x-8x more active than normal. Preclinical and clinical data show that AAV5-based gene therapies may be clinically effective in the 95 percent of hemophilia B patients with pre-existing antibodies to AAV vectors, thereby potentially increasing patient eligibility for treatment compared to other AAV gene therapy product candidates.
About CSL Behring
CSL Behring is a global biotherapeutics leader driven by our promise to save lives. Focused on serving patients’ needs by using the latest technologies, we discover, develop and deliver innovative therapies for people living with conditions in the immunology, hematology, cardiovascular and metabolic, respiratory, and transplant therapeutic areas. We use three strategic scientific platforms of plasma fractionation, recombinant protein technology, and cell and gene therapy to support continued innovation and continually refine ways in which products can address unmet medical needs and help patients lead full lives.
CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. The parent company, CSL Limited (ASX:CSL;USOTC:CSLLY), headquartered in Melbourne, Australia, employs more than 25,000 people worldwide, and delivers its life-saving therapies to people in more than 100 countries. For inspiring stories about the promise of biotechnology, visit Vita CSLBehring.com/vita and follow us on Twitter.com/CSLBehring.
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