Yahoo Finance uniQure (QURE) Partner Gets FDA Nod for Hemophilia B Gene Therapy
Shares of uniQure QURE were up 7.08% during market hours after the company announced that its partner CSL Behring received FDA approval for one-time gene therapy, Hemgenix (etranacogene dezaparvovec-drlb), for treating hemophilia B patients above 18 years.
uniQure conducted the research and clinical development for the product. CSL Behring is a subsidiary of CSL Limited.
Hemgenix is the first FDA-approved gene therapy for treating hemophilia B.
The biologics license application (BLA) for etranacogene dezaparvovec, was submitted in March 2022 by CSL, and accepted for priority review by the FDA in May 2022.
CSL Behring is uniQure’s global commercialization partner for etranacogene dezaparvovec and is solely responsible for the development, regulatory activities and commercialization associated with etranacogene dezaparvovec, since May 2021. uniQure is now responsible for the global manufacturing of the product.
Hemophilia B is a rare bleeding disorder caused by a missing factor IX, known as the clotting protein. Symptoms of the disease include profuse bleeding after an injury, surgery and sometimes even without a clear cause. These bleeding episodes can lead to serious complications for an individual.
Hemgenix has been approved for treating adults with hemophilia B who are currently being treated with factor IX prophylaxis therapy, have a current or historical life-threatening hemorrhage or have repeated, serious spontaneous bleeding episodes.
It is a one-time gene therapy product consisting of a viral vector-carrying gene for clotting factor IX, which helps increase blood levels of factor IX, thereby limiting bleeding episodes. The treatment is given as a single dose of IV infusion.
Hemgenix’s FDA approval is based on results from the pivotal HOPE-B study, which demonstrated that after administering the drug, patients produced mean factor IX activity of 39% at six months and 36.7% at 24 months. The mean adjusted annualized bleeding rate (ABR) for all bleeds was also reduced by 54% after seven to 18 months of administering Hemgenix compared with the six-month lead-in period on factor IX prophylactic replacement therapy. 94% of the subjects treated with Hemgenix discontinued using prophylaxis therapy.
No severe adverse events were reported from the study.
uniQure has rallied 29.7% in the year-to-date period against the industry’s decline of 17.8%.
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Zacks Rank and Stocks to Consider
uniQure currently has a Zacks Rank #3 (Hold).
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