First Ever Hemophilia Patient to Receive Gene Therapy Since FDA Approval

FIRST EVER HEMOPHILIA PATIENT TO RECEIVE GENE THERAPY SINCE FDA APPROVAL

FOR IMMEDIATE RELEASE

JUNE 20, 2023

GREEN BAY, WI- Wisconsin resident is the first ever hemophilia B patient to receive gene therapy since FDA approval.

“I feel honored to be the first person to participate in this therapy outside of the clinical trials. I am very excited that I am going to be helping to prove that this is a treatment option for individuals with hemophilia B.”-Mason Buxton

HEMGENIX (etranacogene dezaparvovec), manufactured by uniQure Inc. and distributed by CSL Behring LLC, was FDA approved November of 2022 and is for the treatment of adults with Hemophilia B (Factor IX Deficiency) who currently use Factor IX preventative therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

Hemophilia B is a genetic bleeding disorder resulting from missing or insufficient levels of blood clotting Factor IX, a protein needed to produce blood clots to stop bleeding. Symptoms can include prolonged or heavy bleeding after an injury, surgery, or dental procedure; in severe cases, bleeding episodes can occur spontaneously without a clear cause. Prolonged bleeding episodes can lead to serious complications, such as bleeding into joints, muscles or internal organs, including the brain. (Commissioner, O. of the (2022) FDA approves first gene therapy to treat adults with hemophilia B, U.S. Food and Drug Administration. Available at: https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treat-adults-hemophilia-b#:~:text=Today%2C%20the%20U.S.%20Food%20and,current%20or%20historical%20life%2Dthreatening (Accessed: 23 May 2023).

Although HEMGENIX is not a cure for Hemophilia B patients, the medical advancement may reduce, if not, eliminate weekly preventative factor replacement and bleeds.

“HEMGENIX is an important addition to the current treatment paradigm and a long-awaited advancement for the hemophilia B community,” said Matthew Ryan, MD, Adult Hematologist and Medical Director at Hemophilia Outreach Center, Green Bay, WI. “This exciting treatment offers patients a chance to live a life without frequent bleeds or the burden of regular infusions. The availability of this new one-time treatment also sparks additional conversations in the community when it comes to treatment options and goals, which is extremely important when managing a life-long condition, as an individual’s needs can change for a variety of reasons.”

“I’m just excited that patients have this new opportunity that will lead to such a better quality of life versus their current prophylaxis treatment! To live a life free from recurrent prophylaxis, not having that anxiety over if they are going to have a bleed or if they have factor with them, not worrying about damage to their joints from breakthrough bleeds….it will be very lifechanging for them.”- Andrea Miller, APNP, Hemophilia Outreach Center.