First Hemophilia Patient to Receive Gene Therapy Outside Clinical Trials
by Hemophilia Outreach Center – Green Bay, WI

In a recent press release, the Hemophilia Outreach Center of Green Bay, Wisconsin announced that a WI resident is the first ever hemophilia B patient to receive gene therapy since FDA approval of HEMGENIX. Mason Buxton, the individual treated with the gene therapy stated, “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.”

HEMGENIX (etranacogene dezaparvovec), manufactured by uniQure Inc. and distributed by CSL Behring LLC, was FDA approved in 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. The FDA press release can be found here. Although HEMGENIX is not a cure for Hemophilia B patients, the medical advancement may reduce, if not, eliminate weekly preventative factor replacement and bleeds.

The HOC press release included the following quotes from providers:

“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.