GREEN BAY, WI — A Petoskey man with hemophilia B is the first patient from Michigan to receive HEMGENIX gene therapy since its approval by the Federal Drug Administration.
Andrew Bartkowiak’s gene therapy infusion took place July 31 at the Hemophilia Outreach Center in Green Bay.
He said the treatment gives him some relief and hope for a cure in the future for hemophilia B, 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.
Bartkowiak said the treatment provides some relief and hope for a cure in the future.
“With this treatment and the promise of actually feeling even just a little better my hopes and dreams are already starting to become more attainable and desirable as my optimism returns to me,” Bartkowiak said. “Better days lie ahead and I can only hope that everyone else in the community feels this same energy soon as we all deserve to live out these good days we might have told ourselves probably wasn’t in the cards.”
According to Matthew Ryan, MD, the adult hematologist and medical director at Hemophilia Outreach Center, HEMGENIX is an important addition to the current treatment paradigm and a long-awaited advancement for the hemophilia B community.
He said the 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,” Ryan said.
HEMGENIX (etranacogene dezaparvovec) is manufactured by uniQure Inc. and distributed by CSL Behring LLC, and was FDA-approved in November 2022 and is for the treatment of adults with hemophilia B (Factor IX Deficiency) who currently use Factor IX preventative therapy, or have a current or historical life-threatening hemorrhage, or have repeated serious spontaneous bleeding episodes.
HOC advanced practice nurse prescriber Andrea Miller said the chance to give patients more freedom from symptom pain is a positive sign of disease research.
“I’m excited that patients have this new opportunity that will lead to such a better quality of life versus their current prophylaxis treatment,” Miller said. “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 life-changing for them.”
Though HEMGENIX is not a cure for hemophilia B patients, the medical advancement may reduce, if not eliminate weekly preventative factor replacement and bleeds, doctors said.
For more information on HOC and its work, visit www.hemophiliaoutreach.org .
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