Hemgenix (etranacogene dezaparvovec)

Hemgenix is a one-time gene therapy infusion to treat adults with hemophilia-B, a type of bleeding disorder caused by the lack of coagulation factor IX, a clotting protein in the blood.

How it Works

Before treatment, your care team will discuss benefits, potential risks and follow up requirements. After a shared decision making, the team will assess your eligibility, overall health and liver function. If all is well, Hemgenix will be infused into your veins and will be taken up by liver cells to help your body produce coagulation factor IX on its own. Once the infusion is completed, you’ll follow up with your care team at Karmanos to create a plan to monitor your factor IX level and liver enzymes through routine blood test.

Hemgenix received FDA approval for the treatment of adult patients with Hemophilia B in November 2022. The approval was based on HOPE study that showed reduction in annual bleeds with 94% of patients discontinued factor IX prophylaxis. The most frequent side effects reported were:

  • Liver enzyme elevations.
  • Headache.
  • Elevated levels of a certain blood enzyme.
  • Flu-like symptoms.
  • Infusion-related reactions.
  • Fatigue.
  • Nausea.
  • Feeling unwell.

Hematology Oncology at Karmanos