Tampa General Hospital's Cancer Institute has achieved a significant milestone in treating hemophilia B, becoming the first medical center in Florida and one of the earliest in the United States to use Hemgenix, a novel gene therapy. This development, announced earlier this week, marks a significant advancement in hematology and oncology.

Hemophilia B, which is less prevalent than other forms of hemophilia, results from mutations in the F9 gene. This leads to a shortage of clotting factor IX (FIX), Hemgenix, also known as etranacogene dezaparvovec, is specifically designed for severe to moderately severe hemophilia B cases and aims to restore normal FIX activity levels, which are typically 2% or lower in affected individuals. This gene therapy employs a one-time intravenous infusion to introduce a functional gene into liver cells. This process enables these cells to produce factor IX protein, essential for blood clotting and lessening or even eliminating the need for ongoing FIX replacement therapies. 

The introduction of Hemgenix at TGH was led by Dr. Nathan Visweshwar, an associate professor in the Division of Hematology and Medical Oncology at the USF Health Morsani College of Medicine and a specialist at TGH. 

The approval of Hemgenix was primarily based on the results from the Phase 3 HOPE-B clinical trial (NCT03569891), which involved more than 50 men with moderate to severe hemophilia B. The trial showed that Hemgenix successfully increased FIX activity, with sustained levels at an average of 38.6% of normal after three years. Furthermore, the therapy demonstrated a good safety profile, significantly reducing bleed rates and the need for conventional replacement treatments.

The successful administration of Hemgenix at TGH is a milestone in addressing this rare disorder. The therapy's efficacy is supported by the results of the Phase 3 HOPE-B clinical trial, which showed promising outcomes in increasing factor IX activity and reducing bleed rates and the need for conventional treatments.

Dr. Abraham Schwarzberg, Senior Vice President and Chief of Oncology at TGH highlighted the successful administration of this gene therapy as an example of the benefits an academic and research health system can offer. He remarked that introducing new therapeutic options for rare diseases reflects their commitment to applying the latest research and clinical practices to treat complex conditions effectively and safely.

TGH's excellence in cancer care is well-recognized, with its Cancer Institute ranked in the top 10% of hospitals nationally for cancer care by U.S. News & World Report. It has also been named one of America's Best Cancer Hospitals by Newsweek and features among the top 10 cancer facilities in Florida.