Gene therapy for hemophilia shows sustained effect in phase 1/2 study

Updated phase 1/2 data on giroctocogene fitelparvovec (SB-525, or PF-07055480), an investigational gene therapy, showed sustained factor VIII (FVIII) activity level in patients with hemophilia A, according to a statement from Pfizer Inc and Sangamo Therapeutics, Inc.

In the Phase 1/2 Alta study, 5 patients with severe hemophilia A who received the highest dose of 3e13 vg/kg showed sustained factor VIII (FVIII) activity levels. No patients experienced bleeding events or required FVIII infusions.

“We are excited that these data affirm previous findings from this Phase 1/2 study, and that all five patients have sustained levels of factor VIII activity with no bleeding events or use of factor replacement therapy. We are encouraged by the potential of giroctocogene fitelparvovec to demonstrate longer-term durability, an important element for patients living with severe hemophilia A,” said Seng Cheng, Senior Vice President and Chief Scientific Officer of Pfizer’s Rare Disease Research Unit, in the statement. “The Phase 3 lead in study is ongoing, and we look forward to dosing patients with this investigational gene therapy in the pivotal Phase 3 trial later this year.”

Read the full press release here.