Gene therapy superior to routine prophylaxis in hemophilia A
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Giroctocogene fitelparvovec reduced bleeding for men with moderately severe to severe hemophilia A, according to data released by the agent’s manufacturer.
Giroctocogene fitelparvovec (Pfizer) is an investigational gene therapy “that contains a bio-engineered AAV6 capsid and a modified B-domain deleted human coagulation [factor VIII] gene,” according to a Pfizer press release.
The phase 3 AFFINE trial assessed the efficacy and safety of the agent for men with moderately to severe hemophilia A.
Researchers enrolled eligible participants in a lead-in study in which they received routine factor VIII replacement prophylaxis.
Upon completion of the lead-in study, men enrolled into the AFFINE study, in which they received a single 3e13 vg/kg dose of giroctocogene fitelparvovec via IV infusion.
A comparison of the annualized bleeding rate from week 12 through at least 15 months after giroctocogene fitelparvovec infusion with the annualized bleeding rate observed with routine factor VIII replacement prophylaxis in the lead-in study served as the primary outcome.
The primary efficacy analysis included 50 men who completed at least 6 months of routine factor VIII replacement prophylaxis therapy during the lead-in study.
Results showed a significant reduction in annualized bleeding rate after giroctocogene fitelparvovec infusion compared with the pre-infusion period (1.24 vs. 4.73; P = .004).
The mean treated annualized bleeding rate declined from 4.08 in the pre-infusion period to 0.07 in the post-infusion period (P < .0001).
The majority (84%) of men maintained factor VIII activity greater than 5% at 15 months after infusion with giroctocogene fitelparvovec.
“For people living with hemophilia A, the physical and emotional impact of needing to prevent and treat bleeding episodes through frequent IV infusions or injections cannot be underestimated,” trial investigator Andrew Leavitt, MD, director of the Adult Hemophilia Treatment Center at University of California, San Francisco, said in the press release. “I’m excited by the strength of these positive results from the AFFINE trial that show giroctocogene fitelparvovec was generally well tolerated and demonstrate the transformative potential of this gene therapy candidate.”
Researchers noted serious adverse events in 15 patients (20%), including 13 treatment-related events in 10 patients (13.3%).
Fifteen patients (20%) experienced serious adverse events. Researchers determined a combined 13 serious adverse events among 10 patients (13.3%) were related to treatment.
Complete data from the AFFINE study will be presented at a medial meeting.
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