Pfizer has reported positive data from the Phase III AFFINE clinical trial of investigational gene therapy, giroctocogene fitelparvovec, to treat adults with moderately severe to severe haemophilia A.
The multicentre, open-label, single-arm trial is designed to assess the efficacy and safety of a single dose of the therapy in adult male subjects.
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According to the findings, the study met its primary goal of demonstrating non-inferiority and superiority in reducing the total annualised bleeding rate (ABR) from week 12 through at least 15 months post-infusion versus routine Factor VIII (FVIII) replacement prophylaxis.
After a single 3e13 vg/kg dose, the gene therapy showed a significant decrease in mean total ABR compared to the pre-infusion period.
Key secondary endpoints were also met and showed superiority over prophylaxis.
At 15 months post-infusion, 84% of subjects had FVIII activity above 5%, with most achieving FVIII activity of 15% or higher.
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By GlobalDataThe mean treated ABR showed a dramatic 98.3% reduction from 4.08 pre-infusion to 0.07 post-infusion (from Week 12 up to at least 15 months; one-sided p-value < 0.0001).
Furthermore, only one participant (1.3%) resumed prophylaxis post-infusion.
Giroctocogene fitelparvovec was found to be well tolerated in the trial.
Although 49.3% of subjects experienced transient elevated FVIII levels of 150% or higher, these did not impact the efficacy and safety.
The gene therapy features a bio-engineered AAV6 capsid, and a modified B-domain deleted human coagulation FVIII gene.
The US Food and Drug Administration (FDA) has granted fast track and regenerative medicine advanced therapy designations for the therapy earlier, which also obtained orphan drug designations in the US and the European Union.
Pfizer Research and Development Internal Medicine and Infectious Diseases senior vice-president and chief development officer James Rusnak said: “We are very pleased with these positive results from the Phase III AFFINE study demonstrating the safety and efficacy of our one-time gene therapy candidate for people with haemophilia A.
“We look forward to advancing this latest innovation to help address the medical and treatment burden associated with frequent and time-consuming IV infusions or injections, building on Pfizer’s more than 40-year effort to advance haemophilia treatment.”
The latest development comes after the company reported findings from the Phase II MagnetisMM-3 trial of ELREXFIO in patients with relapsed or refractory multiple myeloma.
