Pfizer’s Hemophilia A Gene Therapy Candidate Achieves Phase 3 Success

1. Phase 3 Success: Pfizer's gene therapy candidate, giroctocogene fitelparvovec, has demonstrated positive results in the Phase 3 AFFINE study for the treatment of adults with moderately severe to severe hemophilia A.
2. Bleeding Rate Reduction: The study showed a statistically significant reduction in annualized bleeding rate (ABR) compared to routine Factor VIII (FVIII) replacement therapy, with a mean ABR of 1.24 vs 4.73.
3. Superiority: Giroctocogene fitelparvovec demonstrated superiority in total ABR from week 12 through at least 15 months of follow-up post-infusion compared to prophylaxis.
4. Key Secondary Endpoints: The study met key secondary endpoints, including maintaining FVIII activity greater than 5% at 15 months post-infusion and a significant reduction in treated ABR.
5. Safety Profile: The treatment was generally well-tolerated, with transient elevated FVIII levels and some serious adverse events reported, but most resolved with clinical management.
6. Regulatory Designations: Giroctocogene fitelparvovec has received Fast Track, Orphan Drug, and Regenerative Medicine Advanced Therapy designations from the FDA.
7. Future Plans: Pfizer will discuss the data with regulatory authorities in the coming months and present additional data at upcoming medical meetings.