The AFFINE study evaluating giroctocogene fitelparvovec, a gene therapy for moderate to severe hemophilia A, has achieved significant results. The study’s primary objective of non-inferiority and superiority in total annualized bleeding rate (ABR) compared to routine Factor VIII (FVIII) prophylaxis treatment was met.

After a single infusion, giroctocogene fitelparvovec significantly reduced mean total ABR by 98.3%, with 84% of participants maintaining FVIII activity above 5% at 15 months post-infusion. The majority of participants had FVIII activity of at least 15%.

The gene therapy was generally well-tolerated, with transient elevated FVIII levels observed in some participants without affecting efficacy or safety. Treatment-related adverse events were manageable and resolved with clinical intervention.

Giroctocogene fitelparvovec aims to provide a one-time infusion that enables individuals with hemophilia A to produce their own FVIII, potentially eliminating the need for frequent intravenous infusions or injections. This investigational therapy has the potential to significantly improve the quality of life for those living with hemophilia A by reducing treatment burden and providing superior bleed protection.

Further research is ongoing to evaluate the long-term efficacy and safety of giroctocogene fitelparvovec. The findings from the AFFINE study have sparked excitement and optimism in the medical community, suggesting a potentially transformative treatment for hemophilia A.

Source link: http://www.businesswire.com/news/home/20240724525239/en/Pfizer-Announces-Positive-Topline-Results-From-Phase-3-Study-of-Hemophilia-A-Gene-Therapy-Candidate

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Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.