Pfizer Begins Phase 3 Study for Investigational Hemophilia B Gene Therapy
Pfizer has initiated a pivotal Phase 3 study to evaluate the safety and efficacy of its investigational gene therapy, fidanacogene elaparvovec, for the treatment of hemophilia B.
NEW YORK & PHILADELPHIA – Pfizer Inc. has launched a pivotal Phase 3 study to evaluate the safety and efficacy of its investigational gene therapy, fidanacogene elaparvovec, for the treatment of hemophilia B, a rare bleeding disorder. The clinical trial will include a lead-in period to gather data on patients' current factor IX replacement therapy, which will serve as an internal control group.
The Phase 3 program was initiated following Pfizer's assumption of Spark Therapeutics' hemophilia B gene therapy program. Fidanacogene elaparvovec is an investigational adeno-associated virus (AAV) vector designed to deliver a gene for functional human coagulation factor IX. The therapy aims to enable patients to produce factor IX internally, potentially freeing them from the need for regular infusions.
This milestone marks a significant step in developing a novel treatment for individuals with this inherited bleeding disorder. Hemophilia B results from a deficiency in factor IX, a protein crucial for blood clotting, and can lead to severe and potentially life-threatening bleeds.
Pfizer and Spark Therapeutics have collaborated on this gene therapy candidate since 2014. Previous Phase 1/2 trial data has shown promising results regarding the therapy's effectiveness and safety profile, with no serious adverse events reported. Pfizer will now oversee all subsequent studies, regulatory activities, manufacturing, and global commercialization.