Pfizer/PFE.us (Pfizer/PFE.us) announced that the US FDA has approved the single-use gene therapy Beqvez (fidanacogene elaparvovec) it has developed to treat adults with moderate to severe hemophilia B aged 18 or above.
The Zhitong Finance App learned that Pfizer/PFE.us (Pfizer/PFE.us) announced that the US FDA has approved the single-use gene therapy Beqvez (fidanacogene elaparvovec) it has developed to treat adult patients with moderate to severe hemophilia B aged 18 or above. These patients are receiving coagulation factor IX (FIX) prophylaxis treatment, or have had current or past life-threatening bleeding, or have had serious spontaneous bleeding events repeatedly. Tests approved by the US FDA did not detect neutralizing antibodies targeting adeno-associated virus serotype rh74var (aavrh74var) capsid in these patients.
According to reports, hemophilia B is a life-threatening degenerative disease. Patients lack coagulation factor IX due to genetic mutations. Patients with this condition are prone to joint, muscle, and visceral bleeding, pain, swelling, and joint damage. Current treatment includes regular intravenous infusion of coagulation factor IX every week or several times a month for a lifetime. It may take more than 100 treatments a year to temporarily replace or supplement low levels of coagulation factors.
Beqvez is a novel gene therapy under development containing bioengineered adeno-associated virus capsids and highly active variants of the FIX gene. For patients with hemophilia B, the goal of this gene therapy is to enable them to produce their own FIX protein through a single treatment rather than requiring regular intravenous FIX infusions as is currently the standard treatment. Pfizer received Beqvez from Spark Therapeutics in December 2014 for a $20 million upfront payment. Health Canada (Health Canada) approved the listing of Beqvez in January of this year, and the European Medicines Agency (EMA) is currently reviewing this treatment.
In addition to Beqvez, Pfizer currently has two other gene therapies, giroctocogene fitelparvovec and fordadistrogene movaparvovec, which are in phase 3 clinical trials to treat patients with hemophilia A and Duchenne muscular dystrophy (DMD), respectively. In addition, Pfizer is also conducting a phase 3 trial to examine the tissue factor pathway inhibiting the monoclonal antibody marstacimab for the treatment of patients with hemophilia A and B with (or without) coagulation factor inhibitors in their bodies. Currently, the US FDA and EMA are reviewing Marstacimab's marketing license application.