The US Food and Drug Administration (FDA) has granted marketing authorization to Pfizer (NYSE: PFE) for its gene therapy Beqvez (fidanacogene elaparvovec) for the treatment of adults with moderate-to-severe hemophilia B. The one-time treatment is indicated for patients using factor IX prophylaxis therapy, those experiencing life-threatening hemorrhages, or those with repeated, serious spontaneous bleeding episodes. The therapy has recently been approved in Canada and is currently under review by European regulators.
The FDA’s decision was supported by positive results from a Phase III trial, which demonstrated non-inferiority in the annualized bleeding rate (ABR) compared to a standard prophylaxis regimen with factor IX. The trial also showed that Beqvez eliminated bleeds in 60% of patients in the experimental arm, compared to 29% in the control arm.
Patients who participated in the study will be followed for up to 15 years to assess the long-term safety and efficacy of Beqvez, offering a comprehensive evaluation of the gene therapy’s performance over an extended period.- Flcube.com
