The Food and Drug Administration (FDA) has expanded the approval of Xyntha (antihemophilic factor [recombinant]; Pfizer) to include routine prophylaxis to reduce the frequency of bleeding episodes in patients with hemophilia A. Previously, the product had only been approved for on-demand treatment and perioperative management.
Xyntha works by temporarily replacing the missing clotting factor VIII that is needed for effective hemostasis. The new approval was based on data from clinical studies in which 102 patients with hemophilia A received Xyntha for routine prophylaxis; annualized bleeding rate (ABR) during routine prophylaxis was then compared to on-demand treatment alone.
Results showed that in patients aged ≥12 years, 45% (n=42/93) reported no bleeding while on routine prophylaxis; the mean (±SD) total ABR during routine prophylaxis was reported to be 3.6 ±5.18 (median 1.9, range 0.0-23.2). Moreover, the mean ABR for patients during routine prophylaxis was observed to be 89% lower than the mean ABR for patients during on-demand treatment.
Among patients younger than 12 years, 50% (n=4/8) reported no bleeding while on routine prophylaxis; the mean (±SD) total ABR during routine prophylaxis was reported to be 1.5 ±2.2 (median 0.6, range 0.0-6.2). The mean ABR was found to be 97% lower when compared with on-demand treatment.
Xyntha is available as lyophilized powder in single-use vials containing nominally 250, 500, 1000, or 2000 IU.
For more information visit xyntha.com.
- Xyntha [package insert]. Philadelphia, PA: Pfizer; 2020.
- Food and Drug Administration (FDA) Supplement Approval. https://www.fda.gov/media/141229/download. Accessed August 19, 2020.