The FDA has granted priority review to haemophilia A treatment efanesoctocog alfa (BIVV001) jointly developed by Sanofi and Swedish Orphan Biovitrum (Sobi). The FDA will decide to approve this therapy early next year.
The priority review awarded to efanesoctocog alfa is based on the promising data obtained from the XTEND-1 study as a treatment option for haemophilia patients. The phase-III, open-label, non-randomized XTEND-1 study enrolled 159 patients (age 12 years or more) with severe haemophilia. The clinical study consisted of two arms, i.e. prophylaxis Arm A cohort (patients with factor VIII prophylaxis received intravenous efanesoctocog alfa once a week) and on-demand Arm B cohort (who had received factory VIII therapy received on-demand efanesoctocog alfa for 26 weeks and then switched to once weekly prophylaxis for another 26 weeks).
The findings of the XTEND-1 study showed that the once-weekly use of efanesoctocog alfa led to the median and mean annualised bleeding rates (ABRs) of 0.00 and 0.71, respectively. Further, the therapy was efficient in preventing bleeding events as its use showed an ABR reduction of 77% (0.69) compared to prior factor VIII prophylaxis (2.96). Efanesoctocog alfa was well tolerated with adverse events including headache, arthralgia, fall and back pain.
Overall, the findings suggest the superior efficacy of efanesoctocog alfa in reducing ABRs.