Sanofi, seeking to stem bleeding at hemophilia A unit, gets FDA priority review for rival to Roche

Sanofi, seeking to stem bleeding at hemophilia A unit, gets FDA priority review for rival to Roche

The hemophilia A market has a new date for the diary: Feb. 28. Officials at the FDA put the date on the agenda at Sanofi and Sobi by accepting the partners’ hemophilia A drug candidate efanesoctocog alfa for priority review.

Sanofi and Sobi established the investigational factor VIII therapy, also known as BIVV001, as a potential disruptor of the hemophilia A market when they released their phase 3 results in March. The data drop showed patients who received a weekly prophylactic dose of efanesoctocog alfa for 52 weeks had a median annualized bleed rate (ABR) of 0. The mean ABR came in at 0.71.

With the usual caveats about cross-trial comparisons, the results suggested efanesoctocog alfa can hold its own against BioMarin’s gene therapy Roctavian and improve on Roche’s bispecific antibody Hemlibra. Efanesoctocog alfa is on course to come to market before Roctavian in the U.S.

If the FDA approves efanesoctocog alfa by its Feb. 28 PDUFA date, Sanofi will have a shot at regaining the ground it lost when Hemlibra began eating into sales of Eloctate. The once-weekly, or less, subcutaneous dosing of Hemlibra enabled Roche to muscle in on the hemophilia A market. Efanesoctocog alfa, which is given intravenously once a week, could reduce Hemlibra’s convenience advantage and beat its efficacy.

“Efanesoctocog alfa can deliver close to normal factor activity levels for the majority of the week, potentially offering a new tier of protection. Such therapeutic benefits would represent important advances in unmet medical needs for people with hemophilia A and may transform the prophylactic treatment landscape,” the University of Michigan’s Steve Pipe, M.D., said in a statement.

While efanesoctocog alfa is now up for review at the FDA, Sanofi and Sobi are holding off on filing for approval in the EU until after they have data from the ongoing XTEND-Kids pediatric study. The partners expect to deliver the data and file for EU approval next year.

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