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.
By Nick Paul Taylor
The U.S. Patent and Trademark Office issued a patent to MedTrace for their method of diagnosing the human heart via 15O-water PET. The patented method is the foundation of the company’s software aQuant, currently under development. Hendrik “Hans” Harms, PhD and Senior Scientist at MedTrace, and Jens Soerensen, Professor and Clinical Advisor to MedTrace, are the originators of the method.
Teresa Graham, currently head of global product strategy for Roche pharma, will become the division’s new CEO next month, Roche said Thursday. Simultaneously, Roche is elevating Levi Garraway, chief medical officer, to the executive committee.
Fierce Pharma has obtained internal documents and video of a town hall meeting conducted this week describing what J&J called a “comprehensive review” of its portfolio. Moving forward, J&J plans to operate its vaccines and infectious diseases outfits as one group, the executives explained.