Drugs

Sanofi/Sobi Plan BLA on Improved Factor VIII

Sanofi and partner Swedish Orphan Biovitrum (Sobi) have announced what they call positive topline results from the pivotal XTEND-1 Phase 3 study evaluating efanesoctocog alfa (BIVV001) in previously treated patients 12 years of age and older with severe hemophilia A. The companies say the data will support regulatory submissions at FDA and around the globe beginning later this year.

The study met the primary endpoint — demonstrating a clinically meaningful prevention of bleeds in people with severe hemophilia A receiving weekly prophylaxis with efanesoctocog alfa over 52 weeks. “The median annualized bleeding rate (ABR) was 0 with a mean ABR of 0.71,” they say. “The key secondary endpoint was also met, demonstrating once-weekly efanesoctocog alfa was superior to prior prophylactic factor VIII replacement therapy, showing a statistically significant reduction in ABR based on intra-patient comparison. Efanesoctocog alfa was well-tolerated, and inhibitor development to factor VIII was not detected. The most common treatment-emergent adverse events (>5% of participants overall) were headache, arthralgia, fall, and back pain.”

Sanofi and Sobi say efanesoctocog alfa is a novel recombinant factor VIII therapy that is designed to extend protection from bleeds. “It builds on the innovative Fc fusion technology by adding a region of von Willebrand factor and XTEN polypeptides to extend its time in circulation,” they say. “It is the first investigational factor VIII therapy that has been shown to break through the von Willebrand factor ceiling, which imposes a half-life limitation on current factor VIII therapies.”

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome