Sanofi has received approval from the US Food and Drug Administration (FDA) for ALTUVIIIO [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl], for adults and children with haemophilia A.
Co-developed with Sobi, the factor VIII replacement therapy is claimed as the first-and-only treatment that delivers normal to near-normal factor activity levels. Compared to prior factor VIII prophylaxis, the therapy is said to result in substantial reductions in bleeds with once-weekly dosing,
Previously referred to as efanesoctocog alfa, ALTUVIIIO is approved for routine prophylaxis and on-demand treatment to control bleeding episodes and for perioperative management as well.
Sanofi CEO Paul Hudson said: “Today’s approval of ALTUVIIIO allows patients and physicians to reimagine living with haemophilia. The high sustained factor activity levels that can be achieved with ALTUVIIIO have the potential to change the haemophilia landscape.
“For the first time, with a once-weekly dose, powerful bleed protection is a reality for patients. Significant shifts in treatment paradigms that improve people’s lives, like ALTUVIIIO, are what we have committed to delivering at Sanofi.”
The FDA approval was based on the findings of the XTEND-1 phase 3 study.
As per the data, the once-weekly treatment prophylaxis with the investigational therapy for people with severe haemophilia A met the primary endpoint and significantly reduced bleeding.
The data from the late-stage study revealed a median annualised bleeding rate of 0.0 and a mean annual bleeding rate of 0.7, the France-based Sanofi said.
Additionally, the investigational candidate met the key second endpoint with a substantial reduction of 77% in annual bleeding rate compared to prior factor prophylaxis based on an intra-patient comparison.
The US health regulator had granted priority review to the factor VIII therapy in August 2022, while setting itself a target of taking a decision on its approval by the end of February 2023. Efanesoctocog alfa was also given breakthrough therapy status in May 2022, fast track status in February 2021, and orphan drug designation in August 2017.
In a separate development, the French pharmaceutical firm has reported new data for tolebrutinib, its investigational brain-penetrant oral Bruton’s tyrosine kinase (BTK) inhibitor.
The BTK inhibitor, intended for the treatment of multiple sclerosis (MS), showed a significant effect on vital immune mediators that could drive disease progression within the central nervous system (CNS), said Sanofi.