Largest Phase III hemophilia trial produces positive results

pharmafile | January 11, 2022 | News story | Manufacturing and Production  

BioMarin Pharmaceutical has revealed positive results from its ongoing Phase III GENEr8-1 trial on the viability of valoctocogene roxaparvovec as a treatment for patients diagnosed with severe hemophilia A.

The study analysed 134 adult participants, making it the largest trial of its kind to date. In the second year of the trial, the researchers observed that valoctocogene roxaparvovec caused an 85% reduction in the annualised bleeding rate (ABR), or by 4.1 treated bleeds per year, from a baseline of 4.8.

The drug also reduces the annualised Factor VIII infusion rate by 98%, or 133 infusions per year, from the baseline data. The mean annualised infusion rate throughout the duration was 2.6, starting at 1.5 in year one and then 3.4 in year two. At the end of the second year, the participants demonstrated a mean endogenous Factor VIII activity level of 23 IU/dL (measured by chromogenic substrate assay) and 36.1 IU/dL.

Hemophilia A is characterised by a lack of Factor VIII protein for blood clotting, putting those suffering from it at risk of even the most mild injuries. Sufferers can also experience painful bleeds and into the joints or muscles. Patients with the severe form of the disease make up about 50% of the hemophilia A population. About 10,000 people suffer from this disease.

“A potential single treatment that provides a durable response for years could be a game-changer by offering a transformative treatment choice beyond existing therapies and addressing an unmet medical need for people with hemophilia A,” said Dr Steven W Pipe, an investigator in the Phase III study and professor of paediatrics and pathology at the University of Michigan, in a statement. 

Lina Adams

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