Study: Positive Results for Fabry Disease Treatment

Protalix BioTherapeutics has recently announced the results from the third phase of the BRIDGE trial, which evaluated pegunigalsidase alfa for the treatment of Fabry disease. As these results were positive, researchers are hopeful that this medication will be a viable treatment for those with Fabry disease.

About Fabry Disease

Fabry disease is a lysosomal storage disorder that occurs when globotriaosylceramide, a type of fat, builds up in the cells. It typically affects males. This accumulation can affect multiple organs throughout the body, but typically affects the kidneys and heart. As the buildup usually begins in childhood, symptoms will worsen as one ages. These symptoms include pain in the hands and feet, dull and cloudy vision, the inability to sweat, clusters of red spots on the skin, tinnitus, loss of hearing, and gastrointestinal issues. Heart attacks, strokes, and acute kidney damage may be complications. This disease is usually diagnosed after birth, as it is picked up in newborn screening exams. Treatments are symptomatic.

About the Study

Phase III of this study lasted for twelve months and was open-label, switch-over, and single arm. Researchers evaluated the safety and efficacy of 1 mg/kg of pegunigalsidase alfa administered once every two weeks throughout 22 participants.

Results of this study were positive, demonstrating improvements in renal function in both male and female participants. In fact, 20 patients decided to continue their treatment of pegunigalsidase alfa in an extension of this study.

This therapy is also being studied in two other ongoing phase III trials, the BALANCE and BRIGHT studies. The former is intended to evaluate the effects of this treatment on renal function, while the latter assesses efficacy, safety, and pharmacokinetics.

Hopefully these trials will follow in the BRIDGE study’s footsteps with positive results, as this would offer Fabry disease patients more treatment options.

Find the source article here.


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