Protalix BioTherapeutics has recently completed the treatment portion of their Phase III BRIGHT trial, which is investigating PRX-102 as a treatment for Fabry disease. The data is still being collected and analyzed, and COVID-19 has presented a number of challenges throughout the trial, but researchers are excited by the results as of now.

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

The BRIGHT clinical trial is an open-label, switch over study that is meant to evaluate the safety, efficacy, and pharmacokinetics of PRX-102. Participants were given two mg/kg of the therapy every four weeks through an IV for 52 weeks.

Completing the treatment portion of this trial is an important milestone in developing PRX-102, and medical professionals and patients are both very excited to see the results. They should be available in the final quarter of 2020. Researchers are also waiting on the results of an earlier trial of PRX-102, the Phase III BALANCE study, and they already have the results, which were positive, of the BRIDGE trial.

Hopefully this medication will be able to better the lives of Fabry disease patients. Read more about it here.