New Potential Fabry Disease Treatment Requires Less Frequent Infusions

The typical treatment for Fabry disease is Fabrazyme which is an enzyme replacement therapy (ERT). This therapy must be administered to the patient every two weeks. A new ERT created by Protalix BioTherapeutics has shown better stability than Fabrazyme in a recent Phase 3 trial. In addition, it only has to be administered once every four weeks. This new therapy is called PRX-102. Its ongoing Phase 3 trial is called BRIGHT.

BRIGHT

This trial was created to evaluate PRX-102 as a therapy for Fabry patients in comparison to the typical Fabrazyme treatment. Therefore it enrolled patients who were previously treated with Fabrazyme. 28 patients are currently enrolled in the trial which will evaluate the efficacy, safety, and stability of PRX-102. This trial is still enrolling (the estimated total number of participants is 30) but interim results have shown a lot of promise for this new ERT.

PRX-102 is unique because it was created using the ProCellEx platform which utilizes plant cells as opposed to mammalian cells to create therapeutic proteins. PRX-102 compensates for the enzyme Fabry patients lack called alpha-galactosidase A.

All participants in this trial have been given 2 mg/kg of the treatment once every 4 weeks. They will receive PRX-102 for 12 months total. So far 15 patients have reached the 9 month mark.

In preliminary analysis, researchers found that PRX-102 was not only stable but active during the entire four-week period. After just 28 days from the time of infusion, blood concentrations were increased 7 fold compared to Fabrazyme.

These results were evident even in patients who had pre-existing antidrug antibodies. In fact, PRX-102 levels remained higher than Fabrazyme levels even when these antibodies were present.

This treatment showed safety and tolerability with the most common AEs being parathesia and reactions related to infusion itself.

Looking Forward

These results are so encouraging not only because of the stability showed by PRX-102 but because it can be dosed half as frequently as Fabrazyme. This in and of itself could significantly improve the quality of life of Fabry patients.

Patients who complete all 12 months of treatment with PRX-102 will be eligible to continue the therapy through an open-label extension study. This study will investigate the safety and efficacy of PRX-102 long-term.

You can read more about this new potential ERT for Fabry disease here.


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