Protalix BioTherapeutics Inc (NYSE: PLX) and Chiesi Farmaceutici S.p.A. have provided an update on the clinical development of pegunigalsidase alfa (PRX–102) for the proposed treatment of Fabry disease.
PRX–102 is currently being studied in Phase 3 BALANCE trial evaluating the safety and efficacy of 1 mg/kg of PRX–102 dosed every two weeks compared to agalsidase beta (Fabrazyme).
The primary endpoint of the study is the comparison of mean annualized changes (slope) of the eGFR (CKD-EPI) after completion of at least 12 months of treatment between the two treatment arms.
The initial top-line results show that the lower boundary of the confidence interval for the mean difference between the two treatments was below the non-inferiority margin pre-specified in the Intention to Treat analysis set and above such limit in the Intention to Treat analysis set.
At the time of this analysis (n=77), two patients discontinued participation due to treatment-emergent adverse events - one discontinued participation due to a related adverse event. No deaths were registered.
Unblinded final data are anticipated in Q2 of 2022 after all remaining patients have completed the 24-month treatment period.
Despite reporting safety issues, the company will move forward with marketing application in Europe PRX–102 in Fabry disease.
Regarding the regulatory process in the U.S., the companies plan to submit a Type–A meeting request with the FDA to discuss the path for approval of PRX–102.
In April, the companies received FDA Complete Response Letter for PRX‑102 for Fabry disease, though no safety concerns were noted in the letter.
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