Dimerix Limited confirmed the outcomes of a meeting with the US Food and Drug Administration (FDA) to discuss the paediatric plan for DMX-200 in Focal Segmental Glomerulosclerosis (FSGS) patients under 18 years old. The FDA has consistently recommended the evaluation of DMX200 in paediatric patients, and at the meeting, the FDA confirmed that the inclusion of paediatric patients aged 12 years or older (adolescent) was appropriate in the current global ACTION3 Phase 3 study of DMX-200 for patients with FSGS, which recognises the appropriate safety profile exhibited by DMX-200. No change in dose or regime is required for this population. Importantly, the meeting also provided clarity on the remaining development of DMX-200 required for paediatric patients younger than 12 years old with FSGS through to market approval, broadening the label indication and pool of patients for treatment
if approved. FSGS is a rare disease that attacks the kidney's filtering units causing irreversible scarring. This leads to permanent kidney damage and eventual end-stage kidney failure, requiring dialysis or transplantation. FSGS affects both adults and children as young as two years old, 9 and for those who are fortunate enough to receive a kidney transplant, 60% of patients have reoccurring FSGS after first kidney transplant.10 Furthermore, FSGS is one of the leading causes of kidney failure in children, with 20% of child nephrotic syndrome cases caused by FSGS.1 At this time, there are no drugs specifically approved for FSGS anywhere in the world, leading to limited treatment options and poor prognosis. About ACTION3 Phase 3 clinical study: The Phase 3 trial, which is titled "Angiotensin II Type 1 Receptor (AT1R) & Chemokine Receptor 2 (CCR2) Targets for Inflammatory Nephrosis" - or ACTION3 for short, is a pivotal (Phase 3), multi- centre, randomised, double-blind, placebo-controlled trial of the efficacy and safety of DMX-200 in patients with FSGS who are receiving a stable dose of an angiotensin II receptor blocker (ARB). Once the ARB dose is stable, patients, aged 18 to 80 years (broadening to 12 to 80 years), will be randomized to receive either DMX-200 (120 mg capsule twice daily) or placebo.