Dimerix (ASX:DXB) has confirmed the outcomes of a meeting with the US FDA to discuss the paediatric plan for its DMX-200 in Focal Segmental Glomerulosclerosis (FSGS) patients under 18 years old.
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, and for those who are fortunate enough to receive a kidney transplant, 60 per cent of patients have reoccurring FSGS after first kidney transplant.
The company said that 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.
“No change in dose or regime is required for this population,” said the company, adding, “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.”
“The FDA have consistently encouraged Dimerix to develop DMX-200 for children given the very poor prognosis in this disease and lack of targeted therapies,” said CEO and managing director, Dr Nina Webster.
“This formal meeting with the FDA gave us a valuable opportunity to discuss our paediatric plans for DMX-200 in the US. We are very pleased to be able to open the study to adolescent patients later in the year, which may provide a viable treatment option for these younger patients, if approved.
“The inclusion of patients aged 12 and above will also increase the total pool of patients that could be recruited into Part 2 of the ACTION 3 study – potentially accelerating the rate of recruitment for the study.
“This initial discussion regarding adolescent enrolment in the ACTION3 study builds on the numerous constructive discussions we have had with key regulatory agencies worldwide.
“Following this meeting, we will continue discussions with the FDA to support development of new therapies in this population of adolescent patients who have very few therapeutic options currently available or in development,” added Dr Webster.
ACTION3 is a Phase 3 multicentre, 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 randomised to receive either DMX-200 (120 mg capsule twice daily) or placebo.