The US Food and Drug Administration (FDA) has confirmed proteinuria as an acceptable primary endpoint in Dimerix ‘s Phase III ACTION3 trial of DMX-200 for treating focal segmental glomerular sclerosis (FSGS).
Following a Type C meeting with the company in March this year, the FDA has made this decision for full marketing approval for the therapy.
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Dimerix noted that the FDA has recognised the potential of proteinuria as a primary endpoint, offering two possible metrics: the proportion of individuals achieving a defined proteinuria minimisation or the percentage change in proteinuria from baseline post two years of treatment.
At present, 183 out of 286 subjects have already been randomised/dosed in the trial.
This randomised, placebo-controlled, double-blind, multi-centre trial is assessing the safety and efficacy of the therapy in subjects on a stable dose of an angiotensin II receptor blocker (ARB).
They will be randomised and given either the 120 mg capsule of the therapy or a placebo, with interim analysis points tailored to capture proteinuria and kidney function (eGFR slope) evidence to back marketing approval.
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By GlobalDataThe agency’s agreement with Dimerix’s proposal signifies a collaborative effort to establish this primary endpoint, which is expected to show less variability than estimated glomerular filtration rate (eGFR) in trials.
Based on the discussions held at the meeting, the company is collaborating with the PARASOL working group to further analyse current data, aiming to define a meaningful endpoint for accelerated approval in FSGS subjects in the trial.
A blinded statistical power analysis of the trial is planned by the company, following the further PARASOL work and pre-specification of primary and secondary endpoints in line with the FDA’s guidance.
The analytical work by the PARASOL group is expected to be completed within three to six months.
The meeting with the FDA has yielded a favourable outcome for the therapy, building on Dimerix’s pre-clinical and clinical analysis, which has shown promising changes in proteinuria levels from their initial values.
The agency noted that it is ready to engage in discussions regarding potential endpoints that could facilitate the accelerated approval application and substantiate clinical efficacy.
Dimerix CEO and managing director Dr Nina Webster said: “As part of our study design, both eGFR and proteinuria data are being collected for a total of two years however, the FDA response on proteinuria potentially gives us an opportunity for earlier market entry by working with both PARASOL and FDA on an accelerated approval proteinuria endpoint for use in FSGS.”
