Dimerix Limited (ASX: DXB) has received notification that the Paediatric Committee (PDCO) of the European Medicines Agency (EMA) has accepted its Paediatric Investigation Plan (PIP) for the development of DMX-200 for focal segmental glomerulosclerosis (FSGS).
Dimerix is biopharmaceutical company with Phase 3 clinical studies in inflammatory diseases.
A PIP is a mandatory development plan aimed at ensuring that the necessary data is obtained to support the registration and use of new medicines for children in the European Union (EU).
The PIP addresses the entire paediatric development program for DMX-200 in FSGS and provides a clear framework for the development and registration of DMX-200 for paediatric patients with FSGS in the EU.
The positive final opinion from PDCO ensures that the company’s clinical studies, including its Phase 3 clinical study of DMX-200 in FSGS, will be run in accordance with PDCO expectations for future product approval in children.
The PIP covers all studies needed for registration of DMX-200 in the paediatric population and no safety concerns were raised. The safety package was accepted, and no further non-clinical studies are required.
Paediatric patients from 12 to 17 years of age will be included in current ACTION3 study in patients with FSGS, in line with US FDA advice, and in silico modelling, simulation and extrapolation of paediatric data from ACTION3 will be used to support a confirmatory small open- label study in paediatric patients from one to 11 years of age.
A successful clinical trial in paediatrics will allow Dimerix to file for an additional market authorisation (MAA) in Europe, thereby potentially expanding market access in a paediatric population where no treatment for FSGS exists and associated healthcare costs are high.
This approval from the PDCO of the EMA will allow Dimerix to develop DMX-200 for paediatric patients, including adolescents in the current ACTION3 study,” Dr Nina Webster, CEO and Managing Director of Dimerix, said.
“DMX-200 represents a potentially safe and effective new treatment option in these patients where there is a substantial unmet clinical need. We look forward to including adolescent patients in our existing ACTION3 study and to working with parents and children aged 1-11 years old following a positive outcome to ACTION3.”
Focal Segmental Glomerulosclerosis is one of the leading causes of End Stage Renal Disease (ESRD) in children and is associated with up to 20% of all new cases of Nephrotic Syndrome in children each year.
The single Phase 3 study in FSGS patients has two interim analysis points built in that are designed to capture evidence of proteinuria and kidney function (eGFR slope) during the study, aimed at generating sufficient evidence to support accelerated marketing approval. Part 1 of the study will conclude after the first interim analysis, once 72 patients have completed 35 weeks treatment. The study will continue seamlessly into Part 2 of the study, which has the potential to include adolescents with FSGS.
FSGS Phase 3 Study – ACTION3
The Phase 3 study is a pivotal (Phase 3), multi-centre, randomised, double-blind, placebo-controlled study 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 will be randomized to receive either DMX-200 (120 mg capsule twice daily) or placebo.
