UK Regulatory Agency has given the greenlight for Dimerix’s DMX-200 Paediatric Investigation Plan
The UK Paediatric Investigation Plan aims to ensure sufficient data is generated in Dimerix’s ACTION Phase 3 trial to allow for marketing approval for children, should it be successful
This milestone marks significant progress towards potentially addressing FSGS kidney disease in children globally
Special Report: Dimerix has achieved another key milestone with the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) accepting its Paediatric Investigation Plan (PIP) for the development of lead asset DMX-200 for focal segmental glomerulosclerosis (FSGS).
Clinical-stage biopharmaceutical company Dimerix (ASX:DXB) says the its ACTION Phase 3 study will now be opened up to include children from ages 12-17 in the UK.
DXB says a successful clinical trial in paediatrics would enable the company to file for an extension to any market authorisation (MAA) in the UK, potentially expanding market access in a paediatric population where no treatment for FSGS exists and associated healthcare costs are high.
The approved PIP in the UK is aligned with the previously endorsed European Medicines Agency (EMA) PIP and the US FDA advice.
DXB says FSGS stands as main cause of end stage renal disease (ESRD) among children, contributing to up to 20% of new Nephrotic Syndrome cases in children annually.
PIP mandatory in UK
In the UK, a PIP is mandatory, serving as a developmental roadmap to secure essential data for the registration and use of new medications for children.
This PIP encompasses the entire paediatric development program for DMX-200 in FSGS, offering a structured framework for DMX-200’s development and registration for paediatric FSGS patients in the UK.
The favourable and final opinion from the MHRA ensures that DXB’s ACTION Phase 3 trial in FSGS, will adhere to MHRA expectations to allow for future approval of the product for paediatric use, contingent upon successful outcomes.
The UK PIP developed by DXB includes all necessary studies for DMX-200 registration in the paediatric demographic, with no raised safety concerns.
DXB says the safety package was accepted with no further non-clinical studies required.
Adolescents aged 12 to 17 will now take part in the ongoing ACTION Phase 3 study for FSGS patients in the UK.
Furthermore, in silico modelling, simulation, and extrapolation of paediatric data from the ACTION Phase 3 study will support a confirmatory small open-label study in paediatric patients aged 1 to 11.
Interim trial meets primary endpoint
Titled Angiotensin II Type 1 Receptor (AT1R) & Chemokine Receptor 2 (CCR2) targets for Inflammatory Nephrosis, or ACTION3 for short DXB’s Phase 3 trial is a multi-centre, randomised, double-blind, placebo-controlled study.
In March DXB announced the trial was successful in the pre-specified interim analysis of proteinuria (efficacy) endpoint from the first 72 randomised patients.
The interim analysis showed DMX-200 is currently performing better than the placebo in reducing proteinuria (using a statistical measure) in patients with FSGS in a significantly larger cohort than its prior Phase 2 trial of eight patients.
The full trial is expected to enrol ~286 patients, with a second interim analysis planned after the first 144 patients complete ~35 weeks of treatment.
The ongoing Phase 3 is a double-blind, randomised trial across multiple study sites in 11 countries, with the primary endpoints currently being both eGFR and proteinuria.
DXB chief medical officer Dr David Fuller says it is pleasing to see that the MHRA approved PIP aligns extremely well with the European EMA PIP and the US FDA advice.
“Our single Phase 3 clinical study, having successfully passed its first efficacy interim analysis is now being rapidly expanded to include new adult and paediatric sites, which will now allow recruiting children down to 12 years old as well as adults,” he says.
This article was developed in collaboration with Dimerix, a Stockhead advertiser at the time of publishing.
This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.
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