In a major milestone, Dimerix has received endorsement by the European Medicines Agency (EMA) to include children in Europe in its pivotal ACTION3 trial.

Dimerix (ASX:DXB) has announced  that the Paediatric Committee (PDCO) of the EMA has accepted its Paediatric Investigation Plan (PIP) for development of DMX-200 for focal segmental glomerulosclerosis (FSGS).

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).

DXB said the PIP addresses the entire paediatric development program for DMX-200 in FSGS and provides a clear framework for 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.

DXB said 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 with no further non-clinical studies 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.

DXB said 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 1 to 11 years of age.

The company said a successful clinical trial in paediatrics will enable DXB to file for an additional market authorisation (MAA) in Europe, potentially expanding market access in a paediatric population where no treatment for FSGS exists and associated healthcare costs are high.

Leading cause of kidney disease in kids

FSGS is a leading cause 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 rare disease attacks the kidney’s filtering units causing irreversible scarring. The scarring leads to permanent damage and eventual end-stage kidney failure, requiring   dialysis or transplantation.

For those fortunate enough to receive a kidney transplant, 60% of patients have recurrent FSGS after the first kidney transplant.

“Paediatric nephrologists are desperate for new treatments for their patients, with no drugs registered for paediatric FSGS patients anywhere in the world and very new drugs under investigation for children,” DXB chief medical officer Dr Ash Soman said.

“We are actively engaged with networks of paediatric nephrologists and patient advocacy groups to generate high-quality data to support the registration of DMX-200 in this typically underserved population.”

In January the US Food and Drug Administration (FDA) confirmed adolescent children aged 12-17 could be recruited to DXB’s ACTION3 study.

Dimerix head welcomes PIP

DXB’s pivotal Phase 3 trial is titled Angiotensin II Type 1 Receptor (AT1R) & chemokine Receptor 2 (CCR2) Targets for Inflammatory Nephrosis or ACTION3 for short.

It is a multi-centre, randomised,  double-blind,  placebo-controlled  trial of the efficacy and  safety of DMX-200  inpatients with FSGS who are receiving a stable dose of an angiotensin II receptor blocker (ARB).

Once the ARB dose is stable, patients will be randomised to receive either DMX-200 (120mg capsule twice daily) or a placebo.

CEO and managing director Dr Nina Webster said approval from the PDCO of the EMA will allow DXB to develop DMX-200 for paediatric patients, including adolescents in the current   ACTION3 study.

“DMX-200 represents a potentially safe and effective new treatment option in these patients where there is a substantial unmet clinical need,” she said.

“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.”

The total FSGS market size is estimated to be more than US$3 billion by 2032 driven by  ~220,000  FSGS sufferers across seven major  markets and premium orphan drug pricing.

DMX-200 has received Orphan Drug Designation with the FDA, EMA, and UK, allowing potential fast track of commercialisation if successful.

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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