Argenica Therapeutics (ASX:AGN) has achieved a significant regulatory milestone in Europe, securing a full paediatric waiver from the European Medicines Agency (EMA) for its lead drug candidate, ARG-007, in the treatment of acute ischaemic stroke (AIS).
The decision removes the requirement for Argenica to conduct paediatric clinical trials as part of its European approval process, thereby substantially streamlining the drug's development pathway.
The waiver applies across all paediatric age groups, from birth to under 18 years, and reflects the EMA’s assessment that paediatric studies in acute ischaemic stroke are neither feasible nor scientifically justified. Stroke overwhelmingly affects older adults, while paediatric cases are rare, making recruitment into meaningful clinical trials extremely difficult. In this context, the EMA concluded that mandatory paediatric trials would add cost, delay, and complexity without delivering clinical benefit.
Under European regulations, pharmaceutical companies are generally required to agree on a Paediatric Investigation Plan (PIP) before submitting a marketing authorisation application for an adult indication, unless a waiver is granted. By obtaining a Product-Specific Waiver, Argenica has cleared a key regulatory hurdle, allowing it to advance ARG-007 through the EMA approval pathway for adult stroke patients only.
From a development and commercial perspective, the outcome significantly de-risks Argenica’s European strategy. Paediatric stroke trials would have required complex, global study designs with long enrolment timelines and high operational costs. The waiver avoids this burden, preserving capital and resources for studies that directly support approval in adult acute ischaemic stroke and helping maintain alignment with projected regulatory timelines.
Argenica Managing Director Dr Liz Dallimore described the decision as a key milestone in the company’s global regulatory strategy, noting that it provides clarity and certainty as ARG-007 progresses through development. She added that the streamlined pathway is also expected to be viewed favourably by potential pharmaceutical partners evaluating the program’s risk profile and time-to-market.
ARG-007 is being developed as a neuroprotective therapy designed to reduce brain tissue death and limit secondary damage following an ischaemic stroke.