First full paediatric waiver for MS DMT: Lycalis strategy
You get a waiver ...
A client of Lycalis achieved a full waiver for its novel putative disease modifying drug (DMT) by leveraging Dr Wolf's unique cross-indication expertise in paediatric development programmes.
After a respective full waiver for the US iPSPs, the Paediatric Committee (PDCO) of the European Medicines Agency's (EMA) confirmed today a full waiver for the European paediatric requirements (PIP, PDCO Minutes).
This is the first full waiver for a novel putative disease-modifying drug granted in the absence of safety concerns.
The waiver is valid for all age groups from birth to <18 years.
While the medical need for the registration of safe and effective DMTs for children and adolescents remains pressing, conducting meaningful development programmes is increasingly challenging in face of the many competing paediatric programmes and the widespread off-label use of highly effective drugs registered for adults.
The waiver strategy developed by Dr Wolf could be of interest for companies with Phase II or Phase III programmes in MS.
The PDCO emphasised that "granting of a waiver for the condition [...] should not prevent the applicant from considering a development in the paediatric population in indications where there is a paediatric need. In principle according to the Paediatric Regulation, incentives for the development for use in the paediatric population are available even if a waiver has been granted in another condition."
Dr Wolf's cross-indication expertise in paediatric development programmes for CNS conditions spans from the 1990s with the conduct of the first and only completed placebo-controlled trial in childhood epilepsy to today's EU Paediatric Investigation Plans (PIPs) and US Pediatric Study Plans (iPSPs, PSPs) in MS and stroke.