A regulatory strategy for the use of MP1032 in pediatric patients

By Dr. Volker Blust, Founder of PHOX Consulting e.U.

This is the second article within our series of six covering different aspects of work behind the scenes in the iMPAct project. In today’s article, we explore the role of regulation within the drug development process. We delve into the nuances on why the iMPAct project prepares the necessary regulatory work to allow for initiation of clinical trials in pediatric patients, and subsequently marketing approval. Children are a vulnerable group and therefore require specific regulatory rules with a careful risk-benefit assessment.

For most drugs that are being developed for both adults and children, initial clinical trials are primarily conducted in adults. Pediatric patients are typically excluded during early clinical development due to the risk of unknown adverse effects. Among many other reasons, children are particularly vulnerable because -by law- they cannot give informed consent themselves, and they might not be able to report adverse effects in a timely and sufficient manner. If proven safe and efficacious in adults, young patients are step-wise included in designated clinical studies.

While scientific studies indicate that the effects of COVID-19 appear to be milder in most children, children with compromised immune system have shown an increased risk of severe COVID-19 [1]. Moreover, in some rare cases SARS-CoV-2 infection can lead to the development of serious conditions such as long COVID or post-infectious inflammatory syndrome (PIMS) [1]. This illustrates that also children have an urgent need for new COVID-19

therapies and should also be able to participate in clinical trials against COVID-19, as this disease affects everyone.

To ensure that children have access to new treatment options (including COVID-19 medications), the European Medicine Agency (EMA) made it mandatory to prepare a designated pediatric development plan before the new medicine can enter the market. This plan is known as the pediatric investigation plan (PIP) and it is reviewed and approved by EMA’s Pediatric Committee (PDCO). The PIP represents an independent development plan for a defined indication and the pediatric patient population, elaborating timings and necessary measures to allow for marketing of the medicinal product. Once agreed by the PDCO, a PIP must be followed, and any deviation must be justified.

As children are not simply small adults, it is essential to look for and to exclude specific side effects and determine whether there is a need for further studies to cover safety and efficacy for children.

Children also have special requirements when it comes to the formulation of medicines. Young patients can for instance struggle with swallowing preparations initially developed for adults. Therefore, alternative formulations need to be found. This may involve the development of smaller capsule sizes or the switch to a liquid preparation. Currently MetrioPharm is looking into various options to make MP1032 accessible to very young patients, including newborns.

Within the iMPact project preparation of the PIP is an interdisciplinary task including experts from regulatory, chemistry manufacturing and control (CMC), nonclinical development and clinical development.

MetrioPharm submitted the PIP in September 2022 and successfully passed formal validation, confirming that all necessary data for data evaluation has been received by the PDCO.

Our next article: SARS-CoV-2 strains change- what does that mean for MP1032’s efficacy?

As the pandemic evolves and the SARS-CoV-2 strains change, it is becoming increasingly important to also test the compound’s efficacy against these new strains. In our next article Christian Setz, Scientific Managing Director of ImmunoLogik, explains how they test MP1032’s effects against different strains of SARS-CoV-2.

Posted on the 24th of November 2022.

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References:

[1] A.R. Howard-Jones et al., J Pediatr Child Health 2022  https://onlinelibrary.wiley.com/doi/10.1111/jpc.15811

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