Developing medicines for children

Dr Isabelle Borghini-Fuhrer Director, Clinical Development, MMV

Children under the age of 5 continue to be the main victims of malaria. Yet with the exception of Coartem® Dispersible, developed under the Novartis/MMV partnership, few antimalarial medicines have been specifically formulated to provide this vulnerable group with age and weight-appropriate doses in a palatable form. MMV aims to address this imbalance.

Every new MMV-sponsored medicine developed is followed by a formulation specifically tailored to meet the needs of children. For example, child-friendly formulations of the recently approved Eurartesim® and Pyramax®, are under development and due to be submitted for the same stringent approval as their parent medicines over the next 2 years.

As once-a-day therapies, Eurartesim and Pyramax might be easier to use than the twice-a-day medicines currently available. Additionally, Eurartesim provides better and longer protection from new malaria infections than other ACTs1 and Pyramax is the first ACT to be approved for the treatment of the blood-stage of both P. falciparum and P. vivax malaria.

1. What are the main challenges when developing medicines for children?

Children cannot be considered miniadults. Their bodies are not fully developed, which means they are much more vulnerable to potential side effects. So the main challenge is really ethical; we cannot directly test a medicine in children without evidence of its safety and efficacy in adults. We cannot put children at risk. At the same time, given these physiological differences, it is critical that we do test the medicine and obtain safety and efficacy data in this vulnerable population.

Another challenge, particularly in antimalarial development for children, is the bitter taste of the medicines. This often results in the child vomiting and therefore rejecting the medicine.

2. How can we overcome these challenges?

Clinical development of a new medicine must begin in adults and then move to children. We start with the eldest children first and then progress to smaller ones. To overcome the issue of palatability we develop taste-masked or flavoured formulations.

3. What strategies are in place to minimize the time between the launch of an adult and a child formulation?

In the past, development of paediatric formulations would begin only after an adult medicine had been developed and approved. Coartem® was followed by Coartem® Dispersible and now Eurartesim will be followed by Eurartesim© Paediatric. We tried to speed up the process for Pyramax by developing the two formulations in parallel. However, this proved to be incredibly resource and time intensive, and so we focused first on the approval of the adult formulation. In the future, to accelerate the process, we plan to develop a single paediatric formulation that can also be used in adults.