50% of the people affected by rare diseases are children*
80% of rare diseases have a genetic component*
In 50% of the cases, orphan diseases affect young children and are responsible for 35% of deaths in the first year of life. Since 80% of orphan diseases have a genetic component, they can appear at any point during a person’s life. *
Because orphan diseases affect people from early childhood to adulthood, Advicenne designs and develops medicines in dosage forms that are suitable for both children and adults. Therefore, Advicenne’s development approach is highly differentiated from other companies working to develop new drugs.
We develop small-size dosage forms of therapeutics and offer flexible weight-dependent dosing for either children or adults. Weight-dependent dosing increases safety and reduces the risk of over or under dosing.
Our therapeutic dosage forms are tasteless and easy to administer enabling both improved short and long-term therapeutic outcomes which results in greatly enhanced patient compliance. Advicenne’s dosage forms also aims to improve the treatment experience for both children and adults.
* Neurological Rare Disease « Special Report », February 2015
Children are not just small adults
Many medicinal products are not currently available in formulations suitable for administration to the pediatric population. Consequently, crushing tablets or opening capsules to administer a smaller dose is a common practice.
By manipulating the adult dosage forms, the risk of over or under-dosing children is very high, along with the increased consequences of efficacy, tolerance, safety and compliance issues.