Targeting Critical Unmet Medical Needs
Advicenne is a specialty pharmaceutical company focused on the development of orphan medicines for patients of all ages in the fields of nephrology and neurology.
Our development approach leverages our very strong relationships with Key Opinion Leaders to identify areas of critical unmet medical need either due to a lack of adapted products or the absence of approved products.
Furthermore, we leverage our know-how, expertise and knowledge in the field of formulation, pharmaceutical and clinical development to develop products in the appropriate dosage form with the aim of improving the lives of all our patients, from early childhood and through adulthood by enhancing the management of their disease.
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
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.