Rare diseases

An Orphan Drug is a medicinal product developed for the treatment of a rare disease.

In Europe, the official definition of a rare disease is a disease affecting less than 5 per 10, 000 inhabitants, is fatal, or severely debilitating. Currently over 6000 rare diseases are known, which means that several million people are affected all over the world –over 25 million in Europe alone.

Today, treatment exists for only 200-300 of these 6000 diseases. Rare diseases are often genetic, meaning that newborns, children, and young adults often are affected. There’s always a risk that when a baby is born with a rare disease, a correct diagnosis may not be made and appropriate treatment may not be given.

To provide care for these “forgotten patients” and to encourage pharmaceutical and biotechnology companies to invest in treatment for rare diseases, Governments have created various legal and financial incentives. In 1983 the Orphan Drug Act was introduced in the US and in 1999 the “Regulation on orphan medicinal products” was approved by the European Parliament. This regulation grants, among other things, a ten-year market exclusivity for any new orphan drug.

Noventia Pharma is committed to develop and provide Orphan Drugs for the treatment of Rare Diseases in particular for CNS patients (Wilson Disease).

One of our Company key point mission, is the conviction that every patient has the right to the best possible treatment, especially patients suffering from a rare disease.