Rare disease

Toleranzia’s primary focus is to develop a therapy for myasthenia gravis, which is a rare autoimmune disease.

Rare diseases are conditions that affect not more than 200,000 people in the United States or fewer than 5 people per 10 000 people in Europe and lead to significant disabilities.

Toleranzia’s therapy for myasthenia gravis has been granted orphan drug designation by the Federal Drug Administration (FDA) in the US. Orphan drug designation can be obtained for medicinal products intended for a safe and effective treatment, diagnosis or prevention of rare diseases. Orphan drug designation entitles to significant support from the EMA and FDA; the regulatory authorities that audit designs and results of clinical trials in the EU and the US, and that are responsible for the approval of drugs developed for use within the EU and the US. Orphan drug designation also means that a product is given special marketing exclusivity following market launch.

Several autoimmune diseases are classified as rare. Toleranzia’s focus is currently on the development of disease-specific therapies for myasthenia gravis and ANCA vasculitis. In addition to these rare autoimmune diseases, there are several other rare autoimmune diseases that Toleranzia potentially could incorporate into its pipeline.