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What's Orphan Drug?

Orphan Drugs are pharmaceuticals for the diagnosis, prevention or treatment of rare diseases.

Pharmaceuticals are classical and chemical active substances as well as biological active substances, as for example antibodies, antisense oligonucleotides, peptides, enzymes or vectors.

For the time being, about 7000 rare diseases are known and each week new ones are discovered. In the European Union rare diseases have a prevalence of not more than 5 out of 10 000 people and in Japan rare diseases have a prevalence of not more than 4 out of 10 000 people. In the USA , however, the limit is 200 000 infected people per indication and year. In Australia, the limit is 1.2 out of 10,000 people per indication.

Orphan Drug status in Europe is granted after application and examination by the EMEA (European Medicines Agency), in the US by the FDA (Food and Drug Administration), in Japan by the MHLW (Ministry of Health, Labour and Welfare), and in Australia by the TGA (Therapeutic Goods Administration).

If Orphan Drug status is granted that means also exclusive marketing rights for five years (Australia), for seven years (USA) or respectively ten years (EU and Japan).

Orphan Drug Status can be freely commercialised and can be sold to third parties together with the corresponding patent application.