Orphan Drug Designation granted by FDA

26 February - 2015

Toleranzias therapy for the treatment of the rare disease Myasthenia Gravis has been granted Orphan Drug Designation by the American Food and Drug Administration (FDA).

  • The approval from the FDA is a great acknowledgement of Toleranzia and of its technology platform, says Toleranzias Executive Chairman of the Board Elisabet Litsmark Nordenstam.

According to the FDA: “Orphan designation qualifies the sponsor of the drug for various development incentives of the ODA, including tax credits for qualified clinical testing.” and following market approval an Orphan Drug will get a 7 year exclusivity on the market.

The worldwide market for MG is valued at appr. 500 MSEK today. And that is for therapies that only treat the symptoms, not the cause of the disease. The Tolerogen acts on the root cause of the disease and not only on the symptoms. If a truly disease modifying therapy enters the market it has a large potential to quickly become the first line therapy and be worth at least 5 billion SEK.