Calliditas Therapeutics announced the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to the company for the treatment of Autoimmune hepatitis (AIH).
AIH is a chronic, autoimmune disease of the liver that occurs when the body's immune system attacks liver cells causing the liver to be inflamed. The disease may occur in any ethnic group and at any age but is most often diagnosed in patients between age 40 and 50. It is estimated that the patient population in the US amounts to approximately 50,000.
The company plans to agree the regulatory pathway for this indication in consultation with the FDA later this year.
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"We are very pleased to receive ODD in the US for the treatment of AIH. This confirms the high unmet medical need and further encourages us to continue to explore orphan indications in which we could leverage our existing expertise. This is a devastating disease with few medical alternatives today, which we believe is the case also in other chronic liver indications," said Renée Aguiar-Lucander, CEO of Calliditas Therapeutics.
The company is currently running a global, pivotal Phase 3 with study for the treatment of the rare disease IgA nephropathy, and which has already obtained ODD by the FDA and the European Medicines Agency (EMA). Top line data for IgA nephropathy is expected in H2 2020.