Calliditas Therapeutics AB (publ) (”Calliditas”) today announced that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to the company for the treatment of Primary biliary cholangitis (PBC). More information about the ODD has been posted on www.fda.gov.
PBC, previously known as primary biliary cirrhosis, is an autoimmune disease of the liver, where common symptoms are tiredness, itching and, in more advanced cases, jaundice. PBC is a rare disease, affecting approximately 4.3 people in 10,000 in US. It is much more common in women, with a ratio of at least 9:1 female to male.
The company plans to discuss the regulatory pathway for this indication in consultation with the FDA and investigate the most appropriate way forward for this patient population.
“We are very pleased to receive ODD in the US for the treatment of PBC. 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 disease with few medical alternatives today, but with a lot of exciting research taking place”, commented Renée Aguiar-Lucander, CEO of Calliditas Therapeutics.