FDA orphan drug designation for Alport syndrome with setanaxib


The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Calliditas Therapeutics for the treatment of Alport syndrome with setanaxib.

Based on supportive pre-clinical work, Calliditas plans to initiate a randomised, placebo-controlled Phase II clinical study in Alport syndrome with around 20 patients in the fourth quarter of 2023.

Alport syndrome is a genetic disorder arising from the mutations in the genes that code for type 4 collagen. The type 4 collagen alpha chains are primarily located in the kidneys, eyes, and cochlea, and thus the condition is characterised by kidney disease, loss of hearing, and eye abnormalities. Eventually, patients present with proteinuria, hypertension, progressive loss of kidney function (gradual decline in GFR), and end-stage renal disease (ESRD).

It is estimated that approximately 30,000 to 60,000 people in the United States (US) have this disorder, and it is a significant cause of chronic kidney disease (CKD), leading to ESRD in adolescents and young adults and accounting for 1.5% to 3.0% of children on renal replacement therapies in Europe and the US.1

Through its subsidiary Calliditas Therapeutics Suisse SA, which will sponsor the planned Phase II clinical study of setanaxib in Alport syndrome, Calliditas is currently investigating setanaxib in a Phase II proof-of-concept study in squamous cell carcinoma of the head and neck (SCCHN), as well as in a Phase IIb study in primary biliary cholangitis (PBC). Setanaxib is also being evaluated in an investigator-led study in idiopathic pulmonary fibrosis (IPF).

 “We are excited to start another clinical programme in the renal space targeting an orphan indication where today there are no approved products,” said CEO Renée Aguiar-Lucander.


  1. https://www.ncbi.nlm.nih.gov/books/NBK470419/

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