Calliditas Therapeutics granted orphan drug designation by the FDA for the treatment of Alport syndrome with setanaxib

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The FDA grants orphan drug designation to Calliditas Therapeutics for the treatment of Alport syndrome with setanaxib, prompting plans for a phase 2 clinical study. Alport syndrome affects 30,000 to 60,000 people in the US and is a significant cause of chronic kidney disease. Positive for Calliditas stock.

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• FDA grants orphan drug designation to Calliditas Therapeutics for Alport syndrome treatment with setanaxib, prompting plans for phase 2 clinical study

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STOCKHOLM, Sept. 27, 2023 /PRNewswire/ -- Calliditas Therapeutics AB (Nasdaq: CALT) (Nasdaq Stockholm: CALTX) ("Calliditas") today announced that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to the company for the treatment of Alport syndrome with setanaxib.

Based on supportive pre-clinical work, Calliditas plans to initiate a randomized, placebo-controlled phase 2 clinical study in Alport syndrome with around 20 patients in the fourth quarter of 2023.

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

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 characterized 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 2 clinical study of setanaxib in Alport syndrome, Calliditas is currently investigating setanaxib in a Phase 2 proof-of-concept study in squamous cell carcinoma of the head and neck (SCCHN), as well as in a Phase 2b study in primary biliary cholangitis (PBC). Setanaxib is also being evaluated in an investigator-led study in idiopathic pulmonary fibrosis (IPF).

^[1] Watson S, Padala SA, Hashmi MF, et al. Alport Syndrome. [Updated 2023 Aug 14]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2023 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK470419/

For further information, please contact:

Åsa Hillsten, Head of IR & Sustainability, Calliditas
Tel.: +46 76 403 35 43, Email: asa.hillsten@calliditas.com

The following files are available for download:

https://mb.cision.com/Main/16574/3843536/2324182.pdf

CALSE Alport ODD PR (2023.09.27)

 

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SOURCE Calliditas Therapeutics