European Medicines Agency Committee for Orphan Medicinal Products provides positive opinion on Calliditas’ application for setanaxib in Alport syndrome

Calliditas Therapeutics AB (Nasdaq: CALT, Nasdaq Stockholm: CALTX) (“Calliditas”) today announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion on the company’s application for orphan drug designation in the European Union (EU) for setanaxib in Alport syndrome. The COMP opinion will now go to the European Commission, which is responsible for adopting the decision in relation to the application for orphan designation and adding it to the Community register of orphan medicinal products for human use.  

The European Medicines Agency defines orphan drugs as medicinal products for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition that is rare (affecting not more than five in 10,000 people in the European Union) or where the medicine is unlikely to generate sufficient profit to justify research and development costs. Companies that obtain orphan designation benefit from protocol assistance and market exclusivity once marketing authorization has been granted by the European Commission.

“We are pleased that the COMP has issued a positive opinion for orphan drug designation for setanaxib and 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.

Based on significant pre-clinical work, Calliditas targets initiation of a randomized, placebo-controlled Phase 2 clinical study evaluating setanaxib in Alport syndrome with around 20 patients in Q4 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. The condition is, thus, 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).

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).