EMA endorses Calliditas’ Alport syndrome drug for orphan designation

The European Medicines Agency (EMA) has provided a positive opinion for Calliditas Therapeutics’ setanaxib for the treatment of Alport syndrome, as per a 5 October company statement. This opinion has been passed on to the European Commission who will make a final decision on the drug’s orphan designation.

The EMA currently 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.”

This designation gives the drug manufacturer market exclusivity once the European Commission has granted a market authorisation. If the therapy receives orphan drug status, this will follow a decision made by the US Food and Drug Administration (FDA) in September decision to grant the therapy an orphan drug designation.

Alport syndrome is a rare genetic condition caused by mutations in the genes that code for type 4 collagen. This can lead to symptoms such as kidney disease, loss of hearing and eye abnormalities. It is estimated that, in Europe, one in every 100,000 individuals is affected by the disorder.

Thus far, the EMA has not approved any drug products for the treatment of Alport syndrome. According to GlobalData, setanaxib is one of eight therapies currently in clinical development for Alport syndrome.

GlobalData is the parent company of Pharmaceutical Technology.

Calliditas predicts that it will begin evaluating setanaxib in a Phase II clinical study for Alport syndrome in Q4 2023. The Stockholm, Sweden- headquartered company is also currently developing setanaxib in a Phase II squamous cell carcinoma trial (NCT05323656) as well as a Phase IIb primary biliary cholangitis study (NCT05014672).

In a press release, Calliditas’ CEO, Renée Aguiar-Lucander, said, “[We] are excited to start another clinical program in the renal space targeting an orphan indication where today there are no approved products”. At present, the company is also developing its IgA nephropathy drug, Tarpeyo (nefecon) within its kidney disorder program.