Positive opinion on Calliditas' Alport Syndrome drug from EMA

The company​ made an application for orphan drug designation in the European Union (EU).

The positive opinion came via the Committee for Orphan Medicinal Products (COMP) and 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 orphan medicinal products for human use.

The EC 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.  

AS is a rare genetic disorder of part of the kidney’s filtration system called the glomerular basement membrane. It’s the second most common cause of inherited chronic kidney disease after polycystic kidney disease. The condition can cause hearing loss and might cause minor eye problems in some people. Eventually, patients present with proteinuria, hypertension, progressive loss of kidney function, a gradual decline in the glomerular filtration rate (GFR), and end-stage renal disease (ESRD).

The name comes from South African doctor, Professor Arthur Cecil Alport, the condition is caused by genetic defects in the formation of a particular type of collagen fibre, collagen 4 which is found in the kidneys, ears, and eyes.

Significant pre-clinical work

The EMA 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 Calliditas 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 the last quarter of this year (2023).

Calliditas​ is currently investigating setanaxib in a phase 2 proof-of-concept study in squamous cell carcinoma of the head and neck, as well as in a phase 2b study in primary biliary cholangitis. Setanaxib is also being evaluated in an investigator-led study in idiopathic pulmonary fibrosis.