Positive Opinion from EMA's COMP for Calliditas' Setanaxib Application in Alport Syndrome

Friday, October 06, 2023

Calliditas Therapeutics AB has received a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) for its application seeking orphan drug designation in the European Union (EU) for setanaxib in the treatment of Alport syndrome.

The COMP's favorable opinion will now be forwarded to the European Commission, which holds the responsibility for making a final decision regarding orphan designation and adding the product to the Register of orphan medicinal products for human use in the community.

Orphan drugs, as defined by the European Medicines Agency, are those developed for diagnosing, preventing, or treating life-threatening or chronically debilitating conditions that are rare (affecting fewer than five in 10,000 individuals in the European Union) or cases where the medicine is unlikely to generate sufficient profit to cover research and development costs. Companies that receive orphan designation benefit from protocol assistance and market exclusivity once they obtain marketing authorization from the European Commission.

Calliditas' CEO, Renée Aguiar-Lucander, expressed her satisfaction with the COMP's positive opinion, saying, "We are pleased that the COMP has issued a positive opinion for orphan drug designation for setanaxib and are excited to commence another clinical program in the renal field, targeting an orphan indication where there are currently no approved products."

Drawing from substantial pre-clinical research, Calliditas plans to initiate a randomized, placebo-controlled Phase 2 clinical trial to evaluate setanaxib in Alport syndrome, involving approximately 20 patients, scheduled for the fourth quarter of 2023.

Alport syndrome is a genetic disorder resulting from mutations in genes responsible for type 4 collagen. Type 4 collagen alpha chains are predominantly located in the kidneys, eyes, and cochlea. Consequently, the condition is characterized by kidney disease, hearing loss, and eye abnormalities. Over time, patients typically develop proteinuria, hypertension, progressive kidney function decline (gradual decrease in GFR), and end-stage renal disease (ESRD).

Currently, Calliditas is exploring setanaxib in a Phase 2 proof-of-concept study for squamous cell carcinoma of the head and neck (SCCHN) and a Phase 2b study for primary biliary cholangitis (PBC). Additionally, an investigator-led study is underway to investigate setanaxib in idiopathic pulmonary fibrosis (IPF).

Harvard Medical School - Leadership in Medicine Southeast Asia47th IHF World Hospital CongressHealthcare Innovation & Transformation SummitHealthcare CNO SummitHealthcare CMO SummitThe Healthcare Patient Experience & Engagement Summit 2024