Calliditas Therapeutics

Immunoglobin A nephropathy (IgAN) market to grow at 26% CAGR to $4.2 billion by 2033 due to novel therapies, awareness, and treatment rates. TARPEYO® (budesonide) is the only FDA-approved treatment for IgAN, significantly reducing kidney function loss. Calliditas Therapeutics utilized the FDA’s Orphan Drug Designation pathway for accelerated approval. TARPEYO’s dominance may face challenges from high cost and new competitors.

Hosts discuss PARASOL Initiative's efforts to establish surrogate endpoints for FSGS trials, focusing on proteinuria as a robust predictor of kidney failure, potentially enabling smaller, faster trials.

The PARASOL Initiative aims to revolutionize FSGS drug development by validating proteinuria as a therapeutic endpoint. Experts discuss how proteinuria reduction can accelerate approvals, emphasizing the need for addressing podocyte dysfunction. The initiative also focuses on specimen banking for biomarker discovery and collaborative research models for rare kidney diseases.

FDA approves Phase II trial for Ruxoprubart, a selective alternative pathway inhibitor, in IgAN patients, with potential to treat multiple disorders. Ruxoprubart's unique mechanism avoids blocking the classical pathway, offering advantages over existing treatments.

Iptacopan (Fabhalta) showed a clinically meaningful reduction in proteinuria and confirmed clinical benefit of alternative pathway inhibition in IgA nephropathy, with a 38.3% lower UPCR than placebo by month 9. The study also found significant decreases in complement pathway biomarkers, supporting selective alternative pathway inhibition.

Everest Medicines announces Taiwan TFDA approval of NEFECON for primary IgA nephropathy, expanding access to the world's first FDA-approved IgAN treatment. NEFECON demonstrated significant benefits in reducing kidney function decline and delaying disease progression.

The European Commission approves Ipsen's IQIRVO (elafibranor) for primary biliary cholangitis, the first new therapy in the EU in a decade. Other companies, including CymaBay, Zydus, and GlaxoSmithKline, are preparing to enter the market. The approval is based on the Phase III ELATIVE trial, showing a 47% clinical benefit of IQIRVO combined with UDCA. Additionally, Gilead Sciences' LIVDELZI (seladelpar) received FDA accelerated approval for PBC.

Renee Aguiar-Lucander, CEO of Calliditas Therapeutics, transitioned from finance to biopharma after 12 years investing in life sciences. Her experience was crucial for Calliditas' success, including being the first to use a novel renal primary surrogate endpoint in a Phase III trial, leading to the first-ever accelerated FDA approval for a nephrology drug. She champions women in leadership and has expanded Calliditas globally, doubling staff annually since 2018.