Approvals

Calliditas Therapeutics has announced that the US Food and Drug Administration (FDA) has approved Tarpeyo (budesonide) delayed release capsules to reduce the loss of kidney function in adults with primary immunoglobin A nephropathy (IgAN) at risk of disease progression.

The drug was previously approved in December 2021 under the accelerated approval pathway, however this approval makes Tarpeyo the first fully FDA approved treatment for IgAN based on a measure of kidney function.

The approval follows data from the company’s phase 3 NeflgArd clinical trial, which was randomised, double-blind and multicentre, with the aim of assessing the safety and efficacy of the drug at 16mg doses daily, compared to placebo.

Richard Lafayette MD FACP, from Stanford Healthcare, commented: “The evidence of sustained reductions in proteinuria and a clinically significant reduction in the loss of eGFR, which can help slow the progression towards dialysis or transplant care, highlights the potential of Tarpeyo as a disease-modifying agent in IgAN. Tarpeyo provides physicians and patients an effective treatment option to help improve disease outcomes.”

Renee Aguiar-Lucander, CEO of Calliditas, added: “We are thrilled that adult IgAN patients at risk for progression in the US can now have access to this pioneering treatment option that could help preserve their kidney function and, hence, impact the progression of their disease. This medicine was specifically developed to target an underlying cause of IgAN, and I would like to express my gratitude to the Calliditas team, study investigators and most importantly, the patients and caregivers who made this significant milestone possible. I am incredibly proud of the team’s unwavering commitment to the goal of preventing end-stage renal disease in patients with this challenging rare disease.”

Bonnie Schneider, director and co-founder of the IgAN Foundation, stated: “This first-ever IgAN treatment to get a full approval based on kidney function represents a beacon of hope for the entire IgA nephropathy community and signifies a critical step forward in the battle against IgAN. The foundation is elated and personally this is so rewarding and validating after a near 20-year journey since founding this volunteer-run organisation to raise awareness and promote research for IgAN.”