Calliditas Therapeutics bags TARPEYO FDA approval for IgAN

Calliditas Therapeutics AB (Nasdaq: CALT, Nasdaq Stockholm: CALTX) has announced a landmark FDA approval for TARPEYO (budesonide) delayed release capsules, aimed at reducing kidney function loss in adults with primary immunoglobulin A nephropathy (IgAN) at risk of disease progression. Initially granted accelerated approval in December 2021, TARPEYO now stands as the first fully FDA-approved treatment for IgAN, based on kidney function measures.

TARPEYO: A Novel Approach to IgAN

TARPEYO, originally known as NEFECON, is the only FDA-approved treatment for IgAN that significantly reduces kidney function loss. The approval comes with statistically significant evidence over placebo (p<0.0001) in estimated glomerular filtration rate (eGFR) over a two-year study period. This period included nine months of treatment with TARPEYO plus optimized renin-angiotensin system inhibitor (RASi) therapy and 15 months of follow-up.

Calliditas Therapeutics secures TARPEYO FDA approval for IgAN treatment

At the two-year mark, the TARPEYO group showed a 50% less deterioration of kidney function compared to placebo-treated patients, marking a significant advancement in the treatment of this rare disease.

TARPEYO’s Efficacy and Design

TARPEYO acts as a B-cell immunomodulator, targeting the source of IgAN and reducing the production of pathogenic galactose-deficient IgA1 antibodies. The reduction in proteinuria achieved with TARPEYO plus RASi at nine months was maintained throughout the 15-month off-drug period. This approval by the FDA caters to adults with primary IgAN, irrespective of proteinuria levels.

Phase 3 Clinical Trial Insights

The approval is based on data from the company’s Phase 3 NefIgArd clinical trial, a randomized, double-blind, multicenter study assessing the efficacy and safety of TARPEYO. The trial’s results underscore the drug’s potential as a disease-modifying agent in IgAN, according to Richard Lafayette, MD, FACP, of Stanford Healthcare.

TARPEYO’s Accessibility and Patient Support

TARPEYO is available exclusively through Calliditas’ specialty pharmacy, Biologics by McKesson. To access TARPEYO, prescribers must fill out a TARPEYO Touchpoints Enrollment Form, which includes benefits like financial aid programs, care navigator assistance, and at-home delivery. Remarkably, 97% of patients taking TARPEYO have paid less than $10 per prescription, with 88% paying nothing at all, thanks to TARPEYO Touchpoints.

A Beacon of Hope for the IgAN Community

This first-ever full FDA approval for IgAN treatment based on kidney function is celebrated by the IgAN Foundation. Bonnie Schneider, director and cofounder, sees it as a beacon of hope and a critical step forward in the battle against IgAN.

Conclusion: A New Era in IgAN Treatment

Calliditas Therapeutics’ TARPEYO, now fully FDA-approved, represents a breakthrough in the treatment of IgAN. This approval not only offers new hope to patients but also signifies a major step in the journey towards effective management and potential cure of this challenging rare disease.