RARE Daily

FDA Grants Full Approval of Calliditas’ IgAN Therapy

December 21, 2023

Rare Daily Staff

The U.S. Food and Drug Administration granted full approval to Calliditas Therapeutics TARPEYO delayed release capsules to reduce the loss of kidney function in adults with primary immunoglobulin A nephropathy at risk for disease progression.

Tarpeyo was first approved in December 2021 under accelerated approval, based on the surrogate marker of proteinuria. Tarpeyo is now the first fully FDA-approved treatment for IgAN based on a measure of kidney function.

Primary immunoglobulin A nephropathy (IgAN or Berger’s Disease) is a rare, progressive, chronic autoimmune disease that attacks the kidneys and occurs when galactose-deficient IgA1 is recognized by autoantibodies, creating IgA1 immune complexes that become deposited in the glomerular mesangium of the kidney. This deposition in the kidney can lead to progressive kidney damage and potentially a clinical course resulting in end- stage renal disease. IgAN most often develops between the late teens and late 30s.

Tarpeyo (budesonide) is a B-cell immunomodulator designed to target a source of the disease and reduce the production of pathogenic galactose-deficient IgA1 antibodies, which cause IgAN.

Tarpeyo is now approved with a confirmed and statistically significant benefit over placebo in estimated glomerular filtration rate (eGFR) over the two-year period that consisted of 9 months of treatment with Tarpeyo plus optimized renin-angiotensin system inhibitor (RASi) or placebo and optimized RASi and 15 months of follow-up off study drug.

“This medicine was specifically developed to target an underlying cause of IgAN,” said Renee Aguiar-Lucander, CEO of Calliditas.

The approval is based on data from the Calliditas’ phase 3 NefIgArd clinical trial, a randomized, double-blind, multicenter, study that assessed the efficacy and safety of Tarpeyo dosed at 16 mg once daily versus placebo on a background of optimized RASi therapy in adult patients with primary IgAN. The primary efficacy endpoint was time-weighted average of eGFR over 2 years, which showed a statistically significant treatment benefit with Tarpeyo versus placebo and represented 50 percent less deterioration of kidney function in Tarpeyo treated patients compared to placebo-treated patients over the 2-year period. Significant proteinuria reduction achieved with Tarpeyo plus RASi at 9 months was durable and maintained throughout the 15-month off-drug period.

Tarpeyo was generally well-tolerated in the phase 3 NefIgArd clinical trial. The most common adverse reactions (≥5 percent) in this study were peripheral edema, hypertension, muscle spasms, acne, headache, URT infection, face edema, weight increased, dyspepsia, dermatitis, arthralgia, and white blood cell count increased.

“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,” said Bonnie Schneider, director and cofounder of the IgAN Foundation.

Photo: Renee Aguiar-Lucander, CEO of Calliditas

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