FDA Approves Targeted Release Budesonide for Treatment of Primary Immunoglobulin A Nephropathy

Tarpeyo is now the first fully FDA-approved treatment for primary immunoglobulin A nephropathy, based on measures of kidney function.

The FDA has approved targeted release budesonide (Tarpeyo; Calliditas Therapeutics AB) as a delayed release capsule indicated to reduce the loss of kidney function in adults with primary immunoglobulin A nephropathy (IgAN) at risk of progression, according to a press release from the company.

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In December 2021, Tarpeyo was approved under accelerated approval based on a surrogate marker of proteinuria and is now the first fully FDA-approved treatment for IgAN based on kidney function, according to the press release.

“The evidence of sustained reductions in proteinuria and a clinically significant reduction in the loss of [estimated glomerular filtration rate (eGFR)], which can help slow the progression towards dialysis or transplant care, highlights the potential of Tarpeyo as a disease-modifying agent in IgAN,” Richard Lafayette, MD, FACP, a rheumatologist at Stanford Health Care, said in the press release. “Tarpeyo provides physicians and patients an effective treatment option to help improve disease outcomes.”

The approval is based on data from the NeflgArd clinical trial, which was deigned to assess the efficacy and safety of targeted-release budesonide 16 mg once daily compared to the placebo plus an optimized renin-angiotensin system inhibitor therapy in adults with primary IgAN. The trial was a phase 3, randomized, multicenter study that included 350 individuals with primary IgAN. Treatment was randomized 1:1 with either Tarpeyo or the placebo for 9 months, plus an additional 15-month observational period without the study drug, according to the press release.

About The NefiGard Trial

Trial Name: Efficacy and Safety of Nefecon in Patients With Primary IgA (Immunoglobulin A) Nephropathy

ClinicalTrials.gov ID: NCT03643965

Sponsor: Calliditas Therapeutics AB

Completion Date: July 2023

The primary endpoint included time-weighted average of eGFR over 2 years, which was shown to have a statistically significant benefit with Tarpeyo compared to the placebo. The effect was seen by the third month and did not appear to increase over the 2 years, according to the press release. At the conclusion of the 2 years, there was an approximate 5.9 mL/min/1.73 m² difference in the mean change from baseline in eGFR between Tarpeyo and the placebo.

Further, the effect on kidney function during the 9-month treatment period continued after completion of treatment and the end of the study. Investigator said the long-term rate of decline has not been established.

“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,” Bonnie Schneider, director and cofounder of the IgAN Foundation, said in the press release. “The foundation is elated and personally this is so rewarding and validating after a near 20-year journey since founding this volunteer-run organization to raise awareness and promote research for IgAN.”

In the study, the most common adverse reactions of Tarpeyo were peripheral edema (17%), hypertension (12%), muscle spasms (12%), acne (11%), headache (10%), upper respiratory tract infection (8%), face edema (8%), weight increase (7%), dyspepsia (7%), dermatitis (6%), arthralgia (6%), and white blood cell count increase (6%), according to the press release.

Reference

Calliditas Therapeutics announces full FDA approval of Tarpeyo, the only FDA-approved treatment for IgA nephropathy to significantly reduce the loss of kidney function. News release. Calliditas. December 20, 2023. Accessed December 21, 2023. https://www.calliditas.se/en/calliditas-therapeutics-announces-full-fda-approval-of-tarpeyo-the-only-fda-approved-treatment-for-iga-nephropathy-to-significantly-reduce-the-loss-of-kidney-function/