STOCKHOLM, Sweden — Calliditas Therapeutics (Formerly known as Pharmalink), today announced that the U.S. Food and Drug Administration (FDA) has approved TARPEYO (budesonide) delayed release capsules to reduce the loss of kidney function in adults with primary immunoglobulin A nephropathy (IgAN) at risk for disease progression. TARPEYO was first approved in December 2021 under accelerated approval, based on the surrogate marker of proteinuria. Marking a significant milestone, TARPEYO is now the first fully FDA-approved treatment for IgAN based on a measure of kidney function.
- TARPEYO (investigational name NEFECON) is the only FDA-approved treatment for IgAN to significantly reduce the loss of kidney function.
- TARPEYO is now approved with a confirmed and statistically significant benefit over placebo (p<0.0001) 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.
- At 2 years, there was a 6.11 mL/min/1.73 m2 decline in eGFR in the TARPEYO group compared with a 12.0 mL/min/1.73 m2 decline in the placebo group (p<0.0001), representing 50% less deterioration of kidney function in TARPEYO-treated patients compared to placebo-treated patients over the 2-year period.
- TARPEYO 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.1-3
- Significant proteinuria reduction achieved with TARPEYO plus RASi at 9 months was durable and maintained throughout the 15-month off-drug period.
- The FDA approval is for adults with primary IgAN who are at risk of disease progression, irrespective of proteinuria levels.
“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,” said Richard Lafayette, MD, FACP, Stanford Healthcare. “TARPEYO provides physicians and patients an effective treatment option to help improve disease outcomes.”
The approval is based on data from the Company’s 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.
“We are thrilled that adult IgAN patients at risk for progression in the United States can now have access to this pioneering treatment option that could help preserve their kidney function and, hence, impact the progression of their disease,” said Renee Aguiar-Lucander, CEO of Calliditas. “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.”
TARPEYO was generally well-tolerated in the Phase 3 NefIgArd clinical trial. The most common adverse reactions (≥5%) 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. Please see Important Safety Information below.
“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. “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.”
TARPEYO is available exclusively through Calliditas specialty pharmacy, Biologics by McKesson. To get started with TARPEYO, prescribers must fill out a TARPEYO Touchpoints® Enrollment Form, which serves as a prescription. This Enrollment Form will connect patients with all the benefits provided by TARPEYO Touchpoints®, including financial aid programs that can eliminate or reduce out-of-pocket costs, assistance from our team of care navigators, pharmacists, and nurse educators; and the convenience of at-home, next-day delivery. At Calliditas, we believe that the cost of treatment should never be a barrier to care. With TARPEYO Touchpoints®, 97% of patients taking TARPEYO have paid less than $10 per prescription, and 88% have paid nothing at all.
About TARPEYO
TARPEYO is an oral 4mg delayed release formulation of budesonide, designed to remain intact until it reaches the ileum. Each capsule contains coated beads of budesonide that target mucosal B-cells present in the ileum, including the Peyer’s patches, which are responsible for the production of galactose-deficient IgA1 antibodies (Gd-Ag1) causing IgA nephropathy.
CONTACT
Åsa Hillsten
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