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IMPORTANT SAFETY INFORMATION & INDICATION

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

TARPEYO is contraindicated in patients with hypersensitivity to budesonide or any of the ingredients of TARPEYO. Serious hypersensitivity reactions, including anaphylaxis, have occurred with other budesonide formulations.

WARNINGS AND PRECAUTIONS

Hypercorticism and adrenal axis suppression:

When corticosteroids are used chronically, systemic effects such as hypercorticism and adrenal suppression may occur. Corticosteroids can reduce the response of the hypothalamus-pituitary-adrenal (HPA) axis to stress. In situations where patients are subject to surgery or other stress situations, supplementation with a systemic corticosteroid is recommended. When discontinuing therapy or switching between corticosteroids, monitor for signs of adrenal axis suppression.

Patients with moderate to severe hepatic impairment (Child-Pugh Class B and C, respectively) could be at an increased risk of hypercorticism and adrenal axis suppression due to an increased systemic exposure to oral budesonide. Avoid use in patients with severe hepatic impairment (Child-Pugh Class C). Monitor for increased signs and/or symptoms of hypercorticism in patients with moderate hepatic impairment (Child-Pugh Class B).

Immunosuppression and increased risk of infection:

Corticosteroids, including TARPEYO, suppress the immune system and increase the risk of infection with any pathogen, including viral, bacterial, fungal, protozoan, or helminthic pathogens. Corticosteroids can: reduce resistance to new infections, exacerbate existing infections, increase the risk of disseminated infections, increase the risk of reactivation or exacerbation of latent infections, and mask some signs of infection. Corticosteroid-associated infections can sometimes be serious. Monitor for infection and consider TARPEYO withdrawal as needed.

Avoid corticosteroid therapy, including TARPEYO, in patients with active or quiescent tuberculosis or hepatitis B infection; untreated fungal, bacterial, systemic viral, or parasitic infections; ocular herpes simplex; or Kaposi’s sarcoma. Avoid exposure to active, easily transmitted infections (e.g., chickenpox, measles). Corticosteroid therapy may decrease the immune response to some vaccines.

Other corticosteroid effects:

TARPEYO is a systemically available corticosteroid and is expected to cause related adverse reactions. Monitor patients with hypertension, prediabetes, diabetes mellitus, osteoporosis, peptic ulcer, glaucoma or cataracts, or with a family history of diabetes or glaucoma, or with any other condition where corticosteroids may have unwanted effects.

ADVERSE REACTIONS

In clinical studies, the most common adverse reactions with TARPEYO (occurring in ≥5% of TARPEYO-treated patients, and ≥2% higher than placebo) were peripheral edema (17%), hypertension (12%), muscle spasms (12%), acne (11%), headache (10%), upper respiratory tract infection (8%), face edema (8%), weight increased (7%), dyspepsia (7%), dermatitis (6%), arthralgia (6%), and white blood cell count increased (6%).

DRUG INTERACTIONS

Budesonide is a substrate for CYP3A4. Avoid use with potent CYP3A4 inhibitors, such as ketoconazole, itraconazole, ritonavir, indinavir, saquinavir, erythromycin, and cyclosporine. Avoid ingestion of grapefruit juice with TARPEYO. Intake of grapefruit juice, which inhibits CYP3A4 activity, can increase the systemic exposure to budesonide.

USE IN SPECIFIC POPULATIONS

Pregnancy:

The available data from published case series, epidemiological studies, and reviews with oral budesonide use in pregnant women have not identified a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. There are risks to the mother and fetus associated with IgAN. Infants exposed to in utero corticosteroids, including budesonide, are at risk for hypoadrenalism.

INDICATION

TARPEYO is indicated to reduce the loss of kidney function in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk for disease progression.

eGFR

TARPEYO stabilized eGFR while on treatment, and reduced the loss of kidney function over 2 years1

Primary endpoint

Time-weighted average of eGFR change over 2 years1

  • Statistically significant benefit of 5.05 mL/min/1.73 m2 at 2 years2*
  • LS mean change in eGFR of +1.5 mL/min/1.73 m2 at 9 months1

LS mean change in eGFR from baseline over 2 years (95% CI; p<0.0001)1*

Graph showing eGFR change over 2 years. Patients on TARPEYO + RASi (blue) had improved eGFR compared to those on RASi alone (grey). A >50% difference in kidney function deterioration at 2 years is noted, favoring TARPEYO + RASi. Graph showing eGFR change over 2 years. Patients on TARPEYO + RASi (blue) had improved eGFR compared to those on RASi alone (grey). A >50% difference in kidney function deterioration at 2 years is noted, favoring TARPEYO + RASi.

The favorable effect of TARPEYO on eGFR was seen as early as month 3 and remained consistent over 2 years.1

The effect of TARPEYO on the long-term rate of decline in kidney function has not been established.1

*The primary endpoint for the interim analysis was UPCR at 9 months compared to baseline (N=199).

Estimated from a mixed-model, repeated-measures analysis using untransformed data up to 24 months, regardless of use of rescue medications.1

Calculated as relative reduction (9.4% TARPEYO + RASi vs 20.3% RASi alone).1,3

§Not all patients in the full analysis set (FAS) contributed data at each time point.1

CI=confidence interval; eGFR=estimated glomerular filtration rate; LS=least squares; RASi=renin-angiotensin system inhibitor; UPCR=urine protein-to-creatinine ratio.

eGFR results were consistent across UPCR subgroups5

A post hoc subgroup analysis of eGFR over time in patients with UPCR ≥ or <0.8 g/g5

  • Patients with baseline UPCR <0.8 g/g met trial eligibility criteria with proteinuria ≥1 g/d5

Mean (±SE) absolute change in eGFR in patients with baseline UPCR <0.8 g/g5*

Figure 1: Mean (±SE) change in eGFR in patients with baseline UPCR <0.8 g/g Line graph showing the mean (±SE) change in eGFR over 24 months for patients with baseline UPCR <0.8 g/g. The x-axis represents months (0-24), and the y-axis represents eGFR change (mL/min/1.73 m²). TARPEYO (purple line) shows an initial increase in eGFR, peaking at 4.5 at 3 months, then gradually decreases. The placebo (grey line) shows a consistent decline over time. Figure 1: Mean (±SE) change in eGFR in patients with baseline UPCR <0.8 g/g Line graph showing the mean (±SE) change in eGFR over 24 months for patients with baseline UPCR <0.8 g/g. The x-axis represents months (0-24), and the y-axis represents eGFR change (mL/min/1.73 m²). TARPEYO (purple line) shows an initial increase in eGFR, peaking at 4.5 at 3 months, then gradually decreases. The placebo (grey line) shows a consistent decline over time.

Mean (±SE) absolute change in eGFR in patients with baseline UPCR ≥0.8 g/g5*

Figure 2: Mean (±SE) change in eGFR in patients with baseline UPCR >0.8 g/g (p<0.0001) Second line graph showing the mean (±SE) change in eGFR over 24 months for patients with baseline UPCR >0.8 g/g. The x-axis represents months (0-24), and the y-axis represents eGFR change (mL/min/1.73 m²). TARPEYO + RASi (purple line) initially increases eGFR, peaking at 2.2 at 3 months, then declines. RASi alone (grey line) shows a consistent decrease over time. Figure 2: Mean (±SE) change in eGFR in patients with baseline UPCR >0.8 g/g (p<0.0001) Second line graph showing the mean (±SE) change in eGFR over 24 months for patients with baseline UPCR >0.8 g/g. The x-axis represents months (0-24), and the y-axis represents eGFR change (mL/min/1.73 m²). TARPEYO + RASi (purple line) initially increases eGFR, peaking at 2.2 at 3 months, then declines. RASi alone (grey line) shows a consistent decrease over time.

Small sample sizes and lack of multiplicity adjustments are limitations of these analyses. Results should be interpreted with caution. The clinical significance of these results is unknown.

*Estimated absolute change from baseline=baseline geometric mean for total x (geometric LS mean of post-baseline value/baseline value for each treatment arm –1).5

Estimated absolute change from baseline=baseline geometric mean for total x (geometric LS mean of ratio of AUC over 2 years compared with baseline for each treatment arm –1).5

Not all patients contributed data at each time point.

AUC=area under the curve; SE=standard error.

UPCR

Durable UPCR reduction through 2 years1

Significant reduction with a 9-month fixed course of TARPEYO treatment1

LS mean percent change in UPCR (g/g) from baseline1*

This line graph shows the least squares (LS) mean percent change in UPCR (g/g) from baseline over 24 months. The x-axis represents months (0-24), and the y-axis represents the percent change in UPCR. TARPEYO + RASi (purple line) shows a significant decrease in UPCR, reaching -52% at 12 months and -34% at 24 months. RASi alone (grey line) shows a smaller decrease, around -8% at 12 months and -7% at 24 months. A purple box on the right highlights a 52% reduction in UPCR achieved at 12 months. This line graph shows the least squares (LS) mean percent change in UPCR (g/g) from baseline over 24 months. The x-axis represents months (0-24), and the y-axis represents the percent change in UPCR. TARPEYO + RASi (purple line) shows a significant decrease in UPCR, reaching -52% at 12 months and -34% at 24 months. RASi alone (grey line) shows a smaller decrease, around -8% at 12 months and -7% at 24 months. A purple box on the right highlights a 52% reduction in UPCR achieved at 12 months.
  • 41% average reduction in UPCR during observational follow-up (month 12 to month 24) compared to baseline based on longitudinal mixed-effects model for repeated-measures model (95% CI: 32% to 49%)

*Estimated mean percentage change from baseline in UPCR with 95% CI estimated from a mixed-model, repeated-measures analysis of log-transformed post-baseline to baseline ratios, regardless of use of prohibited medications.1

The interim analysis at 9 months with the first 199 patients showed a 31% reduction in UPCR in patients treated with TARPEYO vs RASi alone (95% CI: 16% to 42% reduction; p=0.0001).1

Not all patients in the FAS contributed data at each time point.1

§Data based on FAS of 364 patients.1

Study Design

The NefIgArd trial was designed to evaluate kidney function at 2 years after 9 months on treatment, as measured by eGFR1

Primary endpoint at full study completion: eGFR at 2 years (N=364)1*

This diagram outlines the study design. The study includes a screening phase, 9 months on TARPEYO treatment, and a 15-month observational follow-up off TARPEYO treatment. During screening, patients receive optimized RASi therapy for at least 3 months. Patients are then randomized into two groups: TARPEYO + RASi (purple arrow) or RASi + placebo (grey arrow) for 9 months. Following treatment, there is a 15-month follow-up where patients receive no TARPEYO treatment but continue optimized RASi therapy. This diagram outlines the study design. The study includes a screening phase, 9 months on TARPEYO treatment, and a 15-month observational follow-up off TARPEYO treatment. During screening, patients receive optimized RASi therapy for at least 3 months. Patients are then randomized into two groups: TARPEYO + RASi (purple arrow) or RASi + placebo (grey arrow) for 9 months. Following treatment, there is a 15-month follow-up where patients receive no TARPEYO treatment but continue optimized RASi therapy.

Key inclusion criteria1,2

Adult patients with2:

  • Biopsy-confirmed IgAN diagnosis
  • Persistent proteinuria ≥1 g/day or UPCR ≥0.8 g/g in 2 consecutive measurements
  • eGFR 35 to 90 mL/min/1.73 m2
  • Receiving RASi therapy (ACEis and/or ARBs) at the maximum allowed/tolerated dose 3 months prior to randomization

Blood pressure and diabetes were well controlled and no prophylaxis with antibiotics was required.1,2

Baseline characteristics2

Table showing baseline patient characteristics for TARPEYO + RASi and RASi alone groups. It includes median age, gender distribution, and racial breakdown (White, Asian, Hispanic or Latino, and Other). Table showing baseline patient characteristics for TARPEYO + RASi and RASi alone groups. It includes median age, gender distribution, and racial breakdown (White, Asian, Hispanic or Latino, and Other). Table comparing disease characteristics between patients treated with TARPEYO + RASi and RASi alone. The table includes baseline proteinuria, UPCR, eGFR, blood pressure, diabetes mellitus incidence, microhematuria incidence, and time from IgAN diagnosis. Table comparing disease characteristics between patients treated with TARPEYO + RASi and RASi alone. The table includes baseline proteinuria, UPCR, eGFR, blood pressure, diabetes mellitus incidence, microhematuria incidence, and time from IgAN diagnosis.

Select baseline characteristics1,2

  • More than half of patients were younger than 45 years of age at study entry
  • Diverse patient population across ethnic backgrounds
  • Blood pressure was well controlled at study entry (recommended target of <125/75 mmHg)
  • Majority of patients had not been on prior steroid therapy

Select secondary efficacy endpoints2

  • Composite endpoint of time from randomization to confirmed 30% reduction in eGFR (confirmed by 2 values over ≥4 weeks)
  • Ratios of UPCR and UACR compared with baseline
  • Proportion of patients without microhematuria during observational follow-up

*The primary endpoint for the interim analysis was UPCR at 9 months compared to baseline (N=199).1
Of the 364 randomized patients evaluated for efficacy, 66% were male, 76% were Caucasian, 23% were Asian, and 20% were from North America. The median age was 43 years (range 20 to 73 years).1
Ratios were averaged over timepoints between 12 and 24 months, inclusive, after the first dose of study drug.2

ACEi=angiotensin-converting enzyme inhibitor; ARB=angiotensin II receptor blocker; UACR=urine albumin-creatinine ratio.
TARPEYO was studied under the name NEFECON.

REFERENCES: 1. TARPEYO. Prescribing Information. Calliditas Therapeutics AB; June 2024. 2. Lafayette R, Kristensen J, Stone A, et al. Efficacy and safety of a targeted-release formulation of budesonide in patients with primary IgA nephropathy (NefIgArd): 2-year results from a randomized phase 3 trial. Lancet. 2023;402(10405):825-936. http://doi.org/10.1016/S0140-6736(23)01554-4 3. Data on file. Part B. Calliditas Therapeutics AB. 4. Barratt J, Lafayette RA, Rovin BH, Fellström B. Budesonide delayed-release capsules to reduce proteinuria in adults with primary immunoglobulin A nephropathy. Expert Rev Clin Immunol. 2023;19(7):699-710. doi:10.1080/1744666X.2023.2206119 5. Barratt J, et al. Nefecon treatment provides kidney benefits for patients with IgAN that extend to those with low levels of UPCR: A subanalysis of the Phase 3 NefIgArd trial. Presented at the International Society of Nephrology World Congress of Nephrology; April 13-16, 2024; Buenos Aires, Argentina. 6. National Kidney Foundation. Stages of Chronic Kidney Disease (CKD). https://www.kidney.org/kidney-topics/stages-chronic-kidney-disease-ckd. Accessed September 20, 2024.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

TARPEYO is contraindicated in patients with hypersensitivity to budesonide or any of the ingredients of TARPEYO. Serious hypersensitivity reactions, including anaphylaxis, have occurred with other budesonide formulations.

WARNINGS AND PRECAUTIONS

Hypercorticism and adrenal axis suppression:

When corticosteroids are used chronically, systemic effects such as hypercorticism and adrenal suppression may occur. Corticosteroids can reduce the response of the hypothalamus-pituitary-adrenal (HPA) axis to stress. In situations where patients are subject to surgery or other stress situations, supplementation with a systemic corticosteroid is recommended. When discontinuing therapy or switching between corticosteroids, monitor for signs of adrenal axis suppression.

Patients with moderate to severe hepatic impairment (Child-Pugh Class B and C, respectively) could be at an increased risk of hypercorticism and adrenal axis suppression due to an increased systemic exposure to oral budesonide. Avoid use in patients with severe hepatic impairment (Child-Pugh Class C). Monitor for increased signs and/or symptoms of hypercorticism in patients with moderate hepatic impairment (Child-Pugh Class B).

Immunosuppression and increased risk of infection:

Corticosteroids, including TARPEYO, suppress the immune system and increase the risk of infection with any pathogen, including viral, bacterial, fungal, protozoan, or helminthic pathogens. Corticosteroids can: reduce resistance to new infections, exacerbate existing infections, increase the risk of disseminated infections, increase the risk of reactivation or exacerbation of latent infections, and mask some signs of infection. Corticosteroid-associated infections can sometimes be serious. Monitor for infection and consider TARPEYO withdrawal as needed.

Avoid corticosteroid therapy, including TARPEYO, in patients with active or quiescent tuberculosis or hepatitis B infection; untreated fungal, bacterial, systemic viral, or parasitic infections; ocular herpes simplex; or Kaposi’s sarcoma. Avoid exposure to active, easily transmitted infections (e.g., chickenpox, measles). Corticosteroid therapy may decrease the immune response to some vaccines.

Other corticosteroid effects:

TARPEYO is a systemically available corticosteroid and is expected to cause related adverse reactions. Monitor patients with hypertension, prediabetes, diabetes mellitus, osteoporosis, peptic ulcer, glaucoma or cataracts, or with a family history of diabetes or glaucoma, or with any other condition where corticosteroids may have unwanted effects.

ADVERSE REACTIONS

In clinical studies, the most common adverse reactions with TARPEYO (occurring in ≥5% of TARPEYO-treated patients, and ≥2% higher than placebo) were peripheral edema (17%), hypertension (12%), muscle spasms (12%), acne (11%), headache (10%), upper respiratory tract infection (8%), face edema (8%), weight increased (7%), dyspepsia (7%), dermatitis (6%), arthralgia (6%), and white blood cell count increased (6%).

DRUG INTERACTIONS

Budesonide is a substrate for CYP3A4. Avoid use with potent CYP3A4 inhibitors, such as ketoconazole, itraconazole, ritonavir, indinavir, saquinavir, erythromycin, and cyclosporine. Avoid ingestion of grapefruit juice with TARPEYO. Intake of grapefruit juice, which inhibits CYP3A4 activity, can increase the systemic exposure to budesonide.

USE IN SPECIFIC POPULATIONS

Pregnancy:

The available data from published case series, epidemiological studies, and reviews with oral budesonide use in pregnant women have not identified a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. There are risks to the mother and fetus associated with IgAN. Infants exposed to in utero corticosteroids, including budesonide, are at risk for hypoadrenalism.

INDICATION

TARPEYO is indicated to reduce the loss of kidney function in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk for disease progression.