Clinical experience of Acthar reducing proteinuria in nephrotic syndrome

In a prospective, open-label study of 15 patients with proteinuria due to nephrotic syndrome in 3 prespecified subgroups*2:

  • All patients had confirmed nephrotic-range proteinuria at the initial study visit and were treated with Acthar 80 units subcutaneously twice weekly for 6 months
    – Baseline proteinuria was calculated by the average of the first 3 weeks’ urine protein:creatinine ratio (UPCR) values
  • Most patients were previously treated with at least one other commonly used therapy for proteinuria due to nephrotic syndrome

Proteinuria was reduced in 2 of 5 patients with MN*2

Patient Age Diagnosis Proteinuria: UPCR (g/g) Outcome† Adverse events
Pre-ACTH Post-ACTH
Male, White 68 MN 6.70 10.50 No response Worsened glycemic control
Female, African American 50 MN 3.05 3.75 No response Worsened glycemic control
Male, Asian 68 MN 3.80 3.88 No response None
Male, White 58 MN 2.23 0.84 Partial remission Temporary increase in skin pigmentation
Male, White 71 MN 12.46 3.14 Partial remission None
    * Results from a prospective, open-label study designed to evaluate the efficacy and safety of Acthar in 3 prespecified subgroups: patients with membranous nephropathy (MN), patients with either focal segmental glomerulosclerosis (FSGS) or minimal change disease (MCD), and patients with immunoglobulin A (IgA) nephropathy. 15 patients with nephrotic syndrome were treated with Acthar 80 units subcutaneously twice weekly for 6 months: 5 with MN, 5 with FSGS or MCD, and 5 with IgA nephropathy.
    †Partial remission was defined as stable or improved renal function with ≥50% reduction in UPCR to 0.50–3.50 g/g.

Partial remission was achieved in 2 of 5 patients with MCD/FSGS*2

Patient Age Diagnosis Proteinuria: UPCR (g/g) Outcome† Adverse events
Pre-ACTH Post-ACTH
Female, Hispanic 29 MCD 4.76 11.35 No response None
Female, White 30 MCD 3.16 0.78 Partial remission None
Male, White 41 FSGS 1.94 0.43 Partial remission None
Male, White 35 FSGS 1.96 4.08 No response None
Male, White 66 FSGS 1.65 1.93 No response Temporary increase in skin pigmentation

≥50% reductions in proteinuria were seen in 2 of 5 patients with IgA nephropathy*2

Patient Age Diagnosis Proteinuria: UPCR (g/g) Outcome† Adverse events
Pre-ACTH Post-ACTH
Female, Hispanic 37 IgA nephropathy 1.95 1.22 No response None
Male, White 46 IgA nephropathy 1.59 0.57 Partial remission None
Female, Asian 55 IgA nephropathy 0.61 (1.54 at screening) 0.21 Complete remission None
Male, Asian 36 IgA nephropathy 4.12 3.51 No response Weight gain, Cushingoid facies,increase in blood pressure
Male, Asian 35 IgA nephropathy 1.51 0.85 No response None
    ‡Remission was defined as stable or improved renal function with ≥50% reduction in UPCR to <0.50 g/g (complete remission) or 0.50–3.50 g/g (partial remission).

Common side effects include fluid retention, behavioral and mood changes, change in glucose tolerance, increased appetite, increased blood pressure, and weight gain.1

These results are based on a prospective, open-label study of 15 patients and may not be fully representative of outcomes in the overall patient population. Most patients were on multiple therapies. The clinical outcomes may not be solely attributable to Acthar.

Clinical experience showed Acthar reduced proteinuria in the majority of patients

In a retrospective case series of 21 patients with nephrotic syndrome of varying etiologies*3:

  • 11 of the 21 patients achieved complete or partial remission with Acthar
    –Of the 11 patients who achieved remission, 9 had MN, 1 had FSGS, and 1 had IgA nephropathy
  • Duration of Acthar therapy ranged from 1 to 14 months; follow-up time ranged from 6 to 14 months
  • Most patients were previously treated with at least one other commonly used therapy for proteinuria due to nephrotic syndrome

Individual results in all 11 patients with nephrotic syndrome due to MN*3

Patient Age Diagnosis Proteinuria (mg/day) Outcome† Adverse events
Pre-ACTH Post-ACTH
Male, White 59 MN 4851 400 Complete remission None
Male, Hispanic 77 MN 6749 1540 Partial remission None
Male, White 58 MN 4598 1242 Partial remission None
Male, White 55 MN 8153 1935 Partial remission Hyperglycemia
Male, White 27 MN 9000 3000 Partial remission None
Female, White 24 MN 8900 6000 No response Weight gain
Female, White 75 MN 3469 34 Complete remission Bone demineralization
Male, White 49 MN 9150 2948 Partial remission None
Male, White 46 MN 11911 13338 No response None
Male, White 53 MN 5700 694 Partial remission None
Female, White 70 MN 2625 240 Complete remission Hyperglycemia
    * Results from a retrospective case series evaluating all known cases of proteinuria due to nephrotic syndrome treated with Acthar outside of research settings before December 31, 2009. 21 patients with nephrotic syndrome of varying etiologies were included: 11 with idiopathic membranous nephropathy (MN), 4 with membranoproliferative glomerulonephritis (MPGN), 1 with focal segmental glomerulosclerosis (FSGS), 1 with minimal change disease (MCD), 1 with immunoglobulin A (IgA) nephropathy, 1 with class V systemic lupus erythematosus (SLE), 1 with monoclonal diffuse proliferative glomerulonephritis (DPGN), and 1 with unbiopsied nephrotic syndrome.
    † Complete remission was defined as stable or improved renal function with final proteinuria falling to <500 mg/day. Partial remission was defined as stable or improved renal function with ≥50% reduction in proteinuria and final proteinuria 500–3500 mg/day.

Individual results in all 10 remaining patients with nephrotic syndrome of varying etiologies*3

Patient Age Diagnosis Proteinuria (mg/day) Outcome† Adverse events
Pre-ACTH Post-ACTH
Male, White 81 MPGN 13073 3741 Did not meet criteria None
Female, White 28 MPGN 5500 4825 No response None
Female, White 53 MPGN 12398 4560 Limited response None
Female, White 47 MPGN 10244 3878 Did not meet criteria None
Female, White 57 MCD 18553 18557 No response None
Female, Hispanic 63 FSGS 10275 2970 Partial remission None
Male, Black 75 IgA nephropathy 4952 42 Complete remission None
Female, White 32 SLE class V 1340 2290 Did not meet criteria Weight gain
Male, White 36 Monoclonal DPGN 8560 8500 No response Acute renal failure
Male, White 74 Nephrotic syndrome 5805 8708 No response None
  • The usual dose of H.P. Acthar Gel is 40 to 80 units given intramuscularly or subcutaneously every 24 to 72 hours1
    –Dosage and frequency may be individualized according to the medical condition, severity of the disease, and initial response of the patient1
    –In this 21-patient case series, the most common Acthar dosing regimen was 80 units given subcutaneously twice a week for 6 months3
    Common side effects include fluid retention, behavioral and mood changes, change in glucose tolerance, increased appetite, increased blood pressure, and weight gain.1

These results are based on a retrospective case series of 21 patients and may not be fully representative of outcomes in the overall patient population. Most patients were on multiple therapies. The clinical outcomes may not be solely attributable to Acthar.