Acthar Gel has been proven in multiple clinical trials to stop spasms and resolve hypsarrhythmia, thereby satisfying the AAN and CNS guidelines.2,5,12-14

In fact, in the pivotal clinical study, 86.7% of children had no spasms and no hypsarrhythmia by week 2 after injections of Acthar Gel 75 U/m2 twice a day compared to 28.6% of patients who received prednisone.2,3

Acthar Gel was shown to be efficacious in symptomatic and cryptogenic cases of IS2,3

  • In the pivotal prospective, randomized, single-blind, crossover efficacy study for Acthar Gel:
    • A 2-week course of high-dose Acthar Gel (75 U/m2 BID) had a higher response rate (87%) than prednisone 1 mg/kg BID (29%) for both cessation of spasms and resolution of hypsarrhythmia
    • Acthar Gel stopped spasms and eliminated hypsarrhythmia in both cryptogenic and symptomatic (including tuberous sclerosis [TS]) cases of IS (n=15)

aP=0.0015 vs prednisone 1 mg/kg BID.

Of the 10 infants who did not respond to prednisone, 8 received Acthar Gel and 7 (87.5%) responded.1

Of the 2 infants who did not respond to Acthar Gel, 2 received prednisone and 1 (50.0%) responded.1

Study design: This prospective, randomized, single-blinded study compared the efficacy of corticotropin (Acthar Gel 150 U/m2/day) and prednisone (2 mg/kg/day) given for 2 weeks. Twenty-nine patients were entered into the study. Response required both cessation of spasms and elimination of hypsarrhythmia (confirmed by electroencephalogram) at the end of the 2-week treatment period.2

Safety findings: Side effects such as hypertension and hyperglycemia were monitored. Irritability and voracious appetite were the most frequent side effects, but no infant required stopping or modifying treatment.2

Acthar Gel treatment resulted in early (2 weeks) and sustained (3 months) responses15

A US, multicenter, prospective study of 230 patients evaluated clinical remission and resolution of hypsarrhythmia.

at 2 weeks (P=0.027)

68% for Acthar Gel
(66 of 97 patients)
49% for vigabatrin
(23 of 47 patients)

at 3 months (P=0.038)

55% for Acthar Gel
(53 of 97 patients)
36% for vigabatrin
(17 of 47 patients)

Common adverse reactions for Acthar Gel are similar to those of corticosteroids and include fluid retention, alteration in glucose tolerance, elevation in blood pressure, behavioral and mood changes, increased appetite, and weight gain.

Study design: A multicenter, prospective study of 230 infants diagnosed with IS. Patients were treated with standard therapy (ACTH, oral corticosteroids, or vigabatrin) or nonstandard therapy (all other medications). The study evaluated early (at 2 weeks) and sustained (at 3 months) response to initial treatments by assessing clinical remission and electrographic resolution of hypsarrhythmia.15

Study limitations:

  • Therapies were not randomized, and providers were allowed to choose the medications. The authors did not observe significant differences in overall treatment response patterns based on underlying etiology or development but cannot exclude unknown confounders given the observational nature of the study design
  • Developmental status classifications were based on clinical examination and not on formal assessment tools
  • Although this study showed a trend for higher response rates with high-dose Acthar Gel compared with low/intermediate-dose Acthar Gel, it was not sufficiently powered to determine differences in outcome between high-dose and low/intermediate-dose Acthar Gel response
  • Study was not sufficiently powered to identify response to treatment based on each specific etiology, and merging of etiological categories was necessary
  • Study was not a head-to-head trial between Acthar Gel, vigabatrin, OCS, and nonstandard therapies

For increased efficacy, Acthar Gel should be dosed as recommended in the FDA-approved label1

In an independent analysis of previously published data from randomized controlled trials, greater overall efficacy of Acthar Gel was seen with BID administration than QD administration.3
The analysis included 3 randomized controlled trials and considered safety data from a total of 319 patients.

Cessation of Spasms and Resolution of Hypsarrhythmia

In the treatment of IS, Acthar Gel is given as an injection into the muscle. Do not inject it under the skin, into a vein, or give it to a child by mouth. Acthar Gel is contraindicated where congenital infections are suspected in infants. Acthar Gel can cause GI bleeding and gastric ulcer. There is also an increased risk for perforation in patients with certain gastrointestinal disorders. Monitor for signs of bleeding.

Previously published data from nonrandomized controlled trials were consistent with this analysis

  • 80% (4 of 5 patients) overall response rate when dosed 100 to 150 U/m2/d (40-80 U/d)3,12
  • 93% (14 of 15 patients) and 97% (29 of 30 patients) overall response rate when dosed 75 U/m2 BID3,13,14

Helpful Resources

Step-by-Step Injection Training Guide

Download this convenient and easy-to-follow Injection Training Guide for caregivers of children with IS.

Acthar Referral Form

Download, complete, and fax the Referral Form to order Acthar Gel for your appropriate patients.

Take the guesswork out of dosing Acthar Gel for IS

From per-injection volume to number of vials, our calculator simplifies the process.