14. Clinical Studies

The effectiveness of H.P. Acthar Gel as a treatment for infantile spasms was demonstrated in a single blinded (video EEG interpreter blinded) clinical trial in which patients were randomized to receive either a 2 week course of treatment with H.P. Acthar Gel (75 U/m² intramuscular twice daily) or prednisone (1 mg/kg by mouth twice daily). The primary outcome was a comparison of the number of patients in each group who were treatment responders, defined as a patient having complete suppression of both clinical spasms and hypsarrhythmia on a full sleep cycle video EEG performed 2 weeks following treatment initiation, rated by an investigator blinded to treatment. Thirteen of 15 patients (86.7%) responded to H.P. Acthar Gel as compared to 4 of 14 patients (28.6%) given prednisone (p<0.002). The 2-week treatment was followed by a 2-week period of taper. Nonresponders to the prednisone treatment were eligible to receive H.P. Acthar Gel treatment. Seven of 8 patients (87.5%) responded to H.P Acthar Gel after not responding to prednisone. Similarly, the 2 nonresponder patients from the H.P. Acthar Gel treatment were eligible to receive treatment with prednisone. One of the 2 patients (50%) responded to the prednisone treatment after not responding to H.P. Acthar Gel.

A supportive single-blind, randomized clinical trial comparing high-dose, long-duration treatment (150 U/m² once daily for 3 weeks, n=30) of H.P. Acthar Gel with low-dose, short-duration treatment (20 U once daily for 2 weeks, n=29) for the treatment of infantile spasms was also evaluated in infants and children less than 2 years of age. Nonresponders (defined as in the previously described study) in the low-dose group received a dose escalation at 2 weeks to 30 U once daily. Nominal statistical superiority of the high dose treatment, as compared to the low dose treatment, was observed for cessation of spasms but not for the resolution of hypsarrhythmia.

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Questcor

Indications for Use

H.P. Acthar^® Gel (repository corticotropin injection) is an adrenocorticotropic hormone (ACTH) analogue used for:

Monotherapy treatment of infantile spasms (IS) in infants and children under 2 years of age.
The treatment of exacerbations of multiple sclerosis in adults.
For inducing a diuresis or a remission of proteinuria in the nephrotic syndrome without uremia of the idiopathic type or that due to lupus erythematosus.
The following: rheumatic disorders; collagen diseases; dermatologic diseases; allergic states; ophthalmic diseases and respiratory diseases.

Important Safety Information

Acthar should never be given intravenously. It is contraindicated in patients with scleroderma, osteoporosis, systemic fungal infections, ocular herpes simplex, recent surgery, history of or the presence of a peptic ulcer, congestive heart failure, uncontrolled hypertension, primary adrenocortical insufficiency or adrenocortical hyperfunction or sensitivity to proteins of porcine origin. Acthar is contraindicated in children under 2 years of age with suspected congenital infections. Administration of live or live attenuated vaccines is contraindicated in patients receiving immunosuppressive doses of Acthar.

The adverse effects that may occur with Acthar are related primarily to its steroidogenic effects and are similar to corticosteroids. There may be increased susceptibility to new infection and increased risk of reactivation of latent infections. Adrenal insufficiency may occur after abrupt withdrawal of the drug following prolonged therapy. Cushing’s syndrome, elevated blood pressure, salt and water retention, and hypokalemia may be seen. Masking of symptoms of other underlying disease/disorders may occur. There is a risk of gastrointestinal perforation and bleeding with increased risk of perforation in patients with certain GI disorders. Onset or worsening of euphoria, insomnia, irritability (especially in infants), mood swings, personality changes, depression, and psychosis may occur. Caution should be used when prescribing Acthar to patients with diabetes or myasthenia gravis. Prolonged use may produce cataracts, ocular infections or glaucoma. Use in patients with hypothyroidism or liver cirrhosis may result in an enhanced effect. There may be negative effects on growth and physical development and decreases in bone density.

Specific adverse reactions reported in IS clinical trials in infants and children under 2 years of age included: infection, hypertension, irritability, Cushingoid symptoms, constipation, diarrhea, vomiting, pyrexia, weight gain, increased appetite, decreased appetite, nasal congestion, acne, rash, and cardiac hypertrophy. Convulsions were also reported, but these may actually be occurring because some IS patients progress to other forms of seizures and IS sometimes mask other seizures, which become visible once the clinical spasms from IS resolve. Other adverse reactions in adults and children over 2 years of age included: abdominal distension, anxiety, asthma, chest discomfort, congestive heart failure, dizziness, dyspnea, erythema, fatigue, flushing, headache, hyperhidrosis, hypersensitivity or allergic reactions, injection site pain, muscle weakness, palpitations, peripheral edema, tachycardia, and weakness.

This is a summary only. For a full list of warnings, precautions, and adverse events related to Acthar, please refer to the full Prescribing Information including Medication Guide for the treatment of Infantile Spasms.

H.P. Acthar^® Gel and Questcor^® are registered trademarks of Questcor Pharmaceuticals, Inc.