Facts About Acthar® Gel

Updated August 31, 2021

Acthar® Gel (repository corticotropin injection) Supporting Patients

See Full Prescribing Information and Important Safety Information for Acthar Gel.

Our mission at Mallinckrodt is Managing Complexity. Improving Lives. Our employees live this mission every day, and we're focused on providing safe, effective treatments that make a difference in the lives of underserved patients, especially those with severe and critical conditions. 

Mallinckrodt is committed to supporting patients and healthcare providers. We have a dedicated team ready to help patients and healthcare providers work through the Acthar Gel approval process, assist patients with advocating for themselves as well as provide support throughout treatment. Watch the video below to hear Bianca’s story and how our patient support services assisted her.

The Facts About Acthar Gel

Acthar Gel is a naturally sourced complex mixture of adrenocorticotropic hormone analogs and other pituitary peptides that is indicated in multiple therapeutic areas including rheumatology, ophthalmology, nephrology, pulmonology and neurology.1 

Acthar Gel is FDA-Approved for 19 Indications
Acthar Gel has an established efficacy and safety profile as well as a long track record of clinical evidence that spans nearly 70 years. In addition, Acthar Gel has 19 indications, including infantile spasms which was approved in 2010.Please see Indications and Important Safety Information for Acthar Gel below.

Acthar Gel is often prescribed by doctors predominately as a later-line treatment to a small subset of patients suffering from various, sometimes devastating, diseases and may be the last treatment option for those who need an alternative therapy.

Mark Trudeau’s U.S. House Committee on Oversight and Reform Testimony on Mallinckrodt’s Pricing Policies and Modernization Strategy for Acthar Gel on October 1, 2020

Acthar Gel’s label was updated in February 2021 after the FDA was provided with safety monitoring and reporting data spanning more than 60 years. In addition to modernizing the prescribing information, the statement, "Common adverse reactions for Acthar Gel are similar to those of corticosteroids," was removed in the prescribing information (PI) highlights section of the label; however, the warnings and precautions section, which notes potential steroidogenic effects, remains largely unchanged. These important updates provide up-to-date safety information based on a sizable accumulation of pharmacovigilance reporting and clinical trial data, including phase IV clinical studies. The PI was previously updated in 2019 to characterize Acthar Gel as a naturally sourced complex mixture of adrenocorticotropic hormone analogs and other pituitary peptides.1

Mallinckrodt’s Investment in Acthar Gel

Since acquiring Acthar Gel in 2014, Mallinckrodt has made, and continues to make, a significant investment in the drug. For example, the company built on existing data through further studies that evaluated the safety and efficacy of Acthar Gel within our indications. Mallinckrodt initiated eight studies targeting combined enrollment of nearly 900 patients, including five randomized controlled clinical trials.2 The company has modernized manufacturing, further characterized the mechanism of action and expanded medical affairs and research activities. In addition, Mallinckrodt has a dedicated team prioritizing access and providing support.

Significant Clinical Evidence Supports the Efficacy of Acthar Gel

On April 15, 2020, Mallinckrodt announced the publication of findings from its randomized, placebo-controlled, double-blind Phase 4 study to assess the safety and efficacy of Acthar in patients with persistently active rheumatoid arthritis (RA) despite treatment with stable background disease-modifying antirheumatic drugs (DMARDs) and low-dose glucocorticoids.3 Results of the study were published in Rheumatology and Therapy, an open access peer-reviewed journal. 

Two randomized clinical trial were submitted in support of FDA approval of Acthar Gel’s effectiveness as a treatment for Infantile Spasms (IS)1, one of which compared Acthar Gel to prednisone. In this pivotal trial, 13 of 15 (86.7%) patients had a positive response to Acthar Gel vs. 4 of 14 (28.6%) that responded to prednisone (p<0.002). Patients who responded in the study treated with a two-week course of Acthar Gel therapy experienced complete suppression of the two key measures of disease – spasms and hypsarrhythmia.1 Irritability and voracious appetite were the most frequent side effects, but no infant required stopping or modifying treatment.4 The IS clinical trial results appear in Section 14 of the full Prescribing Information1 for the drug.

 

Completed and Ongoing Clinical Studies

Completed and ongoing clinical studies are referenced below. Each link takes you to details about the study that can be found on www.clinicaltrials.gov. We expect these important investments will build upon the existing body of clinical evidence, further evaluating the effectiveness and safety of Acthar Gel.

• Phase 4 trial in Pulmonary Sarcoidosis
• Phase 4 trial in Lupus
• Phase 4 trial in Rheumatoid Arthritis
• Phase 4 trial in Multiple Sclerosis
• Phase 4 trial in Uveitis
• Phase 4 study in Focal Segmental Glomerulosclerosis (Nephrotic Syndrome)
• Phase 4 study in Keratitis
• Observational study in the treatment of MS Relapse

About Acthar Gel (repository corticotropin injection)

INDICATIONS

Acthar® Gel is indicated for:
  • Inducing a diuresis or a remission of proteinuria in nephrotic syndrome without uremia of the idiopathic type or that due to lupus erythematosus
  • Monotherapy for the treatment of infantile spasms in infants and children under 2 years of age
  • Treatment of acute exacerbations of multiple sclerosis in adults. Controlled clinical trials have shown Acthar to be effective in speeding the resolution of acute exacerbations of multiple sclerosis. However, there is no evidence that it affects the ultimate outcome or natural history of the disease
  • Severe acute and chronic allergic and inflammatory processes involving the eye and its adnexa such as: keratitis, iritis, iridocyclitis, diffuse posterior uveitis and choroiditis, optic neuritis, chorioretinitis, anterior segment inflammation
  • Symptomatic sarcoidosis
  • Treatment during an exacerbation or as maintenance therapy in selected cases of systemic lupus erythematosus
  • Treatment during an exacerbation or as maintenance therapy in selected cases of dermatomyositis (polymyositis)
  • Adjunctive therapy for short-term administration (to tide the patient over an acute episode or exacerbation) in: psoriatic arthritis; rheumatoid arthritis, including juvenile rheumatoid arthritis (selected cases may require low-dose maintenance therapy); ankylosing spondylitis
 

IMPORTANT SAFETY INFORMATION

Contraindications

Acthar is contraindicated:
  • For intravenous administration
  • In infants under 2 years of age who have suspected congenital infections
  • With concomitant administration of live or live attenuated vaccines in patients receiving immunosuppressive doses of Acthar
  • 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, adrenocortical hyperfunction, or sensitivity to proteins of porcine origin

Warnings and Precautions

  • The adverse effects of Acthar are related primarily to its steroidogenic effects
  • Acthar may increase susceptibility to new infection or reactivation of latent infections
  • Suppression of the hypothalamic-pituitary-adrenal (HPA) axis may occur following prolonged therapy with the potential for adrenal insufficiency after withdrawal of the medication. Adrenal insufficiency may be minimized by tapering of the dose when discontinuing treatment. During recovery of the adrenal gland patients should be protected from the stress (e.g., trauma or surgery) by the use of corticosteroids. Monitor patients for effects of HPA axis suppression after stopping treatment
  • Cushing’s syndrome may occur during therapy but generally resolves after therapy is stopped. Monitor patients for signs and symptoms
  • Acthar can cause elevation of blood pressure, salt and water retention, and hypokalemia. Monitor blood pressure and sodium and potassium levels
  • Acthar often acts by masking symptoms of other diseases/disorders. Monitor patients carefully during and for a period following discontinuation of therapy
  • Acthar can cause gastrointestinal (GI) bleeding and gastric ulcer. There is also an increased risk for perforation in patients with certain GI disorders. Monitor for signs of perforation and bleeding
  • Acthar may be associated with central nervous system effects ranging from euphoria, insomnia, irritability, mood swings, personality changes, and severe depression to psychosis. Existing conditions may be aggravated
  • Patients with comorbid disease may have that disease worsened. Caution should be used when prescribing Acthar in patients with diabetes and myasthenia gravis
  • Prolonged use of Acthar may produce cataracts, glaucoma, and secondary ocular infections. Monitor for signs and symptoms
  • Acthar is immunogenic and prolonged administration of Acthar may increase the risk of hypersensitivity reactions. Neutralizing antibodies with chronic administration may lead to loss of endogenous ACTH and Acthar activity
  • There may be an enhanced effect in patients with hypothyroidism and in those with cirrhosis of the liver
  • Long-term use may have negative effects on growth and physical development in children. Monitor pediatric patients
  • Decrease in bone density may occur. Bone density should be monitored in patients on long-term therapy
  • Pregnancy Class C: Acthar has been shown to have an embryocidal effect and should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus

Adverse Reactions

  • Commonly reported postmarketing adverse reactions for Acthar include injection site reaction, asthenic conditions (including fatigue, malaise, asthenia, and lethargy), fluid retention (including peripheral swelling), insomnia, headache, and blood glucose increased
  • The most common adverse reactions for the treatment of infantile spasms (IS) are increased risk of infections, convulsions, hypertension, irritability, and pyrexia. Some patients with IS progress to other forms of seizures; IS sometimes masks these seizures, which may become visible once the clinical spasms from IS resolve

Other adverse events reported are included in the full Prescribing Information. 

Please see full Prescribing Information for additional Important Safety Information which can found at Acthar.com. 

 

1Acthar® Gel (repository corticotropin injection) [prescribing information]. Mallinckrodt ARD LLC.

2Data on File--ref-04625

3Fleischmann, R, Furst, D.E., Connolly-Strong, E., Liu, J., Zhu, J, Brasington, R. Repository Corticotropin Injection for Active Rheumatoid Arthritis Despite Aggressive Treatment: A Randomized Controlled Withdrawal Trial, Rheumatology Therapy, https://link.springer.com/article/10.1007/s40744-020-00199-3

4Baram, T.Z., Mitchell, W.G., Tournay, A., Snead, O.C., Hanson, R.A., Horton, E.J., High-dose Corticotropin (ACTH) Versus Prednisone for Infantile Spasms: A Prospective, Randomized, Blinded Study. Pediatrics, 1996; 97 (3) 375-379

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