Acthar

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Acthar

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Medically reviewed

Rosario Oropesa

Last updated on 22/12/2025

This page provides general, reference-level information compiled from official medical sources. It is not a substitute for professional medical advice, diagnosis, or treatment. For decisions about your health, please consult a qualified healthcare professional.

Overview of Acthar

Acthar: Definition, Composition, and Class

Property Description
Active ingredient Corticotropin (ACTH)
Form Injectable gel (Repository injection)
Pharmacological class Adrenal cortex stimulant, Peptide hormone
General purpose Systemic anti-inflammatory and immune modulation
Origin Natural polypeptide (Porcine pituitary glands)

Acthar is a prescription medication containing the single active ingredient Corticotropin, formally known as Adrenocorticotropic hormone (ACTH). It is fundamentally classified as an adrenal cortex stimulant and a peptide hormone.

This pharmaceutical entity is structurally a natural polypeptide, derived from a natural animal source, specifically the porcine pituitary glands. The Corticotropin molecule itself is a complex protein composed of 39 amino acids, and its purpose is to mimic the action of the body's own pituitary hormone, placing it within the domain of biologic products designed to modulate internal hormonal systems. As an ACTH analogue, Corticotropin stimulates the adrenal cortex, acting as a signal that prompts the body's own adrenal glands into action.


What Type of Preparation is Acthar?

Acthar is supplied as a highly purified injectable gel and is specifically identified as a repository injection. This form is administered via injection (subcutaneously or intramuscularly) and is not a conventional liquid solution. Its prescription-only status is a key feature, emphasizing required medical oversight.

The distinguishing feature of the repository gel formulation is that it is engineered to release the Corticotropin slowly after administration. Unlike fast-acting, aqueous injections that provide an immediate peak, this long-acting delivery method creates a depot effect, ensuring the active ingredient remains available in the body over an extended period to provide sustained stimulation. This formulation is a repository corticotropin injection designed for prolonged action, delivering a steady, sustained effect over time.


The General Purpose of Acthar

The general purpose of Acthar is to activate the body's own internal mechanism for systemic modulation and relief. It achieves this by acting as a signal to the adrenal glands, two small organs responsible for producing crucial steroid hormones. A typical, neutral scenario for this action is helping patients manage chronic inflammatory conditions.

By compelling these glands to release their natural steroid hormones, such as cortisol, Acthar helps the body manage and counteract widespread inflammation and to alter the activity of the immune system. This mechanism provides a broad, systemic approach to modulating immune and inflammatory responses across various body systems.

What side effects are possible with Acthar?

Possible Side Effects and Safety Information

The officially documented safety profile for Acthar (corticotropin repository injection) is characterized by systemic effects similar to those produced by corticosteroids, reflecting its role as an adrenal cortex stimulant. Adverse reactions are classified by frequency and the organ system affected, as outlined in regulatory documents.

Adverse Reaction Scope

Classification Examples of Reactions
Most Common (in clinical data) Infections, Convulsions, Hypertension, Irritability, Pyrexia
System-Organ Classes Endocrine, Fluid and Electrolyte (e.g., fluid retention, hypokalemia), Musculoskeletal (e.g., osteoporosis), Psychiatric and Nervous System (e.g., mood disturbances)

Serious Adverse Reactions and Constraints

The regulatory labeling identifies the potential for serious adverse reactions, including the risk of severe infections, gastrointestinal perforation and bleeding, and generalized hypersensitivity reactions such as anaphylaxis.

Safety concerns are explicitly linked to the duration of therapy. Prolonged use increases the risk of developing a Cushingoid state and conditions like posterior subcapsular cataracts and glaucoma. The sudden withdrawal of the medication after prolonged use may lead to adrenal insufficiency.

Population-Specific Notes

The label includes specific safety considerations for certain groups. Pediatric patients may experience suppressed growth and development with prolonged administration. The medication is also noted for its potential for fetal harm based on animal studies, necessitating careful observation of infants born to treated mothers for signs of hypoadrenalism.

Safety-Related Restrictions

Acthar is formally contraindicated (should not be used) in patients with specific conditions, including systemic fungal infections, osteoporosis, recent surgery, history of peptic ulcer, congestive heart failure, and sensitivity to proteins of porcine origin.

Overdose and Emergency Response

The official regulatory profile for repository corticotropin injection describes overdosage as the exaggeration of known pharmacological effects resulting from prolonged or excessive adrenal stimulation, rather than a typical single-dose acute event. These manifestations are tied to the overproduction of natural steroid hormones.

Documented Overdose Manifestations

Manifestations of overexposure are characterized by signs of a Cushingoid state, including unusual weight gain, a rounded face, thinning skin, and easy bruising. Over-stimulation can also lead to severe metabolic effects, notably salt and water retention and a dangerous reduction in blood potassium levels (hypokalemia). Serious potential outcomes include severe hypertension and the risk of gastrointestinal perforation or bleeding.

Emergency Actions and When to Seek Help

Regulators define specific clinical thresholds that mandate seeking immediate medical help:

Condition Required Action
Severely Elevated Blood Pressure Seek immediate medical attention for systolic pressure 180 mmHg or diastolic pressure 120 mmHg.
Gastrointestinal Complications Get emergency help for signs of internal bleeding, such as vomiting blood or passing black or tarry stool.

Overdosage management is officially described as symptomatic and supportive, with no specific antidote documented. Required support involves monitoring blood pressure and serum sodium and potassium levels. Children under 2 years of age have a documented increased risk for complications, including Cardiac Hypertrophy.

Therapeutic Uses of Acthar

Acthar is commonly used to help with a wide range of conditions characterized by symptoms related to systemic imbalance, neurological disruption, or fluid imbalance, aiming to provide support that helps ease the overall symptom burden. This medication is relevant when short-term symptomatic assistance is appropriate in contexts involving acute or disruptive episodes.


Therapeutic Scope and Patient Benefit

This medication is applied across various therapeutic domains, including inflammatory diseases like Rheumatoid Arthritis, Systemic Lupus Erythematosus, and Psoriatic Arthritis, as well as severe neurological presentations such as Infantile Spasms and acute exacerbations of Multiple Sclerosis. It is also relevant for certain types of Nephrotic Syndrome. This medication is generally used to help address symptom clusters that may become intense or disruptive, such as severe joint pain, swelling, and fluid retention.

The goal of using this medication is generally to provide supportive relief when symptoms interfere with routine activities or when functional stability becomes affected during periods of heightened symptoms.

Quick Fact: Applied for Acute Symptom Flares The medication is commonly used during phases of heightened symptoms to assist with managing severe inflammation, specific seizure activity, or excess fluid accumulation, and may help patients cope more steadily with symptom fluctuations.

Regulatory References

  1. NIH MedlinePlus guidance

Eligibility and Restrictions for Use

Eligibility: Who Can and Cannot Use Acthar?

Eligibility for Acthar (Repository Corticotropin Injection) is strictly defined by regulatory documents, focusing on patient age, existing conditions, and physiological status. The medicine is formally contraindicated and must not be used by patients with several conditions, including systemic fungal infections, uncontrolled hypertension, scleroderma, osteoporosis, and a history of peptic ulcers. Patients with Primary Adrenocortical Insufficiency or known sensitivity to porcine proteins are also excluded.

Regulatory agencies specify approved use in certain age groups: infants under two years old for Infantile Spasms, and adults for acute exacerbations of Multiple Sclerosis. Use is also established for other listed conditions in adults and children over two years.

Certain populations require caution or conditional use. Caution is advised for those with conditions like liver cirrhosis, renal insufficiency, or diabetes. Furthermore, its use during pregnancy and lactation is only allowed if a benefit-risk assessment justifies the potential risk, and data for the geriatric population is officially noted as not established.

What should I know about interactions with other medicines?

Interactions with other medicines and products

Official regulatory documentation identifies specific considerations regarding the use of Acthar (repository corticotropin injection) with other medical products and procedures. These statements provide essential constraints and requirements for healthcare providers to ensure the safe administration of the medication.

Documented Interaction Requirements

The most stringent requirement concerns the use of vaccines. Concomitant administration of live or live attenuated vaccines is strictly contraindicated in patients who are receiving immunosuppressive doses of Acthar. This constraint is based on the potential for the medication to suppress the immune response required to safely and effectively process the vaccine.

Interacting Product Category Official Constraint/Requirement Basis of Interaction
Live or live attenuated vaccines Contraindicated with immunosuppressive doses Reduced immune response and effectiveness
Diuretics (e.g., thiazides, loop diuretics) Use with caution; Monitor patients closely May accentuate electrolyte loss

General Interaction Profile

Regarding medications that affect fluid and electrolyte balance, the official profile notes a specific interaction with diuretics. Use with diuretics, such as those prescribed for high blood pressure or fluid retention, may accentuate the loss of electrolytes. Close monitoring by a healthcare professional is necessary when these two product classes are used together. No other specific drug classes or individual medicinal products are explicitly designated as interacting in a way that requires specific dose modification or complete avoidance within the core interaction section of the official label.

Mechanism of Action

Mechanism of Action of Acthar (Repository Corticotropin Injection)

Acthar (repository corticotropin injection) is a complex mixture of peptides that functions as an agonist by binding to and activating specific cell-surface receptors, primarily within the Melanocortin Receptor (MCR) family.

The principal mechanistic interaction involves the MC2 receptor located on the adrenal cortex. Activation of the MC2 receptor initiates a signaling cascade involving cAMP and Protein Kinase A (PKA). This intracellular process promotes the synthesis and subsequent release of numerous endogenous corticosteroids and androgenic steroids (e.g., cortisol) from the adrenal gland.

Additionally, the peptide mixture interacts with MC3 and MC5 receptors. Activation of these receptors on immune and inflammatory cells modulates downstream signaling pathways, affecting processes such as leukocyte migration, cellular differentiation, and the production of various cytokines. This systemic modulation results in altered immune and inflammatory cell function.

Dosage and Administration Information

Acthar Gel (repository corticotropin injection) is administered by subcutaneous (SC) or intramuscular (IM) injection. Intravenous administration is strictly contraindicated.

Administration and Dosage Rules

Acthar Gel must be warmed to room temperature before use by leaving the vial out for 15 to 30 minutes, or by rolling it between the palms. This allows the gel to liquefy for proper withdrawal and injection. Dosing is highly individualized based on the medical condition and patient response, but standardized regimens exist for certain conditions:

  • Infantile Spasms (IS): The recommended regimen is 150 USP units/m^2 daily, divided into two IM injections of 75 USP units/m^2 each, administered over a two-week period. Following this initial treatment, the dose must be gradually tapered and discontinued over an additional two weeks to prevent adrenal insufficiency. For IS treatment, only the multi-dose vial should be used, administered intramuscularly (IM).
  • Acute Multiple Sclerosis (MS) Exacerbations: The dose is typically 80-120 USP units administered daily, via IM or SC injection, for two to three weeks. Dosage should be individualized to the patient's medical condition.
  • Other Conditions: The usual dose is 40-80 USP units given IM or SC every 24 to 72 hours. As with other regimens, tapering the dose and increasing the injection interval is necessary to discontinue the medication gradually.

Recent Clinical Evidence

Research Evidence / Overview of Studies for Acthar (Corticotropin)

Evidence for Use in Infantile Spasms (West Syndrome)

Research exploring the use of repository corticotropin injection for Infantile Spasms (a serious form of epilepsy in infants) includes a key randomized, controlled, single-blind trial that evaluated the use of the drug against an active comparator, prednisone. Additionally, studies include analyses of patient data from prospective and retrospective observational settings, which examine how symptoms change over time. These trials were primarily applied in studies examining infants and children under two years old. Primary measures examined were the cessation of clinical spasms and the resolution of hypsarrhythmia (abnormal EEG patterns).

The randomized trial and subsequent studies reported patterns observed in the studies related to both the cessation of spasms and the change in EEG readings over a short-term period (typically two weeks). Follow-up cohorts within the research have also studied patterns related to developmental outcomes in the observed populations.

Evidence for Acute Exacerbations of Multiple Sclerosis (MS)

Research exploring the use of repository corticotropin injection in the context of acute exacerbations (relapses) of Multiple Sclerosis includes historical controlled clinical trials. More recent evidence is derived from observational settings and prospective studies that research examined patterns of use in daily-life functioning contexts. Research examined outcomes related to episodic or acute changes in neurological function, focusing on measures of recovery speed from the acute attack and change in functional deficit scores in adult patients.

Early controlled studies reported patterns related to the monitoring of daily-life functioning or activity level in the observed populations over defined time intervals. Recent observational data provides context related to research scenarios involving patients with prior inadequate responses to standard high-dose corticosteroids.

Evidence for Use in Nephrotic Syndrome

Studies examining the use of the drug for Nephrotic Syndrome include systematic reviews that compile findings from many small, uncontrolled, or retrospective studies. The research has focused primarily on populations presenting with resistant or refractory disease. Studies monitored outcomes related to physiological strain or stress, specifically measurements related to the change in proteinuria (too much protein in the urine) and the monitoring of fluid-related outcomes (fluid loss).

Research Gaps and Areas of Uncertainty

A research limitation frame is that sample sizes were modest in many of the controlled or randomized trials across the indications. Comparative evidence against some other standard treatments is limited for many indications. The evidence quality varies across studies, particularly for the inflammatory conditions, where findings were often based on observational cohorts rather than large-scale, controlled trials. Because the treatment is typically used for short-term management of acute events or flares, the long-term effects are not fully established, and there is limited information on the impact on overall disease progression.

Key Studies & References

  1. A Prospective Observational Registry of Repository Corticotropin Injection (Acthar® Gel) for the Treatment of Multiple Sclerosis Relapse
  2. Repository Corticotropin Versus Glucocorticoid for Nephrotic Syndrome: When Will We See the Evidence?

Frequently Asked Questions (FAQ)

Common questions about Acthar (FAQ)

Q: Is Acthar the same kind of medicine as a corticosteroid like prednisone?

Acthar is officially classified as an adrenal cortex stimulant. This means it signals your body's adrenal glands to produce and release its own natural steroid hormones, such as cortisol. It is not an exogenous corticosteroid, which is a synthetic steroid hormone that is taken directly, like prednisone.

Q: Does Acthar cause weight gain or changes in appetite?

Weight gain is a reported side effect, especially when the medicine is used for a prolonged time. This may be associated with the drug's effects on steroid hormones, as documented in official safety information. Official reports also indicate that changes in appetite (either a loss or decrease) have been reported by patients.

Q: Can Acthar cause my body to stop producing its own natural stress hormone, and what does that mean?

Yes, using Acthar for a long time can cause the adrenal glands to stop making enough of the natural stress hormone, cortisol. This condition is called adrenal insufficiency and can occur after the medicine is stopped. Official prescribing information notes that tapering the dose is necessary to help the adrenal glands recover.

Q: Does the medicine come in a pre-filled syringe or a multi-dose vial?

Acthar Gel may be supplied in two forms, depending on the indication: a multi-dose vial with a syringe or a single-dose pre-filled self-injection device. The choice of formulation is determined by the healthcare provider based on the patient's condition and the official prescribing information.

Q: Why do some people experience injection site reactions, and what are they?

Injection site reactions are a commonly reported adverse reaction documented in the official product information. These reactions are typically localized and can include symptoms such as pain, itching, redness, swelling, or inflammation at the area where the injection was given.

Q: Can Acthar treatment be used during pregnancy or while breastfeeding?

Official warnings state that this medicine may cause fetal harm when administered during pregnancy. Therefore, women should be informed of the potential risks to the fetus. Regarding breastfeeding, it is unknown if the drug passes into human milk, so the decision regarding breastfeeding or discontinuing the drug must be made by the healthcare provider after assessing the risks and benefits.

Q: Is Acthar appropriate for elderly patients with multiple health conditions?

The official label does not specifically establish safety and effectiveness data for the geriatric population. However, regulatory documents list several conditions common in the elderly (such as heart failure, high blood pressure, and osteoporosis) as formal reasons not to use the drug (contraindications). The presence of comorbid diseases may require careful consideration by the healthcare provider.

Q: What kind of financial assistance or patient support programs are available for Acthar?

The manufacturer offers support programs to help eligible patients with the costs of the medication. These programs may include a commercial co-pay program for insured patients and a patient assistance program for those who have no insurance and meet income requirements.

Q: Do I need to change my diet while I am taking Acthar?

Because Acthar can cause fluid retention, elevated blood pressure, and low potassium levels (hypokalemia), a healthcare provider may recommend dietary changes. Specifically, salt restrictions or the use of potassium supplements may be recommended to help manage these documented fluid and electrolyte effects.

Q: Why is Acthar considered a 'specialty' medicine that is often difficult to get covered by insurance?

The manufacturer acknowledges that, like other specialty medicines, the process for obtaining coverage can be complex. This often involves steps like prior authorization and appeals with insurance companies to confirm the medical necessity of the treatment for a patient's condition.

Q: Are there different brand names or formulations of the drug that work similarly to Acthar?

Official documentation confirms that another brand name product, Purified Cortrophin Gel, which also contains repository corticotropin injection, is available for some of the same FDA-approved conditions as Acthar Gel.

Q: What is the expected duration of treatment for Acthar in most cases?

The duration of treatment is highly individualized and is determined based on the patient’s condition and response. For acute events like MS flares, the official regimen is typically 2–3 weeks. For other conditions, it is generally administered as adjunctive therapy for short-term periods.

Q: If I am taking Acthar long-term, what kind of monitoring will my doctor need to do?

Official warnings note that prolonged use increases the risk of certain serious conditions. Therefore, long-term monitoring by a healthcare provider is recommended, which includes checking for signs of Cushing’s syndrome, changes in bone density (osteoporosis), and changes in eye health (e.g., cataracts and glaucoma).

Q: What are the signs of a serious side effect that require medical attention?

Official product information notes that patients should watch for signs of severe allergic reaction (such as difficulty breathing or swelling of the face), signs of infection (such as fever or chills), or signs of gastrointestinal bleeding/perforation (such as vomiting blood or severe abdominal pain). New or worsening symptoms are typically reported to a healthcare professional right away.

Q: Is there a risk of Acthar masking the symptoms of another developing illness?

Yes, regulatory documents indicate that Acthar may mask the typical signs and symptoms of other underlying diseases or disorders. This includes serious conditions like infections and gastrointestinal problems. Monitoring by a healthcare provider is necessary to watch for signs of these masked conditions.

Q: Can Acthar be taken at the same time as my other regular medications?

Providing a complete list of all medications, including prescription and nonprescription drugs, vitamins, and herbal supplements, to the healthcare provider is an important step. Specific drug classes that may require caution include diuretics, due to the risk of accentuating electrolyte loss.

Q: What types of over-the-counter pain relievers or supplements should be discussed with a doctor before starting Acthar?

It is important for patients to discuss all nonprescription medicines, including common over-the-counter pain relievers and herbal supplements, with their doctor. This is necessary because the official warnings note risks such as gastrointestinal bleeding and fluid/electrolyte imbalances, which may be affected by certain nonprescription products.

Q: What happens when a person needs to stop taking Acthar?

Official instructions state that treatment is typically discontinued slowly by gradually reducing the dose (tapering), especially after prolonged use. Sudden withdrawal carries the risk of adrenal insufficiency, where the body does not make enough cortisol, or a return of symptoms.

Q: Are patients required to get specialized training on how to use the injection?

While the official prescribing information details the method of injection, patient support programs often provide free injection training. This training helps prepare patients who need to self-inject Acthar Gel at home.

Q: How does the medication Acthar differ from a generic corticotropin injection?

Acthar is supplied as a repository corticotropin injection. This repository (gel) formulation is designed to release the active ingredient slowly after injection, providing a sustained, long-acting effect over an extended period.

Q: What does it mean for Acthar to be effective in 'speeding the resolution' of an MS flare but not affecting the 'ultimate outcome'?

Studies on Acthar for acute MS relapses focus on outcomes related to recovery speed from the acute attack. This indicates the drug can shorten the duration of the relapse, but it is not intended to change the long-term course of the disease or affect the patient's ultimate level of disability (the 'ultimate outcome').

Q: How does Acthar interact with medications that affect the immune system?

Official regulatory documents specifically contraindicate the use of live or live attenuated vaccines while receiving immunosuppressive doses of Acthar. Patients should discuss all immune-modulating drugs, including biologics and other therapies, with their healthcare provider.

Q: Can Acthar cause low potassium levels, and is that dangerous?

Official information lists low potassium (hypokalemia) as a possible adverse reaction related to fluid and electrolyte effects. Because low potassium can be serious, monitoring of the patient’s blood levels is standard during treatment, and potassium supplementation may be recommended to manage this effect.

How should Acthar be stored and disposed of?

How to Store and Dispose of Acthar

Official regulatory guidelines define specific requirements for the storage, handling, and disposal of Acthar (repository corticotropin injection).


Storage Conditions and Stability

Condition Requirement
Temperature Keep refrigerated between 36 F and 46 F (2 C to 8 C). Do not freeze.
Handling The gel may be stored at room temperature for a maximum of 24 hours. It must be warmed (e.g., by rolling) before injection.
Stability Multi-dose vials must be discarded 28 days after the first puncture, even if refrigerated.

Disposal and Safety Rules

Acthar must be stored out of the sight and reach of children to prevent accidental access. Used single-dose injectors, needles, and syringes must be placed immediately in an FDA-cleared sharps disposal container after use. Regulatory instructions prohibit throwing used sharps into household trash. Unused or expired medication should be disposed of via an official medication take-back program or as advised by a healthcare professional.

Attention! Always consult to a doctor or pharmacist before using pills or medicines.

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