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Medical Disclaimer: This information is for educational purposes only and is not a substitute for professional medical advice.
Corticosteroid [EPC]
Deflazacort is a corticosteroid medication primarily used to treat Duchenne muscular dystrophy (DMD). It works by reducing inflammation and modifying the body's immune response to preserve muscle function.
Name
Deflazacort
Raw Name
DEFLAZACORT
Category
Corticosteroid [EPC]
Drug Count
8
Variant Count
43
Last Verified
February 17, 2026
RxCUI
1870964, 153098, 153880, 1870950, 1870952, 1870954, 1870956, 1870958, 1870960, 1870968, 2716704, 2716706, 2716708, 2716710, 2725491, 2725493, 2725495, 2725497, 2719198, 2727381
UNII
KR5YZ6AE4B
About Deflazacort
Deflazacort is a corticosteroid medication primarily used to treat Duchenne muscular dystrophy (DMD). It works by reducing inflammation and modifying the body's immune response to preserve muscle function.
Detailed information about Deflazacort
This page is for informational purposes only and does not replace medical advice. Consult a qualified healthcare professional before using any medication containing Deflazacort.
Deflazacort belongs to a class of drugs known as corticosteroids, which are synthetic versions of hormones naturally produced by the adrenal glands. While many corticosteroids are used for a wide range of inflammatory conditions—such as asthma, rheumatoid arthritis, and severe allergies—deflazacort's primary clinical utility in the modern era is focused on DMD. This condition is a rare, progressive genetic disorder characterized by muscle degeneration and weakness due to the absence of dystrophin, a protein that helps keep muscle cells intact. By modulating the immune system and reducing the chronic inflammation associated with muscle fiber breakdown, deflazacort helps slow the progression of muscle weakness and may prolong the period during which a patient can walk independently. It is important to note that only your healthcare provider can determine if deflazacort is the appropriate therapeutic choice for your specific medical situation.
The mechanism of action for deflazacort is complex and occurs at the molecular level within the cell's nucleus. As a glucocorticoid receptor agonist, deflazacort (specifically its active metabolite, 21-desacetyl-deflazacort) crosses the cell membrane and binds with high affinity to glucocorticoid receptors (GR) located in the cytoplasm. Once the drug-receptor complex is formed, it translocates (moves) into the cell nucleus.
Inside the nucleus, the complex binds to specific DNA sequences known as glucocorticoid response elements (GREs). This binding leads to two primary actions: transactivation and transrepression. Transactivation involves the induction of anti-inflammatory proteins, such as lipocortin-1, which inhibits the enzyme phospholipase A2. This inhibition prevents the release of arachidonic acid, the precursor to inflammatory mediators like prostaglandins and leukotrienes. Transrepression, on the other hand, involves the inhibition of pro-inflammatory transcription factors, such as nuclear factor-kappa B (NF-κB) and activator protein-1 (AP-1). By suppressing these factors, deflazacort reduces the production of pro-inflammatory cytokines, including interleukin-1 (IL-1), interleukin-6 (IL-6), and tumor necrosis factor-alpha (TNF-α). In the context of Duchenne muscular dystrophy, this reduction in chronic inflammation is believed to decrease the rate of muscle cell death (necrosis) and promote a more stable environment for the remaining muscle fibers.
Understanding how the body processes deflazacort is essential for optimizing therapy. The pharmacokinetic profile of this drug is characterized by rapid conversion and high bioavailability.
Deflazacort is FDA-approved for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older. While its primary indication in the U.S. is DMD, in other parts of the world, deflazacort has been used for decades to treat a variety of other conditions due to its anti-inflammatory properties. These off-label or international uses include:
Despite these potential uses, in the United States, it is specifically marketed and utilized for its unique benefit-to-risk profile in DMD patients, where it is often preferred over other steroids like prednisone because some studies suggest it may cause less weight gain or have a different impact on bone density, though individual results vary.
Deflazacort is available in two primary oral dosage forms to accommodate different patient needs and ages:
> Important: Only your healthcare provider can determine if Deflazacort is right for your specific condition. This medication requires careful monitoring by a specialist, typically a neurologist or a physician experienced in managing neuromuscular disorders.
While Duchenne muscular dystrophy (DMD) is primarily a pediatric-onset condition, some patients continue treatment into adulthood. The standard adult dosage for deflazacort is weight-based, typically calculated at 0.9 mg/kg of body weight taken once daily. For an adult weighing 70 kg, this would equate to approximately 63 mg per day, which may be rounded to the nearest available tablet strength (e.g., 60 mg or 66 mg) as directed by a healthcare provider. Because corticosteroids can cause insomnia or increased energy, it is generally recommended to take the dose in the morning.
Deflazacort is approved for use in pediatric patients aged 2 years and older for the treatment of DMD. The dosage is strictly weight-based to ensure efficacy while minimizing the risk of growth suppression and other side effects.
No specific dosage adjustments are provided in the manufacturer's labeling for patients with renal (kidney) impairment. The metabolites are excreted renally, but because the drug is typically used in a population with normal renal function, specific guidelines for dialysis or severe renal failure are not well-established. Your doctor will monitor your kidney function periodically.
Deflazacort is converted to its active metabolite by esterases, which are present throughout the body, not just in the liver. However, the active metabolite is further processed by the liver (CYP3A4). In patients with mild to moderate hepatic (liver) impairment, no dosage adjustment is generally required. There is limited data regarding the use of deflazacort in patients with severe hepatic impairment, and such use should be approached with extreme caution.
Deflazacort is indicated for DMD, a condition that rarely affects the geriatric population. Consequently, there is no established data for dosage adjustments in elderly patients. If used for other inflammatory conditions in the elderly, doctors would typically start at the lowest possible dose due to the increased risk of osteoporosis, hypertension, and diabetes in this age group.
If a dose of deflazacort is missed, it should be taken as soon as the patient remembers. However, if it is almost time for the next scheduled dose, the missed dose should be skipped. Do not take two doses at once to make up for a missed one. Frequent missed doses can reduce the effectiveness of the treatment and may lead to withdrawal symptoms.
An acute overdose of deflazacort is unlikely to be life-threatening, but chronic overdosage can lead to severe systemic corticosteroid effects (Cushing's syndrome). Signs of acute overdose may include severe gastric upset or exaggerated side effects. In the event of a suspected overdose, contact a poison control center (1-800-222-1222 in the U.S.) or seek emergency medical attention immediately. Treatment is generally supportive, focusing on managing symptoms.
> Important: Follow your healthcare provider's dosing instructions exactly. Do not adjust your dose or stop taking this medication without medical guidance, as sudden discontinuation can be dangerous.
Corticosteroids like deflazacort affect almost every organ system in the body. The most frequently reported side effects in clinical trials for Duchenne muscular dystrophy include:
> Warning: Stop taking Deflazacort and call your doctor immediately if you experience any of these serious symptoms.
Prolonged use of deflazacort (months to years) carries significant risks that require proactive management:
No FDA black box warnings currently exist for Deflazacort. However, the absence of a black box warning does not imply the drug is without risk. The warnings regarding HPA axis suppression and infection risk are considered "Major Warnings" in the prescribing information and carry similar weight in clinical practice.
Report any unusual symptoms to your healthcare provider immediately. Regular monitoring of weight, height, blood pressure, and bone density is standard of care during deflazacort therapy.
Deflazacort is a high-alert medication because of its systemic effects on the endocrine and immune systems. It must be managed by a healthcare provider who is familiar with the long-term consequences of glucocorticoid therapy. Patients and caregivers must be educated on the risks of sudden discontinuation and the necessity of "stress dosing" during periods of physical trauma or illness.
There are currently no FDA-mandated black box warnings for deflazacort.
To ensure safety, healthcare providers will typically require the following tests:
Deflazacort generally does not interfere with the ability to drive or operate machinery. However, if a patient experiences side effects like dizziness, blurred vision, or severe mood changes, they should avoid these activities until they know how the medication affects them.
There is no direct chemical interaction between deflazacort and alcohol. However, both alcohol and corticosteroids can irritate the stomach lining, increasing the risk of gastric ulcers and bleeding. Furthermore, alcohol can contribute to weight gain and blood sugar fluctuations, which are already concerns with deflazacort. It is best to limit or avoid alcohol while on this medication.
Never stop taking deflazacort suddenly. If you have taken the drug for more than a few weeks, your adrenal glands have "gone to sleep." Stopping the drug abruptly can cause an adrenal crisis, characterized by vomiting, low blood sugar, dehydration, and even death. Your doctor will provide a tapering schedule to slowly reduce the dose over weeks or months, allowing your adrenal glands to resume normal function.
> Important: Discuss all your medical conditions, including any history of infections, heart problems, or mental health issues, with your healthcare provider before starting Deflazacort.
Deflazacort can interfere with several diagnostic tests:
> Important: Tell your doctor about ALL medications, supplements, and herbal products you are taking. Keep an up-to-date list to share with every healthcare provider you visit.
There are very few situations where deflazacort is absolutely forbidden, but they are critical for patient safety:
In these conditions, the benefits of deflazacort must be carefully weighed against the significant risks:
Patients who have had a severe allergic reaction to other corticosteroids (such as prednisone, prednisolone, or dexamethasone) may also be sensitive to deflazacort. While the chemical structures differ slightly, the risk of cross-reactivity exists and should be discussed with an allergist or the prescribing physician.
> Important: Your healthcare provider will evaluate your complete medical history, including any past infections or chronic conditions, before prescribing Deflazacort to ensure the benefits outweigh the potential risks.
Deflazacort is classified by the FDA as a drug that should only be used during pregnancy if the potential benefit justifies the potential risk to the fetus. There are no adequate and well-controlled studies in pregnant women. However, data from other corticosteroids suggest:
It is known that systemically administered corticosteroids appear in human milk and could suppress growth, interfere with endogenous corticosteroid production, or cause other untoward effects in the nursing infant. Because of the potential for serious adverse reactions in nursing infants from deflazacort, a decision should be made whether to discontinue nursing or to discontinue the drug, taking into account the importance of the drug to the mother.
Deflazacort is specifically indicated and approved for the treatment of Duchenne muscular dystrophy in patients 2 years of age and older.
Duchenne muscular dystrophy is a disease of childhood and young adulthood; therefore, deflazacort has not been extensively studied in patients over the age of 65. If used in older adults for other conditions, the risk of side effects such as osteoporosis, diabetes, and hypertension is significantly higher. Clinical studies of other corticosteroids suggest that the dose should be started at the low end of the range, reflecting the greater frequency of decreased hepatic, renal, or cardiac function.
No formal studies have been conducted in patients with renal impairment. However, since the active metabolite is primarily eliminated through the kidneys, patients with severe renal disease should be monitored closely for signs of steroid toxicity. Dosage adjustments are not standard but may be necessary based on clinical response.
Deflazacort is a prodrug converted by esterases (found in many tissues) to its active form. The active form is then metabolized by the liver. In patients with mild to moderate hepatic impairment (Child-Pugh Class A or B), no dose adjustment is required. There is no data for patients with severe hepatic impairment (Child-Pugh Class C), and use in this population is not recommended unless the benefits clearly outweigh the risks.
> Important: Special populations require individualized medical assessment and more frequent monitoring to ensure safety and efficacy.
Deflazacort is a glucocorticoid prodrug. Its therapeutic effects are mediated through its active metabolite, 21-desacetyl-deflazacort. This metabolite acts as a potent agonist at the glucocorticoid receptor. Once bound, the receptor-ligand complex moves into the nucleus and binds to DNA. This results in the inhibition of inflammatory genes (transrepression) and the activation of anti-inflammatory genes (transactivation). Specifically, it inhibits the production of prostaglandins and leukotrienes by inducing lipocortins, and it suppresses the release of various cytokines (IL-1, IL-6, TNF-alpha) that drive the chronic inflammation and muscle fiber damage seen in Duchenne muscular dystrophy.
The pharmacodynamic effects of deflazacort include a reduction in the number and activity of circulating lymphocytes, eosinophils, and monocytes. It also stabilizes lysosomal membranes and decreases capillary permeability. In patients with DMD, these actions translate to a slower rate of muscle strength loss. The onset of anti-inflammatory action is typically within 1-2 hours of administration, but the clinical benefits in DMD (such as improved motor function) may take several weeks or months of consistent therapy to become apparent.
| Parameter | Value |
|---|---|
| Bioavailability | 70% to 100% (as active metabolite) |
| Protein Binding | ~40% (Active metabolite) |
| Half-life | 1.1 to 1.9 hours (Active metabolite) |
| Tmax | 1 to 2 hours |
| Metabolism | Esterases (prodrug to active); CYP3A4 (active to inactive) |
| Excretion | Renal 70%, Fecal 30% |
Deflazacort is classified as a systemic corticosteroid, specifically a glucocorticoid. It belongs to the same therapeutic class as prednisone, prednisolone, and dexamethasone. Within the context of Duchenne muscular dystrophy, it is considered a disease-modifying therapy aimed at preserving muscle function and delaying the loss of ambulation.
Medications containing this ingredient
Common questions about Deflazacort
Deflazacort is primarily used for the treatment of Duchenne muscular dystrophy (DMD) in patients who are at least 2 years old. DMD is a rare genetic disorder that causes progressive muscle weakness and loss of muscle mass over time. Deflazacort works by reducing the chronic inflammation in the muscle tissue, which helps to slow down the breakdown of muscle fibers. By preserving muscle strength for a longer period, it can help patients maintain their ability to walk and perform daily activities. While it is not a cure for DMD, it is a standard-of-care treatment recommended by specialists worldwide. It is important to use this medication only under the strict supervision of a doctor experienced in neuromuscular diseases.
The most common side effects associated with deflazacort include weight gain, an increased appetite, and a rounded appearance of the face known as 'moon face' or cushingoid features. Many patients also experience an increased frequency of urination and a higher susceptibility to upper respiratory tract infections like the common cold. In children, it can lead to irritability, mood swings, and unwanted hair growth (hirsutism). Some patients may also notice skin changes, such as thinning or easy bruising. Because it is a steroid, it can also cause a temporary increase in blood sugar levels. Most of these side effects are dose-dependent and can be managed with the help of your healthcare provider.
It is generally advised to avoid or significantly limit alcohol consumption while taking deflazacort. Both alcohol and corticosteroids can irritate the lining of the stomach and intestines, and taking them together significantly increases the risk of developing stomach ulcers or gastrointestinal bleeding. Additionally, deflazacort can cause weight gain and affect blood sugar levels, and alcohol can worsen these metabolic issues. Alcohol may also interfere with the quality of sleep and mood, which can already be affected by steroid therapy. If you choose to consume alcohol, you must discuss it with your doctor to understand your specific risks. Always prioritize the safety of your stomach and metabolic health while on chronic steroid therapy.
Deflazacort should only be used during pregnancy if the potential benefit to the mother outweighs the potential risk to the developing fetus. There is limited data on the use of deflazacort specifically in pregnant women, but general knowledge of corticosteroids suggests they may increase the risk of cleft palate or low birth weight when used in the first trimester. If used later in pregnancy, there is a risk that the baby may be born with adrenal insufficiency, requiring medical monitoring. It is essential to inform your doctor if you are pregnant, planning to become pregnant, or breastfeeding before starting this medication. Your healthcare provider will help you weigh the risks of the medication against the risks of leaving the underlying condition untreated.
The time it takes for deflazacort to show results can vary depending on the goal of treatment. While the anti-inflammatory effects begin within hours of taking the first dose, the visible clinical benefits for Duchenne muscular dystrophy typically take longer to manifest. In clinical trials, improvements in muscle strength and motor function tests were usually measured after several months of consistent daily use. It is not a medication that provides immediate relief of symptoms like a painkiller would. Instead, it works gradually to stabilize muscle health over the long term. Patients and caregivers should remain patient and continue the medication as prescribed, even if improvements are not immediately obvious.
No, you must never stop taking deflazacort suddenly if you have been using it for more than a few days. When you take a corticosteroid like deflazacort, your body's natural production of cortisol (the 'stress hormone') slows down or stops entirely. If you stop the medication abruptly, your body will not have enough cortisol to function properly, leading to a dangerous condition called adrenal crisis. Symptoms of an adrenal crisis include severe weakness, dizziness, nausea, vomiting, and dangerously low blood pressure. To stop the medication safely, your doctor will provide a tapering schedule to gradually reduce the dose over several weeks or months. This allows your adrenal glands time to wake up and start producing cortisol naturally again.
If you miss a dose of deflazacort, you should take it as soon as you remember on that same day. However, if it is already very close to the time for your next scheduled dose, you should skip the missed dose and simply take your next dose at the regular time. Do not take two doses at once to make up for the one you missed, as this can increase the risk of side effects. Consistency is very important with steroid therapy to keep the drug levels steady in your body. If you find yourself missing doses frequently, talk to your doctor or pharmacist about using a pillbox or a reminder app. Keeping a consistent schedule helps ensure the medication works as effectively as possible for your condition.
Yes, weight gain is one of the most common and significant side effects of deflazacort. This occurs because the medication increases appetite and changes how the body stores and distributes fat. Patients often notice increased fat in the abdominal area and the face. While deflazacort is sometimes reported to cause slightly less weight gain than other steroids like prednisone, it is still a major concern for most patients. Managing weight while on deflazacort usually requires a proactive approach, including a balanced, low-calorie diet and regular physical activity as tolerated. Working with a nutritionist or dietitian who understands corticosteroid therapy can be very helpful for patients and families.
Deflazacort can interact with many other medications, so it is vital to provide your doctor with a complete list of everything you take. Certain drugs, like the antibiotic rifampin or anti-seizure medications like phenytoin, can make deflazacort less effective. Other drugs, such as certain antifungals or the antibiotic clarithromycin, can increase the levels of deflazacort in your blood, leading to more side effects. You should also be careful with over-the-counter NSAIDs like ibuprofen, which increase the risk of stomach ulcers when combined with steroids. Always check with your pharmacist before starting any new medication, including herbal supplements like St. John's Wort. Your doctor will monitor you closely for any signs of interactions.
As of 2024, deflazacort is available in the United States primarily as the brand-name drug Emflaza. While generic versions exist in other countries (such as the UK, India, and parts of Europe) where the drug has been used for many years, the regulatory pathway in the U.S. has maintained brand exclusivity for a period. However, generic availability can change as patents expire and new manufacturers receive FDA approval. You should check with your insurance provider or pharmacist for the most current information regarding the availability of a generic version. If the brand-name version is too expensive, your doctor or the manufacturer may have patient assistance programs to help cover the cost.