Emflaza 6mg Tablets
It is used to treat Duchenne muscular dystrophy (DMD).
Drug Class
Glucocorticoid
Pharmacologic Class
Corticosteroid
Pregnancy Category
Not available
FDA Approved
Feb 2017
DEA Schedule
Not Controlled
Overview
What is this medicine?
Emflaza is a type of steroid medicine used to treat Duchenne muscular dystrophy (DMD). It works by reducing inflammation and slowing down muscle damage. It's important to take it exactly as prescribed and not to stop it suddenly, as this can cause serious problems.
How to Use This Medicine
Taking Your Medication Correctly
To get the most benefit from your medication, follow your doctor's instructions carefully. Read all the information provided with your prescription and follow the instructions closely. You can take this medication with or without food. Swallow the tablet whole with a full glass of water. If needed, the tablet can be crushed and mixed with applesauce. If you mix it with applesauce, take the dose immediately and do not store it for later use. Continue taking your medication as directed by your doctor or healthcare provider, even if you start to feel better.
Storing and Disposing of Your Medication
Store your medication at room temperature in a dry place, away from the bathroom. Keep all medications in a safe location, out of the reach of children and pets. Dispose of unused or expired medications properly. Do not flush them down the toilet or pour them down the drain unless instructed to do so. If you have questions about disposing of your medication, consult your pharmacist. You may also want to check if there are drug take-back programs in your area.
What to Do If You Miss a Dose
If you miss a dose, take it as soon as you remember. However, if it's close to the time for your next dose, skip the missed dose and resume your regular schedule. Do not take two doses at the same time or take extra doses to make up for the missed one.
To get the most benefit from your medication, follow your doctor's instructions carefully. Read all the information provided with your prescription and follow the instructions closely. You can take this medication with or without food. Swallow the tablet whole with a full glass of water. If needed, the tablet can be crushed and mixed with applesauce. If you mix it with applesauce, take the dose immediately and do not store it for later use. Continue taking your medication as directed by your doctor or healthcare provider, even if you start to feel better.
Storing and Disposing of Your Medication
Store your medication at room temperature in a dry place, away from the bathroom. Keep all medications in a safe location, out of the reach of children and pets. Dispose of unused or expired medications properly. Do not flush them down the toilet or pour them down the drain unless instructed to do so. If you have questions about disposing of your medication, consult your pharmacist. You may also want to check if there are drug take-back programs in your area.
What to Do If You Miss a Dose
If you miss a dose, take it as soon as you remember. However, if it's close to the time for your next dose, skip the missed dose and resume your regular schedule. Do not take two doses at the same time or take extra doses to make up for the missed one.
Lifestyle & Tips
- Do not stop taking Emflaza suddenly without talking to your doctor, as this can lead to serious withdrawal symptoms.
- Avoid exposure to people with infections (especially chickenpox or measles) as Emflaza weakens your immune system.
- Report any signs of infection (fever, chills, sore throat) immediately to your doctor.
- Follow a diet that is low in sodium and high in potassium to help manage potential side effects like fluid retention and electrolyte imbalance.
- Discuss calcium and vitamin D supplementation with your doctor to help protect bone health.
- Regular exercise, as advised by your doctor, can help maintain muscle strength and bone density.
- Carry a steroid card or medical alert identification indicating you are on corticosteroid therapy.
Available Forms & Alternatives
Available Strengths:
Generic Alternatives:
Dosing & Administration
Adult Dosing
Standard Dose:
0.9 mg/kg once daily
Dose Range:
0.9 - 1.5 mg
Condition-Specific Dosing:
Duchenne Muscular Dystrophy (DMD):
0.9 mg/kg once daily. Dosing is based on body weight and should be rounded to the nearest 0.1 mg/kg. Maximum daily dose typically up to 36 mg, though higher doses may be used based on clinical judgment.
Pediatric Dosing
Neonatal:
Not established
Infant:
Not established for patients under 2 years of age
Child:
0.9 mg/kg once daily for patients 2 years of age and older with Duchenne Muscular Dystrophy (DMD).
Adolescent:
0.9 mg/kg once daily for patients 2 years of age and older with Duchenne Muscular Dystrophy (DMD).
Dose Adjustments
Renal Impairment:
Mild:
No dose adjustment necessary
Moderate:
No dose adjustment necessary
Severe:
No specific dose adjustment recommended, but monitor for adverse effects.
Dialysis:
No specific recommendations; deflazacort and its active metabolite are not significantly removed by hemodialysis.
Hepatic Impairment:
Mild:
No dose adjustment necessary
Moderate:
No dose adjustment necessary
Severe:
Consider dose reduction; monitor for adverse effects. Specific recommendations are not provided in the label, but caution is advised due to hepatic metabolism.
Pharmacology
Mechanism of Action
Deflazacort is a prodrug that is converted to its active metabolite, 21-desacetyl deflazacort. This active metabolite is a corticosteroid that binds to glucocorticoid receptors, modulating gene expression. This leads to a wide range of anti-inflammatory and immunosuppressive effects, including inhibition of leukocyte infiltration at the site of inflammation, interference with mediators of inflammatory response, and suppression of humoral immune responses.
Pharmacokinetics
Absorption:
Bioavailability:
Not precisely quantified, but rapidly absorbed.
Tmax:
1.5-2 hours (for active metabolite)
FoodEffect:
Food slightly increases the extent of absorption (AUC) but is not considered clinically significant; can be taken with or without food.
Distribution:
Vd:
Approximately 1.3 L/kg (for active metabolite)
ProteinBinding:
Approximately 40% (for active metabolite)
CnssPenetration:
Limited
Elimination:
HalfLife:
1.7-2.7 hours (for active metabolite)
Clearance:
Not precisely quantified, but rapid.
ExcretionRoute:
Primarily renal (approximately 70%), with some fecal excretion (approximately 30%).
Unchanged:
Negligible
Pharmacodynamics
OnsetOfAction:
Variable, generally within hours to days for anti-inflammatory effects.
PeakEffect:
Variable, dependent on clinical outcome being measured.
DurationOfAction:
Variable, generally once daily dosing is sufficient due to sustained effects.
Safety & Warnings
Side Effects
Urgent Side Effects: Seek Medical Help Right Away
While rare, some people may experience severe and potentially life-threatening side effects when taking this medication. If you notice any of the following symptoms, contact your doctor immediately or seek emergency medical attention:
Signs of an allergic reaction: rash, hives, itching, red, swollen, blistered, or peeling skin (with or without fever), wheezing, tightness in the chest or throat, difficulty breathing, swallowing, or talking, unusual hoarseness, or swelling of the mouth, face, lips, tongue, or throat.
Signs of infection: fever, chills, severe sore throat, ear or sinus pain, cough, increased or discolored sputum, painful urination, mouth sores, or a wound that won't heal.
Signs of high blood sugar: confusion, drowsiness, excessive thirst or hunger, frequent urination, flushing, rapid breathing, or fruity-smelling breath.
Signs of Cushing's syndrome: weight gain in the upper back or abdomen, moon face, severe headache, or slow wound healing.
Signs of adrenal gland problems: severe nausea or vomiting, extreme dizziness or fainting, muscle weakness, fatigue, mood changes, decreased appetite, or weight loss.
Signs of high blood pressure: severe headache or dizziness, fainting, or changes in vision.
Signs of electrolyte imbalance: mood changes, confusion, muscle pain, cramps, or spasms, weakness, shakiness, balance problems, abnormal heartbeat, seizures, loss of appetite, or severe nausea or vomiting.
Shortness of breath, sudden weight gain, or swelling in the arms or legs.
Skin changes: acne, stretch marks, slow healing, or excessive hair growth.
Changes in vision.
Bone or joint pain.
Black, tarry, or bloody stools.
Vomiting blood or coffee ground-like material.
New or worsening mental, mood, or behavioral changes.
Purple, red, blue, brown, or black bumps or patches on the skin or in the mouth.
Signs of a blood clot: chest pain or pressure, coughing up blood, shortness of breath, swelling, warmth, numbness, color changes, or pain in a leg or arm, or difficulty speaking or swallowing.
A severe skin reaction (toxic epidermal necrolysis) may occur, causing serious health problems or even death. Seek immediate medical attention if you experience: red, swollen, blistered, or peeling skin (with or without fever), red or irritated eyes, or sores in your mouth, throat, nose, or eyes.
Other Possible Side Effects
Most people experience few or no side effects when taking this medication. However, if you notice any of the following side effects, contact your doctor if they bother you or persist:
Weight gain.
Increased appetite.
Common cold symptoms.
Nose and throat irritation.
Frequent urination.
Stomach pain.
Constipation.
Back pain.
Upset stomach.
* Headache.
This is not an exhaustive list of possible side effects. If you have questions or concerns about side effects, consult your doctor. You can also report side effects to the FDA at 1-800-332-1088 or online at https://www.fda.gov/medwatch.
While rare, some people may experience severe and potentially life-threatening side effects when taking this medication. If you notice any of the following symptoms, contact your doctor immediately or seek emergency medical attention:
Signs of an allergic reaction: rash, hives, itching, red, swollen, blistered, or peeling skin (with or without fever), wheezing, tightness in the chest or throat, difficulty breathing, swallowing, or talking, unusual hoarseness, or swelling of the mouth, face, lips, tongue, or throat.
Signs of infection: fever, chills, severe sore throat, ear or sinus pain, cough, increased or discolored sputum, painful urination, mouth sores, or a wound that won't heal.
Signs of high blood sugar: confusion, drowsiness, excessive thirst or hunger, frequent urination, flushing, rapid breathing, or fruity-smelling breath.
Signs of Cushing's syndrome: weight gain in the upper back or abdomen, moon face, severe headache, or slow wound healing.
Signs of adrenal gland problems: severe nausea or vomiting, extreme dizziness or fainting, muscle weakness, fatigue, mood changes, decreased appetite, or weight loss.
Signs of high blood pressure: severe headache or dizziness, fainting, or changes in vision.
Signs of electrolyte imbalance: mood changes, confusion, muscle pain, cramps, or spasms, weakness, shakiness, balance problems, abnormal heartbeat, seizures, loss of appetite, or severe nausea or vomiting.
Shortness of breath, sudden weight gain, or swelling in the arms or legs.
Skin changes: acne, stretch marks, slow healing, or excessive hair growth.
Changes in vision.
Bone or joint pain.
Black, tarry, or bloody stools.
Vomiting blood or coffee ground-like material.
New or worsening mental, mood, or behavioral changes.
Purple, red, blue, brown, or black bumps or patches on the skin or in the mouth.
Signs of a blood clot: chest pain or pressure, coughing up blood, shortness of breath, swelling, warmth, numbness, color changes, or pain in a leg or arm, or difficulty speaking or swallowing.
A severe skin reaction (toxic epidermal necrolysis) may occur, causing serious health problems or even death. Seek immediate medical attention if you experience: red, swollen, blistered, or peeling skin (with or without fever), red or irritated eyes, or sores in your mouth, throat, nose, or eyes.
Other Possible Side Effects
Most people experience few or no side effects when taking this medication. However, if you notice any of the following side effects, contact your doctor if they bother you or persist:
Weight gain.
Increased appetite.
Common cold symptoms.
Nose and throat irritation.
Frequent urination.
Stomach pain.
Constipation.
Back pain.
Upset stomach.
* Headache.
This is not an exhaustive list of possible side effects. If you have questions or concerns about side effects, consult your doctor. You can also report side effects to the FDA at 1-800-332-1088 or online at https://www.fda.gov/medwatch.
Seek Immediate Medical Attention If You Experience:
- Signs of infection (fever, chills, body aches, sore throat, cough, unusual fatigue, localized pain/redness)
- Severe abdominal pain, black or tarry stools, or vomiting blood (signs of GI bleeding)
- Increased thirst, frequent urination, or blurred vision (signs of high blood sugar)
- Swelling in your hands, ankles, or feet, or sudden weight gain (signs of fluid retention)
- Mood changes, depression, anxiety, or unusual behavior
- Muscle weakness or severe fatigue
- Vision changes, eye pain, or halos around lights
- Any new or worsening pain, especially bone pain
Before Using This Medicine
Before Taking This Medication: Important Information to Share with Your Doctor
It is essential to inform your doctor about the following:
Any allergies you have, including allergies to this medication, its components, or other substances, such as foods or drugs. Be sure to describe the allergic reaction you experienced, including any symptoms that occurred.
If you have an active infection, including bacterial, viral, or fungal infections, such as:
+ Amoeba infection (e.g., traveler's diarrhea)
+ Herpes infection of the eye
+ Malaria infection in the brain
+ Threadworm infestation
+ Any other type of infection
Certain health conditions, including:
+ Diverticulitis
+ Hole in the gastrointestinal (GI) tract
+ Stomach or bowel infection
+ Ulcers
Recent stomach or bowel surgery
* Any medications you are taking, including prescription and over-the-counter (OTC) drugs, natural products, and vitamins, as some may interact with this medication. Specifically, inform your doctor if you are taking:
+ Certain medications for HIV
+ Infection treatments
+ Seizure medications
+ Other medications that may interact with this drug
Please note that this is not an exhaustive list of all potential interactions. It is crucial to discuss all your medications and health conditions with your doctor and pharmacist to ensure safe use of this medication. Do not start, stop, or change the dose of any medication without first consulting your doctor.
It is essential to inform your doctor about the following:
Any allergies you have, including allergies to this medication, its components, or other substances, such as foods or drugs. Be sure to describe the allergic reaction you experienced, including any symptoms that occurred.
If you have an active infection, including bacterial, viral, or fungal infections, such as:
+ Amoeba infection (e.g., traveler's diarrhea)
+ Herpes infection of the eye
+ Malaria infection in the brain
+ Threadworm infestation
+ Any other type of infection
Certain health conditions, including:
+ Diverticulitis
+ Hole in the gastrointestinal (GI) tract
+ Stomach or bowel infection
+ Ulcers
Recent stomach or bowel surgery
* Any medications you are taking, including prescription and over-the-counter (OTC) drugs, natural products, and vitamins, as some may interact with this medication. Specifically, inform your doctor if you are taking:
+ Certain medications for HIV
+ Infection treatments
+ Seizure medications
+ Other medications that may interact with this drug
Please note that this is not an exhaustive list of all potential interactions. It is crucial to discuss all your medications and health conditions with your doctor and pharmacist to ensure safe use of this medication. Do not start, stop, or change the dose of any medication without first consulting your doctor.
Precautions & Cautions
Important Warnings and Cautions
It is essential to inform all your healthcare providers, including doctors, nurses, pharmacists, and dentists, that you are taking this medication.
Regular Health Checks
Your doctor will monitor your blood work regularly. Additionally, if you take this medication for an extended period, you may need to undergo eye pressure and bone density checks to assess potential long-term effects.
Potential Long-Term Risks
Prolonged use of this medication may increase the risk of developing cataracts or glaucoma. Discuss these potential risks with your doctor. Furthermore, long-term use may cause osteoporosis (weak bones). Your doctor can help determine if you are at a higher risk of developing osteoporosis and address any concerns you may have.
Blood Pressure and Sugar Monitoring
This medication may cause high blood pressure. Regular blood pressure checks are necessary, as advised by your doctor. If you have diabetes, it is crucial to closely monitor your blood sugar levels.
Dietary Considerations
You may need to reduce your salt intake and increase your potassium consumption. Consult your doctor for personalized dietary advice.
Grapefruit and Grapefruit Juice
Avoid consuming grapefruit and grapefruit juice while taking this medication.
Vaccinations
If you are not up to date with all recommended vaccines, discuss your vaccination status with your doctor. You may need to receive certain vaccines before starting treatment with this medication. Additionally, some vaccines may not be effective or may increase the risk of infection when taken with this medication. Consult your doctor before receiving any vaccines.
Infection Risks
Steroid medications like this one can increase the risk of infection, which can be severe or even life-threatening. Avoid close contact with individuals who have chickenpox or measles, especially if you have not had these illnesses before. If you have been exposed to chickenpox or measles, inform your doctor immediately.
General Infection Precautions
To minimize the risk of infection, wash your hands frequently, and avoid people with infections, colds, or flu. Notify your doctor if you experience any signs of infection.
Reactivation of Latent Infections
This medication may reactivate latent infections, such as tuberculosis or hepatitis B. Inform your doctor if you have a history of these infections.
Adrenal Insufficiency
This medication may decrease the natural production of steroids in your body. If you experience a fever, infection, surgery, or injury, consult your doctor, as you may require additional oral steroids to help your body cope with these stresses. Carry a warning card indicating that you may need extra steroids in certain situations.
Rare but Serious Risks
Long-term use of this medication may lead to rare but serious health problems, including changes in adrenal gland function and the development of a tumor on the adrenal gland (pheochromocytoma). Discuss these risks with your doctor.
Blood Clots
This medication may increase the risk of blood clots. Inform your doctor if you have a history of blood clots.
Kaposi's Sarcoma
Long-term use of this medication has been associated with an increased risk of Kaposi's sarcoma, a type of cancer. Discuss this risk with your doctor.
Stopping the Medication
Do not stop taking this medication abruptly without consulting your doctor, as this may increase the risk of side effects. If you need to stop taking this medication, your doctor will guide you on how to gradually discontinue it.
Missed Doses or Recent Discontinuation
If you miss a dose or have recently stopped taking this medication and experience symptoms such as fatigue, weakness, shakiness, fast heartbeat, confusion, sweating, or dizziness, inform your doctor.
Effects on Growth in Children and Teens
This medication may affect growth in children and teens. Regular growth checks may be necessary. Discuss this with your doctor.
Pregnancy and Breast-Feeding
If you are pregnant, plan to become pregnant, or are breast-feeding, consult your doctor to discuss the potential benefits and risks of this medication to you and your baby.
It is essential to inform all your healthcare providers, including doctors, nurses, pharmacists, and dentists, that you are taking this medication.
Regular Health Checks
Your doctor will monitor your blood work regularly. Additionally, if you take this medication for an extended period, you may need to undergo eye pressure and bone density checks to assess potential long-term effects.
Potential Long-Term Risks
Prolonged use of this medication may increase the risk of developing cataracts or glaucoma. Discuss these potential risks with your doctor. Furthermore, long-term use may cause osteoporosis (weak bones). Your doctor can help determine if you are at a higher risk of developing osteoporosis and address any concerns you may have.
Blood Pressure and Sugar Monitoring
This medication may cause high blood pressure. Regular blood pressure checks are necessary, as advised by your doctor. If you have diabetes, it is crucial to closely monitor your blood sugar levels.
Dietary Considerations
You may need to reduce your salt intake and increase your potassium consumption. Consult your doctor for personalized dietary advice.
Grapefruit and Grapefruit Juice
Avoid consuming grapefruit and grapefruit juice while taking this medication.
Vaccinations
If you are not up to date with all recommended vaccines, discuss your vaccination status with your doctor. You may need to receive certain vaccines before starting treatment with this medication. Additionally, some vaccines may not be effective or may increase the risk of infection when taken with this medication. Consult your doctor before receiving any vaccines.
Infection Risks
Steroid medications like this one can increase the risk of infection, which can be severe or even life-threatening. Avoid close contact with individuals who have chickenpox or measles, especially if you have not had these illnesses before. If you have been exposed to chickenpox or measles, inform your doctor immediately.
General Infection Precautions
To minimize the risk of infection, wash your hands frequently, and avoid people with infections, colds, or flu. Notify your doctor if you experience any signs of infection.
Reactivation of Latent Infections
This medication may reactivate latent infections, such as tuberculosis or hepatitis B. Inform your doctor if you have a history of these infections.
Adrenal Insufficiency
This medication may decrease the natural production of steroids in your body. If you experience a fever, infection, surgery, or injury, consult your doctor, as you may require additional oral steroids to help your body cope with these stresses. Carry a warning card indicating that you may need extra steroids in certain situations.
Rare but Serious Risks
Long-term use of this medication may lead to rare but serious health problems, including changes in adrenal gland function and the development of a tumor on the adrenal gland (pheochromocytoma). Discuss these risks with your doctor.
Blood Clots
This medication may increase the risk of blood clots. Inform your doctor if you have a history of blood clots.
Kaposi's Sarcoma
Long-term use of this medication has been associated with an increased risk of Kaposi's sarcoma, a type of cancer. Discuss this risk with your doctor.
Stopping the Medication
Do not stop taking this medication abruptly without consulting your doctor, as this may increase the risk of side effects. If you need to stop taking this medication, your doctor will guide you on how to gradually discontinue it.
Missed Doses or Recent Discontinuation
If you miss a dose or have recently stopped taking this medication and experience symptoms such as fatigue, weakness, shakiness, fast heartbeat, confusion, sweating, or dizziness, inform your doctor.
Effects on Growth in Children and Teens
This medication may affect growth in children and teens. Regular growth checks may be necessary. Discuss this with your doctor.
Pregnancy and Breast-Feeding
If you are pregnant, plan to become pregnant, or are breast-feeding, consult your doctor to discuss the potential benefits and risks of this medication to you and your baby.
Overdose Information
Overdose Symptoms:
- Acute overdose is rare but may include: increased blood pressure, fluid retention, hyperglycemia, and electrolyte imbalances. Chronic overdose can lead to Cushingoid features, adrenal suppression, and other long-term corticosteroid side effects.
What to Do:
Seek immediate medical attention. Call 911 or your local emergency number. For advice, call a poison control center at 1-800-222-1222.
Drug Interactions
Contraindicated Interactions
- Live or live attenuated vaccines (due to risk of disseminated infection)
Major Interactions
- Strong CYP3A4 inhibitors (e.g., ketoconazole, ritonavir, clarithromycin, grapefruit juice) - may increase deflazacort exposure and adverse effects.
- Strong CYP3A4 inducers (e.g., rifampin, phenytoin, carbamazepine, phenobarbital) - may decrease deflazacort exposure and efficacy.
- Non-steroidal anti-inflammatory drugs (NSAIDs) - increased risk of gastrointestinal ulceration and bleeding.
- Antidiabetic agents (e.g., insulin, oral hypoglycemics) - deflazacort may increase blood glucose, requiring dose adjustment of antidiabetics.
- Warfarin - corticosteroids may alter anticoagulant effects (monitor INR).
Moderate Interactions
- Diuretics (especially potassium-depleting diuretics) - increased risk of hypokalemia.
- Cardiac glycosides (e.g., digoxin) - increased risk of toxicity with hypokalemia.
- Anticholinesterases (e.g., neostigmine, pyridostigmine) - may cause severe weakness in myasthenia gravis patients.
- Oral contraceptives/estrogens - may increase corticosteroid effects.
Minor Interactions
- Not available
Monitoring
Baseline Monitoring
Blood Pressure
Rationale: Corticosteroids can cause hypertension.
Timing: Prior to initiation
Blood Glucose
Rationale: Corticosteroids can cause hyperglycemia and unmask latent diabetes.
Timing: Prior to initiation
Electrolytes (especially Potassium)
Rationale: Corticosteroids can cause electrolyte disturbances, including hypokalemia.
Timing: Prior to initiation
Bone Mineral Density (BMD)
Rationale: Long-term corticosteroid use increases risk of osteoporosis.
Timing: Prior to initiation (DEXA scan recommended)
Ophthalmologic Exam
Rationale: Long-term corticosteroid use increases risk of cataracts and glaucoma.
Timing: Prior to initiation
Growth (in pediatric patients)
Rationale: Corticosteroids can suppress growth.
Timing: Prior to initiation
Routine Monitoring
Blood Pressure
Frequency: Regularly (e.g., at each clinic visit)
Target: Age-appropriate normal range
Action Threshold: Sustained elevation requiring intervention
Blood Glucose (HbA1c or fasting glucose)
Frequency: Periodically (e.g., every 3-6 months or as clinically indicated)
Target: Normal range or individualized target for diabetic patients
Action Threshold: Persistent hyperglycemia requiring intervention
Electrolytes
Frequency: Periodically (e.g., every 3-6 months or as clinically indicated)
Target: Normal range
Action Threshold: Significant abnormalities (e.g., hypokalemia) requiring intervention
Bone Mineral Density (BMD)
Frequency: Every 1-2 years for long-term therapy
Target: T-score > -1.0 (adults), Z-score > -2.0 (children)
Action Threshold: Significant bone loss or fracture
Ophthalmologic Exam
Frequency: Annually for long-term therapy
Target: Normal intraocular pressure, no cataracts
Action Threshold: Signs of cataracts or glaucoma
Growth (height and weight)
Frequency: At each clinic visit for pediatric patients
Target: Normal growth velocity for age
Action Threshold: Significant growth deceleration
Signs of Infection
Frequency: Continuously
Target: Absence of fever, localized infection, or systemic symptoms
Action Threshold: Any signs of infection (fever, malaise, localized pain/redness)
Adrenal Function (HPA axis suppression)
Frequency: Consider if discontinuing therapy or during periods of stress
Target: Normal cortisol response
Action Threshold: Symptoms of adrenal insufficiency or abnormal cortisol levels
Symptom Monitoring
- Signs of infection (fever, chills, sore throat, cough, unusual fatigue, localized pain/redness)
- Hyperglycemia (increased thirst, frequent urination, increased hunger, blurred vision)
- Hypertension (headache, dizziness, blurred vision)
- Cushingoid features (moon face, buffalo hump, central obesity, striae, easy bruising)
- Mood and behavioral changes (irritability, anxiety, depression, insomnia, euphoria)
- Gastrointestinal symptoms (abdominal pain, black/tarry stools, vomiting blood)
- Muscle weakness or pain
- Bone pain or fractures
- Vision changes (blurred vision, eye pain)
- Swelling (edema)
Special Patient Groups
Pregnancy
Use during pregnancy should only be considered if the potential benefit justifies the potential risk to the fetus. Corticosteroids can cross the placenta and may be associated with adverse developmental outcomes.
Trimester-Specific Risks:
First Trimester:
Potential for increased risk of cleft palate (though data are inconsistent and overall risk is low).
Second Trimester:
Potential for fetal growth restriction and effects on fetal adrenal development.
Third Trimester:
Potential for fetal adrenal suppression, requiring monitoring of the neonate for signs of hypoadrenalism.
Lactation
Deflazacort and its active metabolite are excreted in human milk. The decision to discontinue breastfeeding or discontinue the drug should take into account the importance of the drug to the mother and the potential for adverse effects in the breastfed infant.
Infant Risk:
Low to moderate. Monitor breastfed infants for signs of adrenal suppression (e.g., poor weight gain, delayed development) or other corticosteroid effects.
Pediatric Use
Emflaza is approved for patients 2 years of age and older with Duchenne Muscular Dystrophy. Long-term corticosteroid use in children can cause growth suppression, delayed puberty, and bone demineralization. Close monitoring of growth, bone health, and development is essential.
Geriatric Use
Clinical studies did not include sufficient numbers of subjects aged 65 and over to determine whether they respond differently from younger subjects. In general, dose selection for an elderly patient should be cautious, reflecting the greater frequency of decreased hepatic, renal, or cardiac function, and of concomitant disease or other drug therapy. Elderly patients may be at increased risk for adverse effects such as osteoporosis, fluid retention, and hypertension.
Clinical Information
Clinical Pearls
- Emflaza is a prodrug; its active metabolite is 21-desacetyl deflazacort.
- Do not discontinue Emflaza abruptly due to the risk of adrenal insufficiency. Tapering is required.
- Patients on Emflaza are at increased risk of infection, including serious and fatal infections. Monitor closely for signs of infection.
- Monitor for hyperglycemia, hypertension, and bone health (osteoporosis) due to long-term corticosteroid effects.
- Growth suppression is a significant concern in pediatric patients; monitor height and weight regularly.
- Patients should carry a steroid card or medical alert identification.
- Vaccinations with live or live attenuated vaccines are contraindicated during Emflaza therapy.
Alternative Therapies
- Prednisone/Prednisolone (other corticosteroids used in DMD)
- Eteplirsen (Exondys 51 - for DMD patients amenable to exon 51 skipping)
- Golodirsen (Vyondys 53 - for DMD patients amenable to exon 53 skipping)
- Viltolarsen (Viltepso - for DMD patients amenable to exon 53 skipping)
- Casimersen (Amondys 45 - for DMD patients amenable to exon 45 skipping)
- Ataluren (Translarna - for nonsense mutation DMD, approved in some regions but not FDA approved in US)
- Gene therapies (e.g., Elevidys - for DMD, approved in US)
Cost & Coverage
Average Cost:
Highly variable, typically >$50,000 - $100,000+ per year
Generic Available:
Yes
Insurance Coverage:
Specialty Tier (requires prior authorization, often subject to high co-pays or deductibles)
General Drug Facts
If your symptoms or health issues persist or worsen, it's essential to contact your doctor for further guidance. To ensure your safety, never share your medication with others or take someone else's medication. Some medications may come with an additional patient information leaflet, so be sure to check with your pharmacist for more information. If you have any questions or concerns about your medication, don't hesitate to reach out to your doctor, nurse, pharmacist, or other healthcare provider. In the event of a suspected overdose, immediately call your local poison control center or seek medical attention. When seeking help, be prepared to provide details about the medication, including the amount taken and the time it happened, to ensure you receive the best possible care.