Emflaza (Deflazacort) - Oral

What Is Emflaza?

Emflaza (deflazacort) is a prescription drug used to treat a rare genetic muscle-wasting disease called Duchenne muscular dystrophy (DMD). It is part of a drug class known as corticosteroids and can be used in people aged 2 years and older. This drug is mainly prescribed to male children because DMD primarily affects males and rarely females.

Emflaza works by reducing inflammation (swelling) and lowering the activity of the immune system. It is available as a tablet and also as an oral suspension.

Drug Facts

Generic Name: Deflazacort

Brand Name(s): Emflaza

Drug Availability: Prescription

Therapeutic Classification: Endocrine-metabolic agent
Available Generically: No

Controlled Substance: N/A

Administration Route: Oral

Active Ingredient: Deflazacort

Dosage Form(s): Tablet, oral suspension

What Is Emflaza Used For?

Emflaza treats Duchenne muscular dystrophy, a rare muscle-wasting disease. The Food and Drug Administration (FDA) approved it for use in people aged 2 years and older.

Emflaza (Deflazacort) Drug Information - Illustration by Dennis Madamba

Verywell / Dennis Madamba

How to Take Emflaza

Emflaza comes in either tablet or oral suspension (liquid) form.

For tablets, take the whole tablet, with or without food. You may crush the tablet and mix it with applesauce if directed by your healthcare provider. Once mixed with applesauce, take or give the medicine immediately.

For the oral suspension, take with or without food by mouth. Remember to:

  • Shake well before use.
  • Measure out the prescribed amount with a measuring cup.
  • Mix very well with 3 or 4 ounces of juice (but not grapefruit juice) or milk and take it immediately.

While taking this medication, do not:

  • Drink grapefruit juice. Grapefruit juice may increase the level of Emflaza in your body, leading to more side effects.
  • Get any live-attenuated or live vaccinations without speaking to your healthcare provider. It would be best if you got live vaccines at least four to six weeks before starting your medication.
  • Stop taking Emflaza without talking to your healthcare provider. Your medical provider needs to taper you off this medicine gradually.

Storage

The best temperature to store your medicine is at room temperature (around 77 degrees Fahrenheit). However, you may keep it during trips between cool and mildly hot temperatures (59–86 degrees F).

If you are on the oral suspension form of this drug, toss any unused liquid after one month of opening the bottle.

Throw away all unused or expired medicine. Do not toss it down the drain, sink, or toilet. Ask your pharmacist about the best ways to discard your medication. Check out take-back programs in your area.

How Long Does Emflaza Take to Work?

Emflaza takes between 15 minutes and two hours to peak in your body. However, taking this drug with a high-fat meal may slow it down by an hour.

What Are the Side Effects of Emflaza?

This is not a complete list of side effects, and others may occur. A medical professional can advise you on side effects. If you experience other effects, contact your pharmacist or a medical professional. You may report side effects to the FDA at www.fda.gov/medwatch or 800-FDA-1088.

Common Side Effects

Let your healthcare provider know if any of these side effects or symptoms do not go away or bother you. Common side effects of Emflaza include:

  • Cushingoid appearance (moon face)
  • Weight gain
  • Increased appetite
  • Cough
  • Frequent daytime urination
  • Upper respiratory infection
  • Hirsuitism (hair growth other than on the head)
  • Obesity around the middle
  • Irritability
  • Runny nose
  • Reddish skin

Severe Side Effects

Emflaza may cause severe and sometimes deadly side effects. Contact your provider right away or seek medical help if you have any of these:

Symptoms of high blood sugar include:

Symptoms of blood clots include:

Higher drug doses may increase the risk of blood clots.

Symptoms of Cushing syndrome (adrenal gland problems) include:

  • Severe headache
  • Weight gain in upper back or abdomen
  • Moon face
  • Slow healing
  • Mood changes
  • Severe nausea and vomiting
  • Muscle weakness
  • Severe dizziness and passing out
  • Lack of appetite 
  • Severe fatigue

Symptoms of electrolyte problems include:

  • Seizures
  • Mood changes
  • Confusion
  • Abnormal heartbeat
  • Lack of appetite
  • Severe nausea or vomiting
  • Muscle pain or weakness

Signs of an allergic reaction include:

  • Wheezing
  • Tightness in the chest or throat
  • Trouble breathing, swallowing, or talking
  • Unusual hoarseness
  • Swelling of the mouth, lips, face, tongue, or throat

Signs of mental change include:

  • Mood swings
  • Depression
  • Insomnia
  • Euphoria
  • Personality changes

Symptoms of Stevens-Johnson syndrome include:

  • Rash or hives
  • Red, blistered, swollen, or peeling skin 
  • Red or irritated eyes
  • Sores in the nose, mouth, throat, or eyes

Other severe side effects include:

  • Tremors
  • Sweating a lot
  • Bone pain
  • Joint pain
  • Infection
  • Vision changes
  • Severe abdominal pain
  • Black, tarry, or bloody stools
  • Decreased growth rate and bone growth in children

This drug may worsen myasthenia gravis, an autoimmune disorder, within the first two weeks of treatment. Some people may notice skin reactions within eight weeks of starting this medicine. Stop taking Emflaza at the first sign of a rash and get medical help immediately.

Long-Term Side Effects

Long-term use of corticosteroids, like Emflaza, may:

  • Cause, mask, prolong or worsen infections
  • Lead to Kaposi's sarcoma (cancer of the skin and mucous lining)
  • Cause cataracts, glaucoma, or eye nerve damage
  • Increase the risk of osteoporosis (bone weakness, loss, or fractures)

Report Side Effects

Emflaza may cause other side effects. Call your healthcare provider if you have any unusual problems while taking this medication.

If you experience a serious side effect, you or your provider may send a report to the FDA's MedWatch Adverse Event Reporting Program or by phone (800-332-1088).

Dosage: How Much Emflaza Should I Take?

Drug Content Provided and Reviewed by IBM Micromedex®

The dose of this medicine will be different for different patients. Follow your doctor's orders or the directions on the label. The following information includes only the average doses of this medicine. If your dose is different, do not change it unless your doctor tells you to do so.

The amount of medicine that you take depends on the strength of the medicine. Also, the number of doses you take each day, the time allowed between doses, and the length of time you take the medicine depend on the medical problem for which you are using the medicine.

  • For oral dosage form (suspension and tablets):
    • For treatment of Duchenne muscular dystrophy
      • Adults and children 2 years of age and older—Dose is based on body weight and must be determined by your doctor. The dose is usually 0.9 milligram (mg) per kilogram (kg) of body weight once a day.
      • Children younger than 2 years of age—Use and dose must be determined by your doctor.

Modification

If you or your child have trouble swallowing pills, you can crush the tablet and mix it into applesauce. Emflaza also comes as a liquid solution, making it easier to measure out doses and give to kids.

Missed Dose

Take the missed dose once you remember. If it is too close to your next dose, skip the missed dose. Go back to your regular dosing frequency. Do not take extra or double the quantity.

Overdose: What Happens If I Take Too Much Emflaza?

Overdosing on Emflaza may cause myopathy, a muscle disease. Overdose symptoms may include: 

  • Confusion
  • Seizures 
  • Tremors
  • Weakness on one side of the body

What Happens If I Overdose on Emflaza?

If you think you or someone else may have overdosed on Emflaza, call a healthcare provider or the Poison Control Center (800-222-1222).

If someone collapses or isn't breathing after taking Emflaza, call 911 immediately.

Precautions

Drug Content Provided and Reviewed by IBM Micromedex®

It is very important that your doctor check your or your child's progress at regular visits to make sure this medicine is working properly. Blood or urine tests may be needed to check for unwanted effects.

Using too much of this medicine or using it for a long time may increase your risk of having adrenal gland problems. Talk to your doctor right away if you have more than one of these symptoms while you are using this medicine: blurred vision, dizziness or fainting, a fast, irregular, or pounding heartbeat, increased thirst or urination, irritability, or unusual tiredness or weakness.

This medicine may cause you to get more infections than usual. Avoid people who are sick or have infections and wash your hands often. If you are exposed to chickenpox or measles, tell your doctor right away. If you start to have a fever, chills, sore throat, or any other sign of an infection, call your doctor right away.

While you are being treated with deflazacort, do not have any live or live-attenuated immunizations (vaccines) at least 4 to 6 weeks before starting this medicine without your doctor's approval. Deflazacort may lower your body's resistance and the vaccine may not work as well or you might get the infection the vaccine is meant to prevent. In addition, you should not be around other persons living in your household who receive live virus vaccines because there is a chance they could pass the virus on to you. Some examples of live vaccines include measles, mumps, influenza (nasal flu vaccine), poliovirus (oral form), rotavirus, and rubella. Do not get close to them and do not stay in the same room with them for very long. If you have questions about this, talk to your doctor.

If you use this medicine for a long time, do not suddenly stop using it without checking first with your doctor. You may need to slowly decrease your dose before stopping it completely.

Using this medicine may increase the risk of stomach or bowel perforation. It usually occurs in patients with stomach ulcers or other digestive problems. Tell your doctor right away if you have severe abdominal or stomach pain, cramping, or burning, bloody, black, or tarry stools, or vomiting of material that looks like coffee grounds, severe and continuing.

This medicine may cause changes in mood or behavior, including thoughts of suicide for some patients. Tell your doctor right away if you have depression, mood swings, a false or unusual sense of well-being, trouble with sleeping, thoughts of killing oneself, or personality changes while using this medicine.

This medicine may cause thinning of the bones (osteoporosis), loss of blood supply, or slow growth in children if used for a long time. Tell your doctor if you have any bone or joint pain or if you have an increased risk for osteoporosis. If your child is using this medicine, tell the doctor if you think your child is not growing properly.

Check with your doctor right away if blurred vision, difficulty in reading, eye pain, or any other change in vision occurs during or after treatment. Your doctor may want your eyes to be checked by an ophthalmologist (eye doctor).

Serious skin reactions can occur with this medicine. Check with your doctor right away if you have blistering, peeling, or loosening of the skin, red skin lesions, severe acne or skin rash, sores or ulcers on the skin, or fever or chills while you are using this medicine.

This medicine may increase your risk for cancer, including Kaposi's sarcoma. Tell your doctor right away if you have flat, painless spots that are red or purple on white skin and bluish, brownish, or black on dark skin.

This medicine may increase your risk of developing blood clots. Check with your doctor right away if you or your child have swelling and pain in your arms, legs, or stomach, chest pain, shortness of breath, loss of sensation, confusion, or problems with muscle control or speech.

This medicine may cause serious allergic reactions, including anaphylaxis, which can be life-threatening and requires immediate medical attention. Call your doctor right away if you have a rash, itching, trouble breathing, trouble swallowing, or any swelling of your hands, face, or mouth while you are using this medicine.

Do not take other medicines unless they have been discussed with your doctor. This includes prescription or nonprescription (over-the-counter [OTC]) medicines and herbal or vitamin supplements.

What Are Reasons I Shouldn't Take Emflaza?

Do not take Emflaza if you are:

The oral suspension form of this drug contains benzyl alcohol. Large amounts of benzyl alcohol can cause "gasping syndrome" and heart collapse in newborns. Avoid deflazacort with benzyl alcohol in newborns.

What Other Medications Interact With Emflaza?

Combining certain medicines with Emflaza may increase side effects or may lower how well either drug works.

Avoid taking Emflaza with:

Avoid receiving live vaccines while taking Emflaza. Live vaccines include:

Grapefruit juice can also affect how well this medication works. Grapefruit juice may increase the level of this drug in your body, leading to more side effects. Antacids can also affect how well this medication works in your body. Take antacids and Emflaza at least two hours apart to avoid drug interaction.

Talk to your healthcare provider before buying over-the-counter products to increase immunity. Some may not be good for you.  For example, Echinacea, the popular remedy for cold symptoms and boosting immunity, may reduce how well this medicine works.

What Medications Are Similar?

Other drugs that are corticosteroids or used to treat DMD include:

Amondys and Vyondys are injectable drugs belonging to a drug group called antisense oligonucleotide. They are used to treat DMD.

Medrol and prednisone are oral corticosteroids, like Emflaza. However, Emflaza is more effective but significantly more costly than prednisone/prednisolone.

Frequently Asked Questions

  • What is Emflaza used to treat?

    Emflaza is a drug used to treat a rare genetic muscle-wasting disease called Duchenne muscular dystrophy (DMD).

  • What should I do if I miss a dose of Emflaza?

    Take the missed dose as soon as you remember. If it is too close to your next dose, skip the missed dose. Return to your regular dosing frequency. Do not take more or double the quantity.

  • What are some side effects of Emflaza?

    Common side effects of Emflaza include:

    • Moon face
    • Common cold symptoms
    • Increased hunger
    • Cough
    • Abdominal discomfort
    • Irritability
    • Passing a lot of urine
  • How long does it take for Emflaza to work?

    It takes between 15 minutes and two hours for Emflaza to peak in your body. But, taking this drug with a high-fat meal may slow it down by an hour.

  • Does Emflaza interact with food?

    Emflaza interacts with grapefruit juice. It worsens the side effects of this drug. Avoid drinking grapefruit juice while you are on Emflaza.

  • Can I crush Emflaza tablets?

    Yes. If instructed by your healthcare provider, crush the tablet, mix in applesauce, and take immediately.

How Can I Stay Healthy While Taking Emflaza?

Living with DMD or watching your child experience this rare muscle disease can be distressing. Although DMD has no cure, you may still slow down its effects with Emflaza.

While taking or giving this drug daily, make sure that you:

Medical Disclaimer

Verywell Health's drug information is meant for educational purposes only and is not intended as a replacement for medical advice, diagnosis, or treatment from a healthcare professional. Consult your healthcare provider before taking any new medication(s). IBM Watson Micromedex provides some of the drug content, as indicated on the page.

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5 Sources
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  1. Food and Drug Administration. Emflaza label.

  2. McDonald CM, Sajeev G, Yao Z, et al. Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta-analysis of disease progression rates in recent multicenter clinical trials. Muscle Nerve. 2020;61(1):26-35. doi:10.1002/mus.26736

  3. Matthews E, Brassington R, Kuntzer T, et al. Corticosteroids for the treatment of Duchenne muscular dystrophy. Cochrane Database Syst Rev. 2016;(5):CD003725. doi:10.1002/14651858.CD003725.pub4

  4. Chrzanowski SM, Poudyal R. Deflazacort—New costs of an old medicine. JAMA Neurology. 2018;75(2):143-144. doi:10.1001/jamaneurol.2017.3704

  5. McDonald CM, Sajeev G, Yao Z, et al. Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta-analysis of disease progression rates in recent multicenter clinical trials. Muscle Nerve. 2020;61(1):26-35. doi:10.1002/mus.26736