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.

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.

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.

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:

• Confusion
• Eye disorders
• Fatigue
• Increased thirst and hunger
• Passing a lot of urine
• Flushing
• Fast breathing
• Breath that smells like fruit

Symptoms of blood clots include:

• Numbness 
• Fast heartbeat
• Coughing up blood
• Tenderness, redness, pain, warmth, or swelling in the arms or legs
• Change in color of an arm or leg
• Chest pain
• Weakness on one side of the body
• Shortness of breath

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)

Dosage: How Much Emflaza Should I Take?

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

Precautions

What Are Reasons I Shouldn't Take Emflaza?

Do not take Emflaza if you are:

• Allergic to deflazacort, any part of its formulation or corticosteroids
• Experiencing herpes eye disease
• Pregnant: This drug can deform and cause serious harm to the fetus. It may cause low birth weight, slow baby growth in your womb, cleft lip or palate, adrenal gland issues.

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:

• Proleukin (aldesleukin) 
• DDAVP (desmopressin)
• Macrilen (macimorelin)
• Mifeprex (mifepristone)
• Tysabri (natalizumab)
• Elidel (pimecrolimus)
• IPOL (poliovirus vaccine) 
• Opzelura (ruxolitinib) 
• Protopic (tacrolimus)

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

• Dengue tetravalent vaccine
• M-M-R II (rubella vaccine)
• Varivax (varicella vaccine)
• Tice BCG (BCG vaccine)
• Vivotif (typhoid vaccine)
• YF-VAX (yellow fever vaccine)

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 45 (casimersen)
• Vyondys 53 (golodirsen)
• Medrol (methylprednisolone) 
• Prednisone/Prednisolone

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.

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:

• Plan routine eye exams.
• Closely watch sugar levels .
• Pay attention to weight loss or weight gain.
• Watch out for signs of infection.
• Monitor your child's growth.