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.
Brand Name(s): Emflaza
Drug Availability: Prescription
Therapeutic Classification: Endocrine-metabolic agentAvailable Generically: No
Controlled Substance: N/A
Administration Route: Oral
Active Ingredient: Deflazacort
Dosage Form(s): Tablet, oral suspension
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:
WheezingTightness in the chest or throatTrouble breathing, swallowing, or talkingUnusual hoarsenessSwelling 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 hivesRed, blistered, swollen, or peeling skin Red or irritated eyesSores 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.
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).
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.
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:
ConfusionSeizures TremorsWeakness on one side of the body
Precautions
What Are Reasons I Shouldn’t Take Emflaza?
Do not take Emflaza if you are:
If someone collapses or isn’t breathing after taking Emflaza, call 911 immediately.
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.
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.
Moon faceCommon cold symptomsIncreased hungerCoughAbdominal discomfortIrritabilityPassing a lot of urine
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.
