What is Emflaza?
Emflaza (deflazacort) is a corticosteroid medication used to treat a rare genetic disorder called Duchenne muscular dystrophy.
Duchenne muscular dystrophy is a severe form of muscular dystrophy, which is caused by an abnormal gene that results in low levels of a protein called dystrophin. Dystrophin helps to keep muscle cells intact. A lack of this protein leads to muscle weakness and wasting (atrophy). Other signs of Duchenne muscular dystrophy include chronic inflammation and activation of the immune system.
Emflaza acts as an anti-inflammatory and immunosuppresive agent. Emflaza is a pro-drug that is converted by the body into an active metabolite called 21-desDFZ. It is this active form of the drug that helps to decrease inflammation and suppress the immune system.
Emglaza was approved by the US Food and Drug Administration (FDA) in 2017.
What is Emflaza used for?
Emflaza is used for the treatment of Duchenne muscular dystrophy in patients 2 years of age and older
Emflaza can cause serious side effects including:
- Changes in endocrine function. Do not stop taking Emflaza, or change the amount you are taking, without first checking with your healthcare provider, as there may be a need for gradual dose reduction to decrease the risk of adrenal insufficiency and steroid “withdrawal syndrome”. Acute adrenal insufficiency can occur if corticosteroids are withdrawn abruptly, and can be fatal. A steroid “withdrawal syndrome,” seemingly unrelated to adrenocortical insufficiency, may also occur following abrupt discontinuance of corticosteroids. For patients already taking corticosteroids during times of stress, the dosage may need to be increased.
- An increased risk of infection. Tell the healthcare provider if the patient has had recent or ongoing infections or if they have recently received a vaccine. Medical advice should be sought immediately if the patient develops fever or other signs of infection. Patients and/or caregivers should be made aware that some infections can potentially be severe and fatal. Warn patients who are on corticosteroids to avoid exposure to chickenpox or measles and to alert their healthcare provider immediately if they are exposed.
- An increase in blood pressure and water retention. If this occurs, dietary salt restriction and potassium supplementation may be needed.
- An increased risk of developing a hole in the stomach or intestines in patients with certain stomach or intestine disorders.
- Severe behavioral and mood changes. Seek medical attention from the health care provider if any behavioral or mood changes develop.
- A risk of osteoporosis with prolonged use, which can lead to vertebral and long bone fractures.
- Cataracts or glaucoma. A health care provider should monitor for these conditions if corticosteroid therapy is continued for more than 6 weeks.
- Serious skin rashes. Seek medical attention at the first sign of a rash.
- Anaphylaxis. In rare instances anaphylaxis has occurred in patients receiving corticosteroid therapy, including Emflaza.
Immunizations should be up-to-date according to immunization guidelines prior to starting therapy with Emflaza. Live‑attenuated or live vaccines should be administered at least 4 to 6 weeks prior to starting this medication. Live‑attenuated or live vaccines should not be used in patients taking Emflaza.
Who should not take Emflaza?
Do not take Emflaza if you are allergic to deflazacort or any of the other ingredients in this medication. See below for a complete list of ingredients.
What should I tell my doctor before taking Emflaza?
Before you or your child take Emflaza, tell your healthcare provider about all of your or your child's medical conditions, including if you or your child:
- have an infection or a history of infections
- have high blood pressure
- have a stomach or intestinal disorder
- have osteoporosis or low bone density
- have cataracts
- have glaucoma
- have a condition affecting the thyroid, pituitary or adrenal glands
- have liver disease
- have kidney disease
Tell your healthcare provider if you or your child has received a live‑attenuated or live vaccines within the last 4 to 6 weeks or are due to receive one.
How should I take Emflaza?
- Take Emflaza tablets or oral suspension exactly as directed by your healthcare provider.
- Emflaza can be taken with or without food. Do not take with grapefruit juice.
- Do not stop taking Emflaza abruptly or without checking with your healthcare provider. You or your child may need to gradually reduce the dose rather than discontinue treatment altogether.
- The dosage of this medication must be decreased gradually if you or your child has taken it for more than a few days
- Weight gain should be assessed to determine whether the dose is appropriate
- You may need to adjust the dose because of weight gain, especially if using this medication for a growing child. Track bodyweight to help determine the appropriate dosage.
- Tablets may be given whole or crushed and mixed with applesauce. Take immediately after mixing with applesauce.
- You can combine multiple tablets to add up to the prescribed dose, rounding up to the nearest dose amount.
- Important information
- Only use the oral dispenser that comes in your carton when using this medicine.
- Take the oral suspension with juice or milk. Do not take with grapefruit juice.
- Throw away (discard) any unused oral suspension after 1 month of first opening the bottle.
- Gather the following supplies:
- 1 Emflaza oral suspension bottle
- 1 oral dispenser (2 oral dispensers are included in the carton. Use 1 oral dispenser to give (administer) the product. The other extra oral dispenser is included as a spare, if needed.
- 1 household cup filled with 3 to 4 ounces of juice or milk
- 1 spoon
- Prepare and give a dose of Emflaza oral suspension
- Step 1. Remove the oral suspension bottle and 1 of the oral dispensers from the carton.
- Step 2. Make sure the bottle cap is put on tightly and shake well before each use.
- Step 3. Remove the cap from the bottle by pushing down firmly on the cap and turning the cap in a counter-clockwise direction (to the left). Place the open bottle upright on a flat surface.
First time use of bottle only:
- Unwrap the bottle adapter.
- Place the ribbed end of the bottle adapter in the bottle top.
- Wrap both hands around the bottle.
- Press down on the bottle adapter using both thumbs.
- Push the ribbed end of the bottle adapter firmly into the neck of the bottle until the adapter top is even with the bottle top.
- Do not remove the bottle adapter from the bottle after it is inserted.
- Write the date that you first open the bottle on the bottle label.
- Step 4. Unwrap the oral dispenser if it is the first time you are using the oral dispenser.
Check your dose in milliliters (ml) as prescribed by your healthcare provider. Find this number on the barrel of the oral dispenser.
The oral dispenser only holds 1 ml of medicine at a time. If your dose is more than 1 ml, you will need to repeat Steps 5 through 8 using the same oral dispenser until your entire dose has been drawn from the bottle.
- Step 5. Push the plunger of the oral dispenser all the way down. Insert the oral dispenser into the bottle adapter.
- Step 6. With the oral dispenser in the bottle adapter, carefully turn the bottle upside down. Slowly pull back on the plunger until the widest part of the plunger is at the line marking on the oral dispenser of the number of ml needed for your dose. Do not use the narrow tip on the end of the plunger to measure the dose.
If you see air bubbles in the oral suspension, fully push in the plunger so the oral suspension flows back into the bottle. Then withdraw the dose of oral suspension that you need.
- Step 7. Leave the oral dispenser in the bottle adapter and turn the bottle to an upright position. Place the bottle onto a flat surface. Remove the oral dispenser from the bottle adapter by gently twisting the oral dispenser while pulling it straight up.
- Slowly push all the way down on the plunger of the oral dispenser to add the Emflaza oral suspension dose to a household cup filled with 3 to 4 ounces of juice or milk.
- Step 9. Stir the oral suspension and juice or milk with a spoon to mix well.
- Step 10. Immediately drink the juice or milk that is mixed with the oral suspension.
- After giving a dose of Emflaza
- Step 11. Replace the cap tightly on the bottle by turning the cap in a clockwise direction (to the right).
- Step 12. Wash the oral dispenser after each use. The oral dispenser should be taken apart by pulling back on the plunger and removing it from the barrel of the oral dispenser.
- Step 13. After the barrel and plunger are dry, put the oral dispenser back together by pushing the plunger back into the barrel.
What happens if I miss a dose?
If you miss a dose take it as soon as you remember. However, if it is almost time for your next dose, skip the missed dose and follow your regular dosing schedule. Do not take two doses at once.
What happens if I overdose?
If you take or give too much Emflaza call your doctor or your poison control center at 1-800-222-1222 right away or go to the nearest hospital emergency room.
The recommended dose of Emflaza is approximately 0.9 mg/kg/day administered orally once a day.
What are the side effects of Emflaza?
Emflaza can cause serious side effect. See 'Important Information" above.
The most common side effects of Emflaza include:
- facial puffiness or Cushingoid appearance
- weight increased
- increased appetite
- upper respiratory tract infection
- frequent daytime urination
- unwanted hair growth
- central obesity
These are not all of the possible side effects. Call your doctor for medical advice about side effects.
To report an adverse event, please call 1‑866‑562‑4620 or email at email@example.com. You may also report side effects to FDA at 1‑800‑FDA‑1088 or at www.fda.gov/medwatch.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines (such as insulin, aspirin or other NSAIDS), vitamins, and herbal supplements.
Especially tell your doctor if you take:
Alternate treatment, dosage adjustment, and/or special test(s) may be needed during the treatment.
The list above is not a complete list of medications that may interact with Emflaza.
Emflaza also interacts with grapefruit juice. Do not& take with grapefruit juice.
Pregnancy and breastfeeding
Tell your doctor if you are pregnant or planning to become pregnant. Emflaza can harm your unborn baby. It should only be used during pregnancy if the potential benefits justify the risk to the fetus. Use of corticosteroid like Emflaza during pregnancy may result in cleft lip with our without cleft palate, intrauterine growth restriction and decreased birth weight.
Tell your doctor if you are breastfeeding or plan to breastfeed. Emflaza may appear in breastmilk and could affect a nursing child. Talk to your doctor about the best way to feed your baby while taking this medication.
- Store Emflaza at room temperature between 68°F to 77°F (20°C to 25°C).
- Store the oral suspension bottle upright.
- Replace cap tightly on bottle and clean oral dispenser after each use.
- Throw away (discard) any unused oral suspension after 1 month of first opening the bottle.
Keep out of sight and reach of children.
What are the ingredients in Emflaza?
Active ingredient: deflazacort
Tablets: silicon dioxide, lactose monohydrate, magnesium stearate, pregelatinized corn starch.
Oral Suspension: acetic acid, aluminum magnesium silicate, benzyl alcohol, carboxymethylcellulose sodium, polysorbate 80, purified water, and sorbitol.
Emflaza is manufactured for PTC Therapeutics, Inc. South Plainfield, NJ 07080 U.S.A.
The cost for 30 Emflaza tablets ranges from $2250 to $13,000 and the cost for the oral suspension is about $4,600 for 13 mL. The daily dose is based on the patient’s weight. Overall cost will depend upon if you pay with cash, insurance, or use a manufacturer’s discount.
Emflaza oral suspension contains benzyl alcohol (10.45 mg per mL) which may cause seizures and other side effects in premature or low birth weight infants. The oral tablets do not contain benzyl alcohol. Emflaza is only approved for use in children 2 years of age and older.
Yes, Emflaza is the brand name product for deflazacort, a prescription corticosteroid ("steroid") medicine. Emflaza is manufactured by PTC Therapeutics and is approved to treat Duchenne muscular dystrophy (DMD) in patients 2 years of age and older. Deflazacort is not available as a generic option at this time.
Emflaza (deflazacort) is a glucocorticoid (a class of corticosteroid drugs) for the treatment of Duchenne Muscular Dystrophy (DMD) in patients age 2 years of age and older. Corticosteroids in general help to reduce inflammation (swelling) and pain. The Emflaza corticosteroid was developed slightly differently and may be more tolerable than some other corticosteroids. Continue reading
The new drugs approved by the FDA for the treatment of Duchenne Muscular Dystrophy (DMD) are Amondys 45 (casimersen), Viltepso (viltolarsen), Vyondys 53 (golodirsen), Exondys 51 (eteplirsen), Elevidys (delandistrogene moxeparvovec), and Emflaza (deflazacort). They include antisense oligonucleotides, a glucocorticoid and a gene therapy.
Duchenne muscular dystrophy (DMD) is a genetic disease that causes progressive weakness, loss of motor function and damage to skeletal and heart muscles. DMD, which has no cure, primarily affects males starting in early childhood, usually between 2 and 3 years of age. Continue reading
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