Deflazacort

What is Deflazacort?

• Deflazacort (Emflaza) is a corticosteroid used treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older.

What are the uses of this medicine?

• This medicine is used for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older.

How does this medicine work?

• Deflazacort is a corticosteroid prodrug, whose active metabolite, 21-desDFZ, acts through the glucocorticoid receptor to exert anti-inflammatory and immunosuppressive effects.
• The precise mechanism by which deflazacort exerts its therapeutic effects in patients with DMD is unknown.

Who Should Not Use this medicine ?

This medicine cannot be used in patients with:

• known hypersensitivity to deflazacort or to any of the inactive ingredients.

What drug interactions can this medicine cause?

• Co-administration of deflazacort with clarithromycin, a strong CYP3A4 inhibitor, increased total exposure to 21-desDFZ by about 3-fold. Give one third the recommended dosage of Emflaza when moderate or strong CYP3A4 inhibitors (e.g., clarithromycin, fluconazole, diltiazem, verapamil, grapefruit juice) are used concomitantly with Emflaza.
• Avoid concomitant use of strong (e.g., efavirenz) or moderate (e.g., carbamazepine, phenytoin) CYP3A4 inducers with Emflaza.
• Patients receiving corticosteroids, including Emflaza, and concomitant therapy with neuromuscular blocking drugs (e.g., pancuronium) may be at increased risk of developing an acute myopathy.

Is this medicine FDA approved?

• It was approved for use in the United States in 2017.

How should this medicine be used?

• Administer all immunizations according to immunization guidelines prior to starting Emflaza.
• Administer live-attenuated or live vaccines at least 4 to 6 weeks prior to starting Emflaza.

Recommended Dosageː

• The recommended oral dosage of Emflaza is approximately 0.9 mg/kg/day once daily.
• Dosage of Emflaza must be decreased gradually if the drug has been administered for more than a few days.

Dosage Modification for Use with CYP3A4 Inhibitors and Inducers CYP3A4 Inhibitorsː

• Give one third of the recommended dosage when Emflaza is administered with moderate or strong CYP3A4 inhibitors.

CYP3A4 Inducersː

• Avoid use with moderate or strong CYP3A4 inducers with Emflaza

Administration

• Emflaza Tablets and Oral Suspension can be taken with or without food. Do not administer Emflaza with grapefruit juice.

Emflaza Tabletsː

• Emflaza Tablets can be administered whole or crushed and taken immediately after mixing with applesauce.

Emflaza Oral Suspensionː

• Shake Emflaza Oral Suspension well before administration.
• Use only the oral dispenser provided with the product. After withdrawing the appropriate dose into the oral dispenser, slowly add the Emflaza Oral Suspension into 3 to 4 ounces of juice (except grapefruit juice) or milk and mix well. The dose should then be administered immediately.
• Discard any unused Emflaza Oral Suspension remaining after 1 month of first opening the bottle.

What are the dosage forms and brand names of this medicine?

This medicine is available in fallowing doasage form:

• As Tablets: 6 mg, 18 mg, 30 mg, and 36 mg
• Oral Suspension: 22.75 mg/mL

This medicine is available in fallowing brand namesː

• Emflaza

What side effects can this medication cause?

The most common side effects of this medicine include:

• Cushingoid appearance
• weight increased
• increased appetite
• upper respiratory tract infection
• cough, pollakiuria
• hirsutism
• central obesity
• nasopharyngitis

What special precautions should I follow?

• Corticosteroids, such as Emflaza, can cause serious and life-threatening alterations in endocrine function, especially with chronic use. Monitor patients receiving Emflaza for Cushing’s syndrome, hyperglycemia, and adrenal insufficiency after Emflaza withdrawal.
• Corticosteroids, including Emflaza, suppress the immune system and increase the risk of infection with any pathogen, including viral, bacterial, fungal, protozoan, or helminthic. Increased risk of new, exacerbation, dissemination, or reactivation of latent infections, which can be severe and at times fatal; Signs and symptoms of infection may be masked.
• Corticosteroids, including Emflaza, can cause elevation of blood pressure, salt, and water retention, and increased excretion of potassium and calcium. Monitor for elevated blood pressure and sodium, and for decreased potassium levels.
• There is an increased risk of gastrointestinal perforation during corticosteroid use in patients with certain gastrointestinal disorders such as active or latent peptic ulcers, diverticulitis, fresh intestinal anastomoses, and non-specific ulcerative colitis.
• Potentially severe psychiatric adverse reactions may occur with systemic corticosteroids, including Emflaza. May include euphoria, insomnia, mood swings, personality changes, severe depression, and psychosis.
• Corticosteroids, including Emflaza, decrease bone formation and increase bone resorption both through their effect on calcium regulation (i.e., decreasing absorption and increasing excretion) and inhibition of osteoblast function. Monitor for decreases in bone mineral density with chronic use of Emflaza.
• Use of corticosteroids, including Emflaza, may produce posterior subcapsular cataracts. Corticosteroids may also cause glaucoma with possible damage to the optic nerves, and may increase the risk of secondary ocular infections caused by bacteria, fungi, or viruses.
• Do not administer live or live attenuated vaccines to patients receiving immunosuppressive doses of corticosteroids. Administer live-attenuated or live vaccines at least 4 to 6 weeks prior to starting Emflaza.
• Discontinue at the first sign of rash, unless the rash is clearly not drug related.

What to do in case of emergency/overdose?

Management for overdosage:

• Treatment of acute overdosage is by immediate gastric lavage or emesis followed by supportive and symptomatic therapy.
• For chronic overdosage in the face of severe disease requiring continuous steroid therapy, the dosage of Emflaza may be reduced temporarily, or alternate day treatment may be introduced.

Can this medicine be used in pregnancy?

• Corticosteroids should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus.
• There are no adequate and well-controlled studies with Emflaza in pregnant women to inform drug-associated risks.

Can this medicine be used in children?

• The safety and effectiveness of Emflaza for the treatment of DMD have been established in patients 2 years of age and older.
• Safety and effectiveness in pediatric patients below the age of 2 years have not been established.

What are the active and inactive ingredients in this medicine?

• Active ingredient: deflazacort
• Inactive ingredients: acetic acid, aluminum magnesium silicate, benzyl alcohol, carboxymethylcellulose sodium, polysorbate 80, purified water, and sorbitol.

Who manufactures and distributes this medicine?

• Manufactured for: PTC Therapeutics, Inc., South Plainfield, NJ, U.S.A.

What should I know about storage and disposal of this medication?

• Store at 20°C to 25°C (68°F to 77°F).
• Excursion permitted between 15°C to 30°C (59°F to 86°F).
• Discard any unused Emflaza Oral Suspension remaining after 1 month of first opening the bottle.

• Dailymed label info on Deflazacort
• FDA Deflazacort

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