Emflaza

(Deflazacort)

The active ingredient in Emflaza is deflazacort, which is a type of corticosteroid therapy. Its active metabolite has an anti-inflammatory and immunosuppressant effect on the body. The exact mechanism by which Emflaza improves DMD is unknown. It has been shown to help improve muscle strength and slow disease progression.

Follow all directions given to you by your doctor and read all documents provided, including the full Prescribing Information, FDA-Approved Product Labeling, Instructions for Use, and Medication Guide before using Emflaza. However, the following is a synopsis of the general guidelines for the use of Emflaza:

• Emflaza should be used exactly as prescribed.
• It is typically taken daily with or without food. Do not take with grapefruit juice.
• If there are difficulties with swallowing, caregivers can crush the tablet and sprinkle it over apple sauce for easier administration.
• If using the Emflaza oral suspension, shake the bottle well before use and measure each dose using the oral dispenser. If taste is an issue, the dose may be mixed in juice or milk and taken immediately. Do not mix with grapefruit juice. Once a bottle is open, it can be used for up to 1 month. Any remainder should be discarded.
• Do not use the oral suspension in children less than 2 years of age. The suspension contains benzyl alcohol preservative, which can cause serious adverse reactions in infants, including difficulty breathing, loss of consciousness, and metabolic abnormalities.
• 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 continue with your regular dosing schedule. Do not take a double dose to make up for a missed one.

The most common side effects of Emflaza seen in clinical trials as compared to placebo include:

• Cushingoid appearance, such as a puffy face, thinning skin, muscle weakness, hirsutism, and excess weight in the abdominal and chest area with skinny arms and legs (central obesity).
• Weight gain.
• Increased appetite.
• Cold & flu symptoms (upper respiratory tract infections). You are at an increased risk of infections while using Emflaza as it suppresses your immune system. Be watchful of signs of infection.
• Runny nose.
• Cough.
• Frequent daytime urination.
• Stomach discomfort.

Some individuals may experience serious side effects while using Emflaza. These side effects may require immediate medical attention. If you experience any of the following, contact your healthcare provider or seek emergency medical help:

• Signs of withdrawal if Emflaza is stopped abruptly, such as nausea, vomiting, unusual fatigue, headache, fever, joint and muscle pain, peeling skin, and unexplained weight loss.
• Hyperglycemia or high blood sugar levels. Patients with diabetes should monitor for loss of blood glucose control.
• Signs of pheochromocytoma crisis such as headache, excessive sweating, high blood pressure, and a panic attack. This is a life-threatening condition that requires prompt medical attention.
• Signs of chicken pox such as a widespread and itchy rash that may blister.
• Signs of measles such as a widespread and non-itchy rash with fever, cough, runny nose, and pink eye.
• Signs of a perforation in the GI tract such as blood in stools or stools that appear dark and tarry in appearance, blood in vomit or vomit that resembles coffee ground, or abdominal pain.
• Signs of severe psychiatric changes, such as mood swings, mania, depression, or suicidal ideation.
• Changes in vision or eye pain. Emflaza can cause cataracts or glaucoma in some patients.
• Signs of TEN (toxic epidermal necrolysis), a life-threatening and severe skin reaction characterized by a widespread, painful skin rash that may blister, form open wounds, and cause skin peeling.
• Any new growths on the skin that may be raised or flat, or are red, purple, or brown in color, should be followed up on. These could be signs of Kaposi’s Sarcoma, which is a type of cancer.
• Signs of anaphylaxis or a severe allergic reaction, such as hives, rash, difficulty breathing, or swelling of the face, lips, eyes, or tongue.

These are not all of the possible side effects of Emflaza. You should always seek medical advice from your healthcare provider for any questions or concerns about your medical condition or treatment. Read all patient information, medication guides, or drug information sheets that come with this medication. You can also report adverse effects to the U.S. Food and Drug Administration at www.fda.gov/medwatch or 1-800-FDA-1088.

Before starting Emflaza, it is crucial to discuss any existing health conditions with your healthcare provider. You should be sure that your healthcare provider is aware of all your medical conditions, including:

• Recent or ongoing infection.
• Recently received a vaccine.
• High blood pressure.
• Kidney problems.
• Thyroid problems.
• Active or latent tuberculosis.
• History of blood clots or VTEs.
• Osteoporosis or other bone problems. Emflaza can decrease bone mineral density and increase the risk of bone fractures.
• Eye problems.

Do not use Emflaza if you have:

• An allergy or hypersensitivity to deflazacort or any of the other ingredients in Emflaza

Emflaza begins to work right after the first dose. However, it can take a few weeks of regular use to see the full benefits.

When Emflaza is taken with other prescription drugs, over-the-counter medications, vitamins, and supplements, it may change how they work or increase the frequency or severity of side effects. Make sure that you tell your healthcare professional about anything that you are taking to avoid any negative drug interactions, including:

• Clarithromycin
• Fluconazole
• Diltiazem
• Verapamil
• Rifampin
• Efavirenz
• Carbamazepine
• Phenytoin
• Pancuronium

Emflaza is available in 2 dosage forms. The oral tablets are available in doses of 6 mg, 18 mg, 30 mg, and 36 mg. The oral suspension is available as 22.75 mg/mL.

Emflaza should be stored at room temperature, away from light and moisture, and out of reach of children.

Emflaza is manufactured by the pharmaceutical company PTC Therapeutics.

Yes, there is a generic available for the brand-name Emflaza.

Emflaza is typically a lifelong medication. If it does need to be stopped, it should be done so slowly to prevent any adverse reactions. Consult your doctor or pharmacist before starting, stopping, or changing medications while taking Emflaza.

It is unknown whether Emflaza is safe in pregnancy. Talk to your doctor if you are already pregnant or if you have any plans to become pregnant. They will know or recommend what is best for you.

Emflaza can pass into the breast milk. Talk to your doctor if you are already breastfeeding or if you have any plans to breastfeed. They will know or recommend what is best for you.