Emflaza 30mg Tablets

Deflazacort is a corticosteroid that acts as a glucocorticoid receptor agonist. It exerts its anti-inflammatory and immunosuppressive effects by binding to intracellular glucocorticoid receptors, leading to modulation of gene expression. This results in decreased synthesis of inflammatory mediators, inhibition of inflammatory cell migration, and suppression of immune responses. In Duchenne Muscular Dystrophy, the exact mechanism by which deflazacort exerts its therapeutic effect is unknown, but it is thought to involve its anti-inflammatory and immunomodulatory properties.

Urgent Side Effects: Seek Medical Help Right Away

Although rare, some people may experience severe and potentially life-threatening side effects while taking this medication. If you notice any of the following symptoms, contact your doctor immediately or seek emergency medical attention:

Allergic Reaction: Rash, hives, itching, red, swollen, blistered, or peeling skin (with or without fever), wheezing, tightness in the chest or throat, difficulty breathing, swallowing, or talking, unusual hoarseness, or swelling of the mouth, face, lips, tongue, or throat.
Infection: Fever, chills, severe sore throat, ear or sinus pain, cough, increased or discolored sputum, painful urination, mouth sores, or a wound that won't heal.
High Blood Sugar: Confusion, drowsiness, excessive thirst or hunger, frequent urination, flushing, rapid breathing, or fruity-smelling breath.
Cushing's Syndrome: Weight gain in the upper back or abdomen, moon face, severe headache, or slow wound healing.
Adrenal Insufficiency: Severe nausea or vomiting, extreme dizziness or fainting, muscle weakness, fatigue, mood changes, decreased appetite, or weight loss.
High Blood Pressure: Severe headache or dizziness, fainting, or changes in vision.
Electrolyte Imbalance: Mood changes, confusion, muscle pain, cramps, or spasms, weakness, shakiness, balance problems, abnormal heartbeat, seizures, loss of appetite, or severe nausea or vomiting.
Respiratory Issues: Shortness of breath, significant weight gain, or swelling in the arms or legs.
Skin Changes: Acne, stretch marks, slow healing, or excessive hair growth.
Vision Changes: Changes in eyesight.
Musculoskeletal Issues: Bone or joint pain.
Gastrointestinal Issues: Black, tarry, or bloody stools, or vomiting blood or coffee ground-like material.
Mental Health Changes: New or worsening mental, mood, or behavioral changes.
Skin Lesions: Purple, red, blue, brown, or black bumps or patches on the skin or in the mouth.
Blood Clots: Chest pain or pressure, coughing up blood, shortness of breath, swelling, warmth, numbness, color changes, or pain in a leg or arm, or difficulty speaking or swallowing.
Toxic Epidermal Necrolysis: A severe skin reaction that can cause serious health problems or death. Seek immediate medical attention if you experience red, swollen, blistered, or peeling skin (with or without fever), red or irritated eyes, or sores in your mouth, throat, nose, or eyes.

Other Possible Side Effects

While many people may not experience side effects or only have mild ones, it's essential to discuss any concerns with your doctor. If you notice any of the following side effects, contact your doctor for guidance:

Weight gain
Increased appetite
Common cold symptoms
Nose and throat irritation
Frequent urination
Stomach pain
Constipation
Back pain
Upset stomach
Headache

This is not an exhaustive list of possible side effects. If you have questions or concerns, consult your doctor. You can also report side effects to the FDA at 1-800-332-1088 or online at https://www.fda.gov/medwatch.

Before Taking This Medication: Important Information to Share with Your Doctor

It is essential to inform your doctor about the following conditions to ensure safe treatment:

Any allergies you have, including allergies to this medication, its components, or other substances, such as foods or drugs. Be sure to describe the allergic reaction and its symptoms.
The presence of any infection, including bacterial, viral, or fungal infections, as well as specific conditions like:
+ Amoeba infection (e.g., traveler's diarrhea)
+ Herpes infection of the eye
+ Malaria infection in the brain
+ Threadworm infestation
+ Other types of infections
Certain health problems, including:
+ Diverticulitis
+ Hole in the gastrointestinal (GI) tract
+ Stomach or bowel infection
+ Ulcers
Recent stomach or bowel surgery
* Any medications you are taking, including prescription and over-the-counter (OTC) drugs, natural products, and vitamins, as some may interact with this medication. Specifically, inform your doctor about:
+ Certain HIV medications
+ Infection treatments
+ Seizure medications
+ Other medications that may interact with this drug

Please note that this is not an exhaustive list of all potential interactions. To ensure your safety, it is crucial to discuss all your medications and health problems with your doctor and pharmacist. Do not start, stop, or change the dose of any medication without first consulting your doctor to confirm that it is safe to do so.

Important Warnings and Cautions

It is essential to inform all your healthcare providers, including doctors, nurses, pharmacists, and dentists, that you are taking this medication. Regular blood tests, as directed by your doctor, are crucial to monitor your health. Additionally, if you are taking this medication long-term, you may need to have your eye pressure and bone density checked.

Long-term Use Risks

Prolonged use of this medication may increase the risk of developing cataracts or glaucoma. Discuss this with your doctor to understand the potential risks. Furthermore, long-term use may cause weak bones (osteoporosis). Consult your doctor to determine if you are at a higher risk or have any questions.

Blood Pressure and Sugar Monitoring

This medication may cause high blood pressure. Regular blood pressure checks, as advised by your doctor, are necessary. If you have diabetes, it is crucial to closely monitor your blood sugar levels. You may need to reduce your salt intake and increase your potassium consumption. Consult your doctor for guidance.

Dietary Restrictions and Vaccine Precautions

Avoid consuming grapefruit and grapefruit juice while taking this medication. If you are not up to date with all your vaccines, discuss this with your doctor. You may need to receive certain vaccines before starting treatment with this medication. However, some vaccines may not be effective or may increase the risk of infection when taken with this medication. Consult your doctor before receiving any vaccines.

Infection Risks

Steroid medications, including this one, increase the risk of infection. They can worsen existing infections and make it more challenging to detect signs of infection. Infections can range from mild to severe and potentially life-threatening. The risk of infection is higher with increased doses of steroids. To minimize the risk, wash your hands frequently, avoid people with infections, colds, or flu, and inform your doctor if you experience any signs of infection.

Reactivation of Infections

In some cases, this medication may reactivate latent infections, such as tuberculosis or hepatitis B. Inform your doctor if you have a history of these infections.

Adrenal Insufficiency and Stress

This medication may decrease the natural production of steroids in your body. If you experience a fever, infection, surgery, or injury, consult your doctor, as you may require additional oral steroids to help your body cope with stress. Carry a warning card indicating that you may need extra steroids in certain situations.

Rare but Serious Risks

Long-term use of this medication, especially at high doses, may lead to rare but potentially life-threatening conditions, including changes in adrenal gland function and the development of a tumor on the adrenal gland (pheochromocytoma). Discuss these risks with your doctor.

Blood Clots and Cancer Risks

This medication may increase the risk of blood clots. Inform your doctor if you have a history of blood clots. Additionally, long-term use of this medication may increase the risk of a type of cancer called Kaposi's sarcoma. Discuss this risk with your doctor.

Discontinuation and Missed Doses

Do not stop taking this medication abruptly without consulting your doctor, as this may increase the risk of side effects. If you need to stop taking this medication, your doctor will guide you on how to gradually discontinue it. If you miss a dose or recently stopped taking this medication and experience fatigue, weakness, shakiness, fast heartbeat, confusion, sweating, or dizziness, inform your doctor.

Effects on Growth and Pregnancy

This medication may affect growth in children and teenagers. Regular growth checks may be necessary. Inform your doctor if you are pregnant, plan to become pregnant, or are breastfeeding, as you will need to discuss the benefits and risks to you and your baby.

• Live or live attenuated vaccines (increased risk of infection)
• Strong CYP3A4 inhibitors (e.g., ketoconazole, ritonavir, clarithromycin) - may increase deflazacort exposure and adverse effects.
• Strong CYP3A4 inducers (e.g., rifampin, phenytoin, carbamazepine, phenobarbital) - may decrease deflazacort exposure and efficacy.

• Anticoagulants (e.g., warfarin) - altered anticoagulant effect (monitor INR)
• Non-steroidal anti-inflammatory drugs (NSAIDs) - increased risk of GI ulceration/bleeding
• Diuretics (e.g., thiazides, loop diuretics) - enhanced potassium loss, increased risk of hypokalemia
• Antidiabetic agents (e.g., insulin, oral hypoglycemics) - deflazacort can increase blood glucose, requiring dose adjustments of antidiabetics
• Cardiac glycosides (e.g., digoxin) - increased risk of toxicity with hypokalemia
• Oral contraceptives - may increase deflazacort exposure
• Cholestyramine - may decrease deflazacort absorption

• Signs of infection (fever, chills, sore throat, cough, unusual fatigue, localized pain/redness)
• Changes in mood or behavior (irritability, anxiety, depression, insomnia, euphoria)
• Gastrointestinal symptoms (abdominal pain, black/tarry stools, vomiting blood)
• Visual changes (blurred vision, eye pain, halos around lights)
• Signs of adrenal insufficiency (severe fatigue, weakness, dizziness, nausea, vomiting, loss of appetite, weight loss) upon withdrawal or stress
• Signs of Cushing's syndrome (moon face, buffalo hump, central obesity, thin skin, easy bruising)
• Muscle weakness or pain (beyond baseline DMD symptoms)
• Increased thirst or urination (signs of hyperglycemia)
• Swelling in ankles or feet, shortness of breath (signs of fluid retention/heart failure)

Category C. Use during pregnancy only if the potential benefit justifies the potential risk to the fetus. Corticosteroids can cross the placenta and may be associated with adverse effects such as fetal growth restriction, cleft palate, and adrenal suppression in the neonate.

Deflazacort and its active metabolite are excreted into human milk. Due to the potential for serious adverse reactions in the breastfed infant (e.g., growth suppression, interference with endogenous corticosteroid production), a decision should be made whether to discontinue nursing or to discontinue the drug, taking into account the importance of the drug to the mother.

Approved for patients 2 years of age and older. Long-term use in pediatric patients can cause growth suppression, osteoporosis, cataracts, and glaucoma. Close monitoring of growth, bone density, and ophthalmologic status is essential.

Clinical studies did not include sufficient numbers of subjects aged 65 and over to determine whether they respond differently from younger subjects. In general, dose selection for an elderly patient should be cautious, usually starting at the low end of the dosing range, reflecting the greater frequency of decreased hepatic, renal, or cardiac function, and of concomitant disease or other drug therapy. Elderly patients may be more susceptible to adverse effects of corticosteroids, such as osteoporosis and fluid retention.

• Emflaza is a corticosteroid specifically approved for Duchenne Muscular Dystrophy (DMD) and should not be used interchangeably with other corticosteroids for other indications due to its unique dosing and potential side effect profile in DMD.
• Patients and caregivers must be educated on the importance of not abruptly discontinuing Emflaza due to the risk of adrenal crisis.
• Monitor for signs of infection, as corticosteroids can mask symptoms and increase susceptibility.
• Regular ophthalmologic exams are crucial due to the risk of cataracts and glaucoma.
• Bone health monitoring (DEXA scans) and calcium/vitamin D supplementation should be considered due to the risk of osteoporosis.
• Patients should carry a steroid card or medical alert identification indicating corticosteroid use.
• Consider stress-dose steroids during periods of severe illness, surgery, or trauma.
• Counsel on potential psychiatric side effects, including mood changes, insomnia, and anxiety.

• Prednisone/Prednisolone (other corticosteroids used off-label or historically for DMD)
• Vamorolone (another corticosteroid-like drug for DMD, approved later)
• Gene therapy (e.g., Elevidys for DMD, for specific mutations)
• Exon-skipping therapies (e.g., eteplirsen, golodirsen, viltolarsen, casimersen for specific DMD mutations)

If your symptoms or health issues persist or worsen, it's essential to contact your doctor for further guidance. To ensure your safety, never share your medication with others or take someone else's medication. Some medications may come with an additional patient information leaflet, so be sure to check with your pharmacist for more information. If you have any questions or concerns about your medication, don't hesitate to reach out to your doctor, nurse, pharmacist, or other healthcare provider. In the event of a suspected overdose, immediately call your local poison control center or seek emergency medical attention. When seeking help, be prepared to provide details about the medication taken, the amount, and the time it happened.