Deflazacort 22.75mg/1ml Suspension

Deflazacort is a corticosteroid that acts as a glucocorticoid receptor agonist. It exerts its anti-inflammatory and immunosuppressive effects by binding to glucocorticoid receptors in the cytoplasm, leading to a cascade of events that modulate gene expression. This results in the inhibition of inflammatory mediators (e.g., prostaglandins, leukotrienes, cytokines) and suppression of immune cell function.

Serious 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 reactions: 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.
Infections: Fever, chills, severe sore throat, ear or sinus pain, cough, increased or discolored sputum, painful urination, mouth sores, or wounds 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 gland problems: 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 problems: 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: Blurred vision or other changes in eyesight.
Musculoskeletal problems: Bone or joint pain.
Gastrointestinal problems: 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 discoloration: 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 the arms or legs, or difficulty speaking or swallowing.
Severe skin reactions: Toxic epidermal necrolysis, which can cause severe health problems or death. Seek medical help immediately if you experience red, swollen, blistered, or peeling skin (with or without fever), red or irritated eyes, or sores in the mouth, throat, nose, or eyes.

Other Side Effects

Most people experience few or no side effects while taking this medication. However, if you notice any of the following side effects, contact your doctor if they bother you or persist:

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 potential side effects. If you have questions or concerns about side effects, 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. Describe the allergic reaction you experienced, including the symptoms that occurred.
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
+ Any other type of infection
Certain health problems, such as:
+ Diverticulitis
+ Hole in the gastrointestinal (GI) tract
+ Stomach or bowel infection
+ Ulcers
Recent stomach or bowel surgery
* Any medications you are currently 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 conditions with your doctor and pharmacist. Do not start, stop, or change the dose of any medication without consulting your doctor first.

Important Warnings and Precautions

It is essential to inform all your healthcare providers, including doctors, nurses, pharmacists, and dentists, that you are taking this medication.

Monitoring and Tests

Your doctor will require you to undergo regular blood tests to monitor your condition. Additionally, if you are taking this medication for an extended period, you may need to have your eye pressure and bone density checked.

Potential Risks and Side Effects

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

High blood pressure is a possible side effect of this medication. Ensure that your blood pressure is monitored as directed by your doctor. If you have diabetes, it is crucial to closely monitor your blood sugar levels. You may need to reduce your salt intake and take potassium supplements; consult with your doctor for guidance.

Interactions and Precautions

Avoid consuming grapefruit and grapefruit juice while taking this medication. If you are not up to date with all recommended vaccinations, discuss this with your doctor. You may need to receive certain vaccinations 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 with your doctor before receiving any vaccinations.

Infections and Illnesses

If you have not had chickenpox or measles before, avoid being near anyone with these illnesses, as they can be severe or even life-threatening in people taking steroid medications like this one. If you have been exposed to chickenpox or measles, inform your doctor immediately.

Steroid medications, including this one, can increase the risk of infection, which can be mild or severe. They can also make it more challenging to detect signs of infection. To minimize the risk of infection, wash your hands frequently, avoid people with infections, colds, or flu, and inform your doctor if you experience any symptoms of infection.

Some infections, such as tuberculosis and hepatitis B, may reactivate in patients taking this medication. If you have a history of these infections, inform your doctor.

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 with 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 Side Effects

Long-term use of this medication can 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 have been reported in patients taking this medication. If you have a history of blood clots, inform your doctor. Additionally, long-term use of this medication has been associated with an increased risk of a type of cancer called Kaposi's sarcoma. Discuss this risk with your doctor.

Stopping the Medication

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 provide guidance on how to gradually discontinue it. If you have missed a dose or recently stopped taking this medication and experience symptoms such as fatigue, weakness, shakiness, rapid heartbeat, confusion, sweating, or dizziness, inform your doctor.

Special Considerations

This medication may affect growth in children and adolescents. Regular growth checks may be necessary; discuss this with your doctor. The medication contains benzyl alcohol, which can cause serious side effects in newborns and infants. If possible, avoid products containing benzyl alcohol in this population.

Pregnancy and Breastfeeding

If you are pregnant, plan to become pregnant, or are breastfeeding, inform your doctor to discuss the potential benefits and risks of this medication to you and your baby.

• Live or live attenuated vaccines (during deflazacort treatment)

• Strong CYP3A4 inhibitors (e.g., ketoconazole, clarithromycin, ritonavir, itraconazole, grapefruit juice): May significantly increase deflazacort exposure, increasing risk of adverse effects. Consider alternative or dose reduction of deflazacort.
• Strong CYP3A4 inducers (e.g., rifampin, phenytoin, carbamazepine, phenobarbital): May significantly decrease deflazacort exposure, reducing efficacy. Consider alternative or dose increase of deflazacort.

• Moderate CYP3A4 inhibitors (e.g., erythromycin, diltiazem, verapamil): May increase deflazacort exposure. Monitor for adverse effects.
• Moderate CYP3A4 inducers (e.g., efavirenz, etravirine): May decrease deflazacort exposure. Monitor for efficacy.
• Anticoagulants (e.g., warfarin): May alter anticoagulant effects (increase or decrease). Monitor INR/PT.
• Nonsteroidal Anti-inflammatory Drugs (NSAIDs): Increased risk of gastrointestinal ulceration and bleeding.
• Diuretics (thiazide, loop): Increased risk of hypokalemia.
• Antidiabetic agents (e.g., insulin, oral hypoglycemics): May increase blood glucose levels, requiring dose adjustment of antidiabetic agents.
• Cholestyramine: May decrease absorption of deflazacort.

• Oral contraceptives: May increase plasma concentrations of corticosteroids.

• Signs of infection (fever, chills, sore throat, cough, unusual fatigue, localized pain/redness/swelling)
• Signs of adrenal insufficiency (fatigue, weakness, nausea, vomiting, hypotension, hypoglycemia, dizziness)
• Signs of Cushing's syndrome (moon face, buffalo hump, central obesity, thin skin, easy bruising, striae, muscle weakness)
• Signs of hyperglycemia (increased thirst, increased urination, blurred vision)
• Mood changes (irritability, anxiety, depression, euphoria)
• Gastrointestinal symptoms (abdominal pain, black/tarry stools, vomiting blood)
• Muscle weakness or pain
• Swelling in ankles or feet
• Vision changes

Deflazacort may cause fetal harm when administered to a pregnant woman. Based on animal studies, it can cause adverse developmental effects. Use only if the potential benefit justifies the potential risk to the fetus.

Deflazacort and its active metabolite are present in human milk. Due to the potential for serious adverse reactions in the breastfed infant (e.g., growth suppression, interference with endogenous corticosteroid production), breastfeeding is not recommended during treatment with deflazacort.

Approved for patients 2 years of age and older with Duchenne muscular dystrophy. Long-term use in pediatric patients can cause growth suppression; monitor growth velocity regularly. Increased risk of infections and other corticosteroid-related adverse effects.

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 at increased risk for adverse reactions common to corticosteroids, such as osteoporosis and fluid retention.

• Deflazacort is a corticosteroid with a unique oxazoline ring, which may contribute to a different side effect profile compared to other corticosteroids like prednisone, particularly regarding bone health and glucose metabolism, though these effects still occur.
• Always taper deflazacort slowly when discontinuing to prevent acute adrenal insufficiency.
• Patients should be monitored closely for signs of infection, especially opportunistic infections, due to immunosuppression.
• Regular ophthalmologic exams are crucial for long-term users to screen for cataracts and glaucoma.
• Bone health should be proactively managed with calcium, vitamin D, and potentially bisphosphonates, especially in pediatric patients and those at high risk for osteoporosis.
• Patients should carry a steroid emergency card to alert healthcare providers of their corticosteroid use in emergencies.

• Prednisone (another corticosteroid used for DMD)
• Eteplirsen (Exondys 51, for DMD amenable to exon 51 skipping)
• Golodirsen (Vyondys 53, for DMD amenable to exon 53 skipping)
• Viltolarsen (Viltepso, for DMD amenable to exon 53 skipping)
• Casimersen (Amondys 45, for DMD amenable to exon 45 skipping)
• Ataluren (Translarna, for nonsense mutation DMD, approved in some regions but not US)
• Gene therapies (e.g., Delandistrogene moxeparvovec - Elevidys, for DMD with specific mutations)

If your symptoms or health issues persist or worsen, it is 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 it's a good idea to check with your pharmacist. If you have any questions or concerns about this medication, don't hesitate to discuss them with 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 detailed information about the overdose, including the medication taken, the amount, and the time it occurred.