EMFLAZA
Clinical safety rating: caution
Comprehensive clinical and safety monograph for EMFLAZA (EMFLAZA).
Agonist at glucocorticoid receptors, modulating gene expression to suppress inflammation and immune response.
| Metabolism | Primarily hepatic via CYP3A4 to active metabolite 6β-hydroxy-deflazacort. |
| Excretion | Renal excretion of inactive metabolites; less than 5% excreted as unchanged drug in urine. Biliary/fecal elimination accounts for <1%. |
| Half-life | 6.2 hours (range 4.5–8.1 h) in healthy adults; prolonged in hepatic impairment. |
| Protein binding | Approximately 85–90% bound to albumin and alpha-1-acid glycoprotein. |
| Volume of Distribution | Vd ~0.6 L/kg; distributes widely into tissues, including skeletal muscle. |
| Bioavailability | Oral bioavailability is approximately 30% (range 20–40%) due to first-pass metabolism; absolute bioavailability is 30%. |
| Onset of Action | Oral: ~1–2 hours for initial anti-inflammatory effect; maximal effect by 4–8 hours. |
| Duration of Action | Duration of anti-inflammatory effects is 24–36 hours after a single dose; therapeutic effects for Duchenne muscular dystrophy persist for 24 hours with once-daily dosing. |
| Molecular Weight | 441.52 |
0.6 mg/kg orally once daily (maximum 60 mg/day); titrate to lowest effective dose based on clinical response.
| Dosage form | SUSPENSION |
| Renal impairment | No dose adjustment required for renal impairment; has not been studied in ESRD. |
| Liver impairment | For Child-Pugh class A or B, reduce dose by 50%; for Child-Pugh class C, contraindicated. |
| Pediatric use | Approved for Duchenne muscular dystrophy in patients aged 2 years and older: 0.6 mg/kg orally once daily (maximum 60 mg/day). |
| Geriatric use | No specific dose adjustments; use caution due to increased risk of adverse effects (e.g., osteoporosis, infections). |
| 1st trimester | Avoid use unless no safer alternative; limited human data, animal studies show teratogenicity and embryotoxicity at clinically relevant doses. |
| 2nd trimester | Avoid unless maternal benefit outweighs fetal risk; may cause fetal adrenal suppression and cleft palate. |
| 3rd trimester | Avoid unless maternal benefit outweighs fetal risk; may cause neonatal adrenal suppression, cleft palate, and low birth weight. |
Clinical note
Comprehensive clinical and safety monograph for EMFLAZA (EMFLAZA).
| Placental transfer | Deflazacort crosses the placenta; its active metabolite 21-desacetyldeflazacort also crosses; ratio of cord to maternal concentration is approximately 0.1-0.2. |
| Breastfeeding | Emflaza (deflazacort) is excreted into human milk in small amounts. Caution should be exercised; monitor infant for adrenal suppression and growth retardation. Consider alternatives with lower transfer such as prednisolone. |
■ FDA Black Box Warning
None
| Serious Effects |
Hypersensitivity to deflazacort or any excipientSystemic fungal infectionsAdministration of live or live-attenuated vaccines in patients on immunosuppressive doses
| Precautions | Increased risk of infection, Adrenal insufficiency, Cushing's syndrome, Osteoporosis, Gastrointestinal perforation, Behavioral and mood disturbances, Immunosuppression |
| Food/Dietary | Grapefruit and grapefruit juice increase deflazacort exposure; avoid concurrent use. High-sodium foods may exacerbate fluid retention; limit salt intake. Calcium and vitamin D supplementation recommended to mitigate osteoporosis risk. Take with food to reduce gastrointestinal irritation. |
Loading safety data…
| Lactation Rating | L3 (Moderately Safe) |
| Teratogenic Risk | EMFLAZA (deflazacort) is a corticosteroid. Corticosteroids are associated with an increased risk of orofacial clefts when used in the first trimester. In the second and third trimesters, prolonged use may cause fetal adrenal suppression, intrauterine growth restriction, and premature birth. Increased maternal blood pressure and glucose levels may also affect the fetus. |
| Fetal Monitoring | Monitor maternal blood glucose, blood pressure, and signs of infection. For the fetus, consider growth ultrasounds every 4-6 weeks during prolonged therapy. Assess for adrenal insufficiency in the newborn if used in the late third trimester. |
| Fertility Effects | Corticosteroids like deflazacort may alter menstrual cycles and reduce sperm motility or count in males, but effects on fertility are generally reversible upon discontinuation. |
| Clinical Pearls |
| EMFLAZA (deflazacort) is a corticosteroid with a 6-7 hour half-life; monitor for adrenal suppression and growth retardation in children. Taper dose when discontinuing to avoid withdrawal symptoms. In Duchenne muscular dystrophy, start at 0.9 mg/kg/day and titrate to effect. Avoid live vaccines during therapy. |
| Patient Advice | Take exactly as prescribed; do not stop suddenly without doctor's guidance. · May increase risk of infections; avoid sick contacts and report fever or sore throat. · Long-term use can cause weight gain, osteoporosis, and high blood sugar; monitor weight and blood glucose. · May cause mood changes, insomnia, or anxiety; report severe mood swings. · Avoid grapefruit and grapefruit juice; discuss all medications and supplements with your doctor. |