Deflazacort

Common name: Deflazacort

Trade name: Defcort

All names: Deflazacort, Desfluzacort, Deflazacort, Emflaza, Defcort

Indications:

Deflazacort is a glucocorticoid indicated for the treatment of Duchenne muscular dystrophy (DMD) in adults and children at least 2 years of age. Deflazacort does not cure muscular dystrophy, but it can improve muscle strength and slow the progression of disability. It may also be used for purposes not listed in this guide.

Usage and dosage:

For Duchenne muscular dystrophy (DMD):

Adults and children 2 years and older: 0.9 mg/kg orally daily /day

Children younger than 2 years: Safety and effectiveness have not been established

If tablets are used, round to the nearest dose; any combination of tablet strengths may be used to achieve the calculated dose. If oral suspension is used, round to the nearest 1/10 milliliter (mL). When discontinuing treatment, gradually reduce the dose if administered for more than a few days.

Dose adjustment:

Coadministration with CYP3A4 inhibitors: For moderate or strong CYP3A4 inhibitors, give 1/3 the recommended dose; Example: When used with moderate or strong CYP3A4 inhibitors, reduce the 36 mg/day dose to 12 mg/day.

Coadministration with CYP3A4 inducers: Avoid use with moderate or strong CYP3A4 inducers.

Specifications:

Tablet 6mg/18mg/30mg/36mg

Oral suspension 22.75mg/ml

Adverse reactions:

Common side effects: weight gain, increased appetite, upper respiratory tract infection, cough, frequent urination

Uncommon side effects: runny or stuffy nose, unwanted hair growth, central obesity, irritability, abdominal discomfort, eye diseases; gastrointestinal diseases: indigestion/ Heartburn, Nausea, General Illnesses and Administration Site Conditions: Thirst; Infections: Stye, Impetigo, Influenza, External Ear Infections, Sore Throat, Dental Abscess, urinary tract infection (UTI), viral infection; poisoning and surgical complications: back injuries, contusions, facial injuries, fibula fractures, bruise fractures, physical exertion; presence of glucosuria, irregular heart rhythm; musculoskeletal and connective tissue disorders: back pain, muscle spasms, muscle pain, neck mass, neck pain, limb pain; neurological disorders: dizziness, psychomotor hyperactivity; mental disorders: affective Unsteadiness, aggression, depression, mood disorders, intermittent insomnia, mood changes, mood swings, sleep disorders; Kidney and urinary system disorders: abnormal discoloration of urine, painful urination, hypertonic bladder; Reproductive system and breast disorders: testicular pain; Respiratory, thoracic and mediastinal disorders: hypoventilation, runny nose; Skin and subcutaneous disorders Tissue disorders: Acne, alopecia, dermatitis; Vascular disorders: Hot flashes

Contraindications:

Allergy to any inactive ingredient of deflazacort

Precautions:

Assessment before initiating treatment: Perform all immunizations according to immunization guidelines; administer attenuated or live vaccines at least 4-6 weeks before initiating treatment.

Corticosteroids (eg, deflazacort) should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. Infants born to mothers who received high doses of corticosteroids during pregnancy should be carefully observed for signs of adrenocortical insufficiency. Consult your doctor.

Systemically administered corticosteroids occur in human milk and may inhibit growth, interfere with endogenous corticosteroid production, or cause other adverse effects. Consider the developmental and health benefits of breastfeeding, as well as the mother's clinical need for deflazacort, and any potential adverse effects on the breastfed infant from this drug or the underlying maternal condition.

Like other glucocorticoids, it is contraindicated in systemic infectious diseases.

Use with caution for conditions such as diverticulitis, recent gastrointestinal surgery, renal failure, hypertension, diabetes, osteoporosis, and myasthenia gravis.

Storage:

Seal and store at 15-30℃.

Mechanism of action:

Deflazacort is a third-generation glucocorticoid that has anti-inflammatory, anti-allergic, and increased gluconeogenesis effects. The effect is equivalent to 10 to 20 times that of prednisolone and 40 times that of hydrocortisone. This product has no mutagenic effect. Research on this product labeled with C14 shows that this product is rapidly absorbed from the gastrointestinal tract, and reaches its peak blood concentration after 1 to 2 hours, and is hydrolyzed to form active substances. The further metabolic process of the latter is similar to other glucocorticoids. The T1/2 of the active metabolites is 2 hours. In rats, it is mainly distributed in tissues such as the kidneys and hematopoietic system.

Safety and Efficacy:

The effectiveness of deflazacort was based on a study of 196 male patients who were 5 to 15 years old at the start of the trial and had documented dystrophin gene mutations who developed muscle weakness before the age of 5 years. At 12 weeks into the trial, patients taking deflazacort had improved clinical assessments of strength in many muscles throughout their body compared to those taking placebo. At the end of the 52-week trial, mean muscle strength remained generally stable among deflazacort-treated patients. In another 104-week trial involving 29 male patients, deflazacort demonstrated a quantitative advantage over placebo on measures of mean muscle strength. In addition, although no statistical pairwise comparisons were made, deflazacort-treated patients appeared to lose the ability to walk later than placebo-treated patients.

Not only that, in addition to its relatively good therapeutic effect in the treatment of our patients, deflazacort also demonstrated very high drug safety in the trial. Therefore, the U.S. FDA granted deflazacort fast track status and priority review status, and it also obtained orphan drug designation (specifically refers to drugs that treat rare diseases and can enjoy R&D and production incentive policies).