デフラザコート

Pharmaceuticals -> Steroids

Pharmaceuticals -> Listed in ZINC15

Breast Feeding; Lactation; Corticosteroids, Systemic; Glucocorticoids; Anti-Inflammatory Agents;

FDA approved drugs -> Active ingredient

FDA approved drugs -> Active ingredient

Danger

H360 (77.4%): May damage fertility or the unborn child [Danger Reproductive toxicity]

P203, P260, P280, P318, P319, P405, and P501 (click each P-code to see the statement)

Aggregated GHS information provided per 31 reports by companies from 8 notifications to the ECHA C&L Inventory. Each notification may be associated with multiple companies.

Drug Induced Liver Injury Rank (DILIrank 2.0)

Deflazacort

vNo-DILI-concern

0

No match

DOI:10.1016/j.drudis.2016.02.015

Deflazacort

1.7

7.1

The recommended oral dosage of deflazacort is approximately 0.9 mg/kg/day once daily. (LDD calculated assuming 80 kg adult.)

Data used to generate the benchmarks include the following: Lowest Daily Dose (LDD): 72 mg/day, Lowest Therapeutic Dose (LTD): 0.9 mg/kg-day, and Screening Dose: 0.0003 mg/kg-day.

6-Oct-21

H02AB13

H - Systemic hormonal preparations, excl. sex hormones and insulins

QH - Systemic hormonal preparations, excl. sex hormones and insulin

Deflazacort exerts anti-inflammatory activity in DMD, likely improving various symptoms, including muscle weakness and cardiorespiratory symptoms in addition to delaying their onset. This allows for an increased quality of life and prevents the necessity for surgical procedures, such as those for scoliosis, which is associated with DMD. Studies showed significant preservation of muscle mass in patients generally treated with 0.9 mg/kg/day of deflazacort compared to a control group. The following findings are based on clinical studies using deflazacort on a long term basis: **Effects on muscle strength** At age 16, individuals treated with long-term deflazacort had 63 ± 4% score in muscle strength compared to a mean muscle strength score of 31 ± 3% for control patients. Significant improvements in climbing stairs and rising from a supine position were also seen in patients taking deflazacort. **Effects on ambulation** Ambulation was significantly higher by 12 years of age and 18 years of age in patients taking deflazacort when compared with the control group. The control group showed a mean loss of ambulation of 2 years sooner than with deflazacort treatment. **Effects on cardiac function** Mean left ventricular ejection fraction (a measure of cardiac function) was higher in patients treated with deflazacort over the long term. Preservation of cardiac function was demonstrated by a mean difference in ejection fraction of about 7%, favoring study groups taking deflazacort over control groups. **Effects on spinal alignment** Children treated with deflazacort also significantly lowered the rate and severity of scoliosis and eliminated the need for scoliosis surgery after long-term treatment.

Deflazacort is a corticosteroid prodrug with an active metabolite, 21-deflazacort, which binds to the glucocorticoid receptor to exert anti-inflammatory and immunosuppressive effects on the body. The exact mechanism by which deflazacort exerts its therapeutic effects in patients with DMD is unknown but likely occurs via its anti-inflammatory activities.