An old glucocorticoid drug for the treatment of Duchenne muscular dystrophy-Deflazacort

Deflazacort is a glucocorticoid drug. It was first developed by Nathansohn of the Italian company Lepitit S.P.A. in the 1960s. It was first launched in Italy and Belgium by the French company Aventis in 1986, and finally became widely available in Europe. It is widely used in some diseases that require glucocorticoid treatment. Among them, the drug is particularly effective in treating Duchenne muscular dystrophy. Today I will introduce to you the magic of the old glucocorticoid drug deflazacort.

Duchenne muscular dystrophy (DMD) is the most common X-linked recessive hereditary muscle degenerative disease. The main clinical features are progressive muscle weakness and pseudohypertrophy of the gastrocnemius muscle. Symptoms usually begin at the age of 3 to 5 years, and the ability to walk independently is lost at the age of 10 to 12 years. People often die from cardiopulmonary failure before the age of 30.

Deflazacort is a prodrug, which is rapidly absorbed and metabolized to the active product 21-hydroxydeflazacort after oral administration. It is mainly excreted by the kidneys, and the peak time is about 1 hour in a fasting state. When used in combination with certain drugs, its efficacy will be affected. For example, when used in combination with rifampicin, the peak plasma concentration of 21-hydroxydesflucorte may be significantly reduced.

During the approval process, deflazacort was granted a series of preferential treatment by the FDA, and was granted expedited review status and adopted the priority approval process. In addition, deflazacort was granted orphan drug status by the FDA. Deflazacort was also granted "Priority Approval for Drugs for the Treatment of Rare Pediatric Diseases" by the FDA. This is the ninth pediatric drug to receive such treatment since the FDA launched this program in 2007.

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