Chemistry and Pharmacology of Deflazacort: A Novel Bioactive Compound for the Treatment of Duchenne Muscular Dystrophy-A Mini Review

Abstract

Objective: Firstly, Deflazacort was synthesized in 1969 and has the structural similarity with cortisol. The present review is an attempt to summarize the updated information related to chemistry and pharmacology of Deflazacort. Development: Deflazacort a synthetic compound synthesized by derivatization in the chemical structure of prednisolone with an aim to improve its potency. Deflazacort is under global development with Marathon Pharmaceuticals, was approved by the U.S. Food and Drug Administration in year 2017 to treat patients with Duchenne muscular dystrophy (DMD). Chemistry and Pharmacology: DMD is one of the rare and genetic disorders with the symptoms of progressive degeneration of muscle tissue. Chemically, it is deacetylated at position 21 to produce active metabolite 21-desacetyl deflazacort (21-desDFZ), which acts through the glucocorticoid receptor. The predominant side effects are cushingoid appearance, increased appetite, upper respiratory tract infection (URTI), pollakiuria, hirsutism, and nasopharyngitis. Conclusion: The present article summarizes the available updated information and work done so far on Deflazacort with special emphasis on its chemistry, pharmacology with detailed mechanism of action, pharmacodynamics, pharmacokinetics, metabolism and clinical trials etc.