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.