Deflazacort

Duchenne muscular dystrophy (DMD), a disease of high concern in the field of rare diseases, has long lacked long-term intervention regimens with both efficacy and safety. The advent of deflazacort has filled this clinical gap. As a third-generation glucocorticoid drug, deflazacort delays the muscle degeneration process of DMD patients by inhibiting inflammatory responses and reducing the activation of immune cells. Compared with traditional prednisone, it has a significantly lower incidence of adverse reactions such as water and sodium retention and weight gain. It can be used for long-term maintenance treatment of DMD patients of all age groups over 2 years old, and is also suitable for anti-inflammatory treatment of autoimmune diseases and allergic diseases.

Against the background of the expansion of the global rare disease drug market, the global market size of deflazacort was approximately USD 480 million in 2023, with a compound annual growth rate of 12.7% in the recent 3 years. The growth momentum mainly comes from the improvement of global DMD diagnosis rate and the implementation of rare disease medical insurance policies in various regions. The competitive landscape presents obvious stratification: the original research product occupies the main share of mature markets such as the United States and the European Union, while generic drugs are rapidly penetrating in emerging markets. At present, 3 enterprises in China have obtained the approval documents for deflazacort preparations, the supply of active pharmaceutical ingredients is still in short supply, and the variety has not been included in centralized procurement, leaving sufficient market expansion space.

The original research enterprise of deflazacort is PTC Therapeutics, with the original trade name Emflaza. Its core compound patent in the United States expires in 2027, and the compound patent in China expired in 2024. The dosage form approved for the original research product is tablet, with specifications including 6mg, 18mg, 30mg and 36mg. It has been included in the FDA Reference Listed Drug Catalog, but has not been included in the *Catalog of Chemical Drugs* in China. At present, there are 5 registration numbers of deflazacort active pharmaceutical ingredients on the domestic API registration platform, 3 of which are in Class A status. In addition, deflazacort preparations from 6 enterprises have been approved for marketing by the National Medical Products Administration. (Data as of May 2025, please refer to the official website of CDE for the latest information)

CATO can provide a full set of impurity reference standards for deflazacort, covering all quality control nodes such as synthesis process impurities and degradation impurities. Most products are regularly in stock, and orders placed before 16:00 can be shipped on the same day. All products meet the requirements of multiple regulatory systems such as the Chinese Pharmacopoeia and FDA, and can meet the reference standard needs of pharmaceutical enterprises in the whole process from API R&D to registration and declaration.