Eliglustat

As a representative drug of glucosylceramide synthase inhibitors, eliglustat inhibits the synthesis of glucosylceramide and reduces the pathological deposition of glycosphingolipids in organ tissues, and is currently an important oral option for long-term maintenance treatment of type 1 Gaucher disease. Compared with the traditional enzyme replacement therapy which requires repeated intravenous infusion, eliglustat is an oral dosage form without injection, which greatly improves the medication compliance of patients. It is especially suitable for adult patients with mild to moderate type 1 Gaucher disease, and can effectively improve relevant clinical symptoms such as hepatosplenomegaly, anemia, thrombocytopenia and bone disease.

The global prevalence of Gaucher disease is about 1 per 100,000 people. As a rare disease, the global market size of eliglustat has long maintained at around US$300 million. In recent years, as more countries have included it in their rare disease security catalogs, the annual compound market growth rate has remained at around 4%. In terms of the domestic market, only the original research product has been approved for marketing at present, and no domestic generic drug has entered the market, so there is still much room for improvement in clinical accessibility. With the gradual improvement of the drug security policy for rare diseases, the market penetration space of this variety is expected to be further expanded in the future.

The original research enterprise of eliglustat is Pfizer, and the original research trade name is Cerdelga. Its core compound patent expires in 2027 in the United States, and the compound patent in China expires in 2024. At present, the main dosage form of the original research marketed product is hard capsule, with two specifications of 84mg and 170mg. It has been included in the FDA Reference Listed Drug Catalog, and also included in the *Catalog of Reference Preparations for Chemical Drugs (Third Batch)* of China. There is no valid API registration number related to eliglustat on the domestic API registration platform, and only the original research oral preparation was approved for marketing in China in 2021 through the priority review channel. (Data as of June 2025, please refer to the official website of CDE for the latest information)

CATO can provide a full set of impurity reference standards for eliglustat, most of which are available from stock. For in-stock products, orders placed before 16:00 can be shipped on the same day. All reference standards comply with the compliance requirements of multiple regulatory systems such as the Chinese Pharmacopoeia and FDA, and can fully meet the reference standard use needs of the whole process of API R&D, quality research and declaration.