Miglustat

Lysosomal storage diseases such as Gaucher disease and Niemann-Pick disease fall into the category of rare diseases. For a long time in the past, there was a lack of specific treatment methods, and this clinical dilemma was not broken until the emergence of small-molecule glycolipid synthesis inhibitors. As the world's first oral small-molecule glucosylceramide synthase inhibitor, miglustat can reversibly inhibit the activity of glucosylceramide synthase, reduce the biosynthesis of glycosphingolipids, and lower its storage level in organs. It has been clinically approved for the long-term treatment of adults and adolescents over 12 years old with non-neuropathic (type 1) Gaucher disease, and is also suitable for the treatment of specific types of Niemann-Pick type C patients, providing medication options for the rare disease population.

The global market size of miglustat has maintained steady growth for a long time. In 2023, the overall market size was approximately USD 420 million, with a compound annual growth rate maintained at around 6%. Due to the high R&D threshold for rare disease drugs and the limited size of the patient population, the current market pattern is highly concentrated, with original research enterprises accounting for more than 90% of the global market share. The domestic market is still in the early stage of development. With the gradual improvement of rare disease security policies, after miglustat was included in the National Medical Insurance Catalogue in 2021, the accessibility of drugs for patients has been greatly improved, and the growth rate of the domestic market has exceeded 15% for three consecutive years. At present, a number of pharmaceutical companies have laid out the R&D of generic drugs of this variety.

The original research enterprise of miglustat is Janssen-Ortho LLC, and the original research trade name is Zavesca®. Its core compound patent in the United States expired in 2022, and the expiration date of the compound patent in China is 2024. The main dosage form approved by the original research is capsules, with a specification of 100mg. It has been included in the FDA Reference Listed Drug Catalogue, and is also included in the Chinese *Catalogue of Reference Preparations for Chemical Drugs*. In terms of domestic API registration, at present, 3 enterprises have obtained CDE registration numbers for miglustat APIs, 1 of which is in A status and can be associated with preparation declaration. In terms of preparations, the original research product has been approved for marketing in China, and another 2 domestic pharmaceutical companies have submitted marketing applications for generic drugs, which are in the review stage. (Data as of May 2025, please refer to the official CDE website for the latest information)

In response to the R&D and quality control needs of miglustat, CATO provides a full set of impurity reference standards for this API, covering all categories such as synthetic process impurities and degradation impurities. Most products are in stock, and orders placed before 16:00 can be shipped on the same day. All products meet the compliance requirements of multiple regulations such as the Chinese Pharmacopoeia and FDA, and can fully meet the use needs of pharmaceutical companies in various stages such as registration declaration and quality research.