Ataluren

For Duchenne muscular dystrophy (DMD), which has a relatively high prevalence among neuromuscular diseases, patients with nonsense mutation type have long lacked targeted treatment regimens, and this dilemma was not broken until the marketing of drugs related to small molecule read-through therapy. Ataluren is a dystrophin protein read-through agent. Its mechanism of action is to bind to ribosomes, enabling ribosomes to skip the premature termination nonsense mutation on mRNA during translation, and synthesize full-length dystrophin protein with normal functions. It is clinically used for patients with Duchenne muscular dystrophy aged 2 years and above who have been confirmed to have nonsense mutations in the dystrophin gene through genetic testing. It is the world's first small molecule drug approved for the treatment of this subtype of DMD, which can significantly delay the decline of motor function in patients and reduce the risk of respiratory and cardiac complications.

At present, the global market size of DMD therapeutic drugs has exceeded 3 billion US dollars, among which therapeutic drugs targeting the nonsense mutation subtype account for about 12%, and the compound annual growth rate remains above 8%. Since DMD is a rare disease, drug R&D is relatively difficult. Currently, the market competition pattern of ataluren worldwide is highly concentrated, and only a few countries have relevant generic drug application progress. At present, no generic ataluren has been approved for marketing in China, and clinical demand mainly relies on the supply of imported original research drugs, so there is still large room for improvement in market accessibility.

The original research enterprise of ataluren is PTC Therapeutics, and the original research trade name is Translarna. Its core compound patent expires in 2024 in Europe, and the core patent in the United States expires in 2027. The main dosage form approved by the original research is oral granule, with specifications of 125mg, 250mg and 1000mg. This dosage form has been included in the FDA Reference Listed Drug Catalog, and has not yet been included in the *List of Chemical Drugs* in China. Up to now, there is no DMF registration information of ataluren active pharmaceutical ingredient in China. The original research preparation became accessible through the policy of Hainan Boao Lecheng Pioneer Zone in 2020, and has not been officially approved for marketing by the National Medical Products Administration. (Data as of July 2025, please refer to the official website of CDE for the latest information)

CATO can provide a full set of ataluren impurity reference standards. Most products support spot supply. For in-stock products, orders placed before 16:00 can be shipped on the same day. All reference standards comply with the regulatory requirements of the Chinese Pharmacopoeia, FDA and other regulations, and can provide stable reference standard support for active pharmaceutical ingredient R&D, quality research and consistency evaluation work.