Mitapivat

Familial chylomicronemia syndrome (FCS) is an extremely rare autosomal recessive genetic disease. Due to the functional defect of lipoprotein lipase, patients have long-term excessive plasma triglyceride levels, which are highly likely to induce recurrent pancreatitis and even organ damage. For a long time, there has been a lack of targeted treatment methods. Volanesorsen is a novel apolipoprotein CIII (apoCIII) antisense oligonucleotide drug. It reduces the level of apoCIII in plasma by targeted inhibition of hepatic apoCIII synthesis, thereby significantly reducing the production of triglyceride-rich lipoproteins. Currently, it has been approved for adjuvant treatment of adult FCS patients, filling the clinical gap in this rare disease field.

The global prevalence of FCS is approximately one in a million. The corresponding applicable population base of volanesorsen is small but the clinical demand is rigid. In 2023, the global market size of volanesorsen was approximately USD 210 million, with a compound annual growth rate maintained at around 18%. At present, the global market of this drug is still in the stage of exclusive supply by the original research manufacturer. Due to the high R&D cost of rare disease drugs and narrow applicable population, generic drug enterprises generally have low willingness to deploy, and there is no public generic drug declaration information in the short term.

Volanesorsen was originally developed jointly by Ionis Pharmaceuticals and Akcea Therapeutics, with the original brand name Waylivra. Its core compound patent in the United States will expire in 2034, and the core patent in Europe will expire in 2035. The currently approved main dosage form is subcutaneous injection, with a specification of 200mg/1.5mL. The original preparation has been included in the FDA Reference Listed Drug Catalog. As of July 2025, the Center for Drug Evaluation (CDE) of the National Medical Products Administration of China has no publicly announced API registration number for volanesorsen, and no preparation of this variety has been approved for marketing in China. (Data as of July 2025, please refer to the official CDE website for the latest information)

In response to the R&D and quality control requirements of volanesorsen, CATO can provide a full set of impurity reference standards for this API. Most products support spot supply. Spot orders placed before 16:00 can be shipped on the same day. All products comply with the compliance requirements of multiple regulatory systems such as Chinese Pharmacopoeia and FDA, and can provide stable reference standard support for pharmaceutical enterprises in impurity research, quality standard establishment and other links.