Stiripentol

For refractory childhood epilepsy such as Dravet syndrome, existing conventional antiepileptic drugs are often difficult to achieve effective seizure control, and the advent of stiripentol has filled this clinical gap. As an aromatic alcohol antiepileptic drug, its mechanism of action is different from that of traditional sodium channel modulators. It can inhibit excessive discharge of the central nervous system by enhancing the mediated response of γ-aminobutyric acid type A (GABAA) receptors, and at the same time can inhibit metabolic enzymes such as CYP3A4 to increase the blood concentration of combined antiepileptic drugs. It is mainly used clinically as adjunctive therapy for epileptic seizures in patients with Dravet syndrome aged 2 years and older. It is also the first drug approved worldwide for this indication, which significantly reduces the frequency of epileptic seizures in patients and improves long-term prognosis.

The global stiripentol market size has exceeded US$280 million in 2023, with a compound annual growth rate of more than 12% in the past five years. The core growth drivers come from the improvement of the diagnosis rate of rare disease epilepsy, the expansion of the indication population, and the implementation of medical insurance policies for children's rare disease drugs in many countries. In terms of competitive landscape, the original research product still accounts for more than 90% of the global market share at present. There is no generic drug approved for marketing in China, and only a small number of enterprises have started the R&D and declaration of generic drugs. With the improvement of the standardization of diagnosis and treatment of Dravet syndrome in China, the market space of local generic products is considerable.

The original research enterprise of stiripentol is Biocodex, a French company, with the original trade name Diacomit. The core compound patents expire in 2024 and 2025 in the European Union and the United States respectively. At present, the main dosage forms approved for the original research are oral capsules (250mg, 500mg) and oral suspension (250mg/5ml). Both dosage forms have been included in the FDA Reference Listed Drug Catalog, among which the oral capsules have been included in China's *Catalog of Reference Preparations for Chemical Drugs (39th Batch)*. In China, the stiripentol API registration numbers submitted by 2 enterprises are currently in the active state. The original imported stiripentol oral capsules were approved for marketing by the NMPA in 2021, and no domestic preparation has been approved yet. (Data as of June 2025, please refer to the official website of CDE for the latest information)

CATO can provide a full set of stiripentol impurity reference standards, covering the needs of the entire research chain such as synthesis process impurities and degradation impurities. Most products support spot supply. Orders for spot products placed before 16:00 can be shipped on the same day. All products meet the requirements of pharmacopoeias of China, FDA and other national regulations, and can fully meet the compliance needs of various stages such as API R&D, quality research and consistency evaluation.