UK pharmaceutical company AstraZeneca (AZ; NASDAQ: AZN) announced last week that it has received an indication extension approval from Japan’s Ministry of Health, Labour and Welfare (MHLW) for its orphan drug Soliris (eculizumab). The drug is now approved as a treatment for generalized myasthenia gravis (gMG) in pediatric patients who are AChR antibody-positive and unresponsive to immunoglobulin or plasmapheresis therapies. This marks Soliris as the only targeted therapy indicated for minors suffering from this autoimmune neuromuscular disease in Japan.
Supporting Clinical Trial Results
The decision by the MHLW is supported by results from a late-stage trial that demonstrated significant improvement in disease severity and function in pediatric patients with refractory gMG, meeting the primary endpoint. The efficacy and safety profiles observed in these trials were consistent with those previously observed in adult patients, further validating Soliris as a treatment option.
Global Registration and Biosimilar Development
Soliris is registered in various markets, including Argentina, Brazil, China, the European Union (EU), and the US. According to Fineline Info & Tech data, Russia-based rare disease specialist Generium manufactures a biosimilar version of Soliris under the brand name Elizaria, indicating the global reach and impact of this therapy.-Fineline Info & Tech
