Huadong Medicine Co., Ltd (SHE: 000963), a Chinese pharmaceutical company, has received approval from the National Medical Products Administration (NMPA) to conduct a multi-center, parallel external control, retrospective real-world study. The study will assess the efficacy and safety of its Category 2.4 product, sirolimus, in epilepsy related to nodular sclerosis in pediatric patients under the age of 18. Sirolimus was initially approved for marketing in China for the prevention of organ rejection in patients undergoing kidney transplantation.
Tubular sclerosis complex (TSC), a rare autosomal dominant genetic disease, involves multiple systems and is characterized by the occurrence of benign hamartomas in various organs, including the skin, brain, eyes, mouth, heart, lungs, kidneys, liver, and bones. The disease’s main symptoms include epilepsy, intellectual disabilities, skin vitiligo, and facial vascular fibroids. Preclinical studies and multiple off-label clinical studies from China and Europe have indicated that sirolimus can reduce overactivation in TSC by targeting the mammalian target of rapamycin (mTOR) pathway, potentially aiding in reducing cell proliferation and growth, thus alleviating some symptoms of TSC. In terms of epilepsy, sirolimus has been shown to reduce the frequency and severity of seizures by improving neuronal function, decreasing neural excitability, reducing neuroinflammation, and enhancing neuroprotection.- Flcube.com
