CSPC Pharma (01093): Intravenous Silmitasertib (albumin-bound) granted breakthrough therapy designation in China.

CSPC PHARMA (01093) announced that the injection of sirolimus (albumin-bound) developed by the group has been granted breakthrough therapy designation by the China National Medical Products Administration for the proposed indication of monotherapy for malignant perivascular epithelioid cell tumors (PEComa). Sirolimus, also known as rapamycin, is a commonly used specific mTOR inhibitor. Previously, oral formulations of sirolimus have been approved for market, mainly for preventing organ rejection in patients receiving kidney transplants. This product uses a special technology to encapsulate sirolimus in human albumin, overcoming the limitations of oral formulations in delivering sufficient drug concentrations to target sites, enabling sirolimus to be administered by injection without the need for steroid pretreatment. Additionally, this product expands the application of sirolimus and is expected to treat a range of diseases driven by the mTOR signaling pathway. The product is currently undergoing Phase II and III clinical trials in China for the treatment of breast cancer, soft tissue sarcomas, lung cancer, kidney cancer, solid tumors, and hematological malignancies. Breast cancer is the most common malignancy among women, with approximately 2.3 million new cases worldwide. There is a significant unmet clinical need for patients with HR+/HER2- advanced breast cancer who have failed CDK4/6 inhibitor therapy. This product, in combination with endocrine therapy, is the first mTOR inhibitor in China to conduct clinical trials in HR+/HER2- advanced breast cancer patients resistant to CDK4/6 inhibitors. The group has submitted an application to regulatory authorities for a key Phase III clinical trial communication. There is currently no standard treatment for malignant PEComa in China, creating an unmet clinical need. Clinical data has confirmed that this product's monotherapy is significantly more effective than historical data, and it has now entered a crucial Phase III enrollment stage. The breakthrough therapy designation granted in this announcement will help accelerate the development progress of this product, making it potentially the first standard treatment for this indication in China.