CSPC PHARMA (01093) has announced that its independently developed injectable sirolimus (albumin-bound) has had its marketing application accepted by the National Medical Products Administration (NMPA) of China.
After review by the Center for Drug Evaluation (CDE), the product has been simultaneously included in the Breakthrough Therapy Drug Program and granted priority review and approval status. The drug is a macrolide immunosuppressant, functioning as a highly selective inhibitor of the mammalian target of rapamycin (mTOR).
Sirolimus binds with the immunophilin FKBP12 to form an immunosuppressive complex, blocking the activation of the mTOR pathway. On one hand, it inhibits cytokine-mediated activation and proliferation of T lymphocytes, halting the cell cycle progression from the G1 phase to the S phase. On the other hand, it also reduces the body's antibody production, exerting a potent immunosuppressive effect.
The product has been submitted for marketing as a Class 2.2 modified new chemical drug, with the proposed indication being unresectable locally advanced or metastatic malignant perivascular epithelioid cell tumor (PEComa). The submission is primarily based on a pivotal Phase Ib/III clinical trial.
The study enrolled patients with histologically confirmed, unresectable locally advanced or metastatic malignant PEComa who had not previously received mTOR inhibitor therapy. The Phase III pivotal clinical trial successfully met its pre-specified primary endpoints.
Compared to the control group receiving conventional treatments selected by investigators, the product demonstrated significant efficacy advantages, with data showing both statistical significance and clinical relevance, enabling rapid deep tumor response. Given the current lack of a standard treatment protocol for malignant PEComa, this product is expected to establish a new treatment standard in this field, helping to build a standardized treatment system and benefit a wide range of PEComa patients.
The product also exhibited an excellent overall safety profile, with manageable adverse reactions and a broad safety window. Its favorable benefit-risk characteristics position it as a core therapeutic agent for this rare and difficult-to-treat tumor type.
Injectable sirolimus (albumin-bound) is the first intravenous sirolimus formulation in China to receive approval for clinical research. Currently, CSPC PHARMA is advancing a pivotal Phase III clinical study evaluating the product in combination with fulvestrant for HR+/HER2- breast cancer. The company is also continuously exploring the efficacy and safety of the product as monotherapy and in combination therapies for various solid tumors, aiming to benefit more cancer patients in the future.