Is the drug nirogacestat available in China?

Nirogacestat(nirogacestat) has not yet received official marketing approval in mainland China, so the drug cannot be purchased normally in domestic hospitals or pharmacy systems. As a new type of γ-secretase inhibitor, nilogalstat is currently approved for marketing in some countries such as the United States and is used to treat specific types of soft tissue tumors, especially patients with progressive desmoid tumors. Since it has been approved by the FDA overseas and has been included in individual precision medicine medication systems, some patients will choose to try to obtain the drug through overseas medical channels, cross-border drug purchase platforms or third-party medical service agencies.

However, there are many uncertainties in this type of cross-border drug use in terms of legal compliance, safety and transportation channels. Its use has not yet been approved in China, which means that the drug has not undergone quality review and clinical data evaluation by the State Food and Drug Administration, and it is difficult for doctors to give specific prescription suggestions based on domestic guidelines. Therefore, patients must consult an oncologist or rare disease doctor before trying to purchase or use nilogalstat and weigh the possible efficacy and risk of adverse reactions. In addition, there are practical problems such as high payment exchange rate, high transportation costs, inability to enjoy medical insurance reimbursement, and lack of after-sales service when purchasing unlisted drugs overseas, which also increases the financial and medication pressure on patients.

From the perspective of domestic drug review trends, with the development of precision medicine, innovative drugs targetingγ-secretase may gradually enter the domestic registration process. In the future, nirogalistat is expected to be included in the priority review list of the National Center for Drug Evaluation. If the clinical research data is further improved, its prospects for listing in mainland China are still promising. For patients who are in urgent need of this drug, they can currently pay attention to whether there are relevant clinical trials in China that are open for recruitment, and obtain opportunities for compliant drug use by participating in research projects.

Reference materials:https://www.drugs.com/mtm/nirogacestat.html