Has nirogacestat been approved for marketing in China?

As ofJuly 2025,nirogacestat (nirogacestat) has not been approved for marketing in mainland China. This means that the drug is still an overseas new drug and has no registration information with the National Food and Drug Administration (NMPA). Domestic medical institutions cannot legally purchase and clinically use it, and it does not have medical insurance coverage. Nilogastat was developed by SpringWorks Therapeutics in the United States. It is currently the only γ-secretase inhibitor that has received accelerated approval from the FDA for the treatment of desmoid tumors. It will be officially launched in the United States in November 2023, marking the entry into the era of targeted intervention in the treatment of progressive desmoid tumors.

Although China has not yet approved its marketing, this drug has attracted widespread attention from the domestic oncology medical community. Because desmoid tumors are more common in young women and have poor sensitivity to traditional chemotherapy and radiotherapy, treatment methods have long relied on surgical resection, and the postoperative recurrence rate is high. Therefore, Nirogacestat, as an alternative to non-surgical therapy, provides potential new hope for domestic patients.

At present, some patients or families will try to use the drug through "overseas drug acquisition channels", such as cross-border drug purchase or introduction through global new drug projects. However, this approach involves many risks such as legal, transportation, dosage guidance and drug safety. Domestic experts also reminded that although Nirogacestat has been approved overseas, it is not recommended for patients to use it on their own before passing local clinical trials and regulatory evaluations in China.

From the perspective of policy trends, China’s drug review is accelerating the introduction of rare disease treatment drugs. In recent years, NMPA has implemented priority review for a number of new oncology drugs. If SpringWorks can submit a Chinese registration application as soon as possible and complete the necessary local bridging trials, nirogalastat is expected to be launched in China within the next two to three years. By then, prices, medical insurance access, hospital equipment and other aspects will also be gradually clarified, bringing formal treatment options to domestic desmoid tumor patients.

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