A new drug for fatal brain tumors receives accelerated approval from the FDA, giving China Resources Sanjiu a leading position in localization.

August 12, 2025  Source: drugdu 40

On August 6th, the U.S. Food and Drug Administration (FDA) granted accelerated approval to Jazz Pharmaceuticals' (Jazz Pharmaceuticals) oral drug, Dordaviprone (trade name: Modeyso). This is the world's first targeted drug for diffuse midline glioma (DMG) with the H3 K27M mutation. It is indicated for patients aged one year and older who have failed first-line therapy. Because these aggressive brain tumors are located in surgically restricted areas such as the brainstem and spinal cord, traditional treatment relies solely on palliative radiotherapy, resulting in a median survival of only approximately 12 months. This approval breaks a 25-year stalemate in this field, which had been characterized by the lack of a systemic treatment.

"/Image source: Jazz Pharmaceuticals official website

During its development, the US biotechnology company Oncoceutics was the first to discover the molecule's dual anti-cancer mechanism: inhibiting the RAS signaling pathway by blocking the tumor-promoting DRD2 receptor while simultaneously activating the mitochondrial protease ClpP to degrade cancer cell energy metabolism proteins. This revolutionary target has attracted increasing investment from the capital market: from Chimerix's acquisition of Oncoceutics for $500 million in January 2021 to Jazz Pharmaceuticals' hefty $935 million premium in March 2025. Even more strategically, China Resources Sanjiu reached an agreement with Oncoceutics as early as December 2020, securing exclusive development, production, and commercialization rights for the drug in Greater China, securing early access to the Chinese market.

Dordaviprone clinical data confirms its breakthrough efficacy. The results of 50 relapsed patients in 5 single-arm studies showed that 22% of the subjects achieved significant tumor regression; the median duration of remission was 10.3 months, and 73% of responders maintained remission for at least half a year. Even more promising is the Phase III ACTION study conducted in newly treated patients. After combined radiotherapy, the median survival time increased to 21.7 months, almost doubling the historical data.

And the layout of the Chinese market has been substantially advanced. In 2023, China Resources Sanjiu obtained approval from the National Medical Products Administration to initiate a clinical trial of recurrent/progressive DMG. In November of the same year, a Phase I study was conducted at centers such as Beijing Tiantan Hospital and Shanghai Huashan Hospital, with plans to enroll 58 patients. With the help of China Resources Sanjiu's mature in-hospital channel network, local patients are expected to have access to this innovative therapy earlier.

When Jazz Pharmaceuticals advances its Phase III confirmatory trial, expected to be completed in 2026, Dordaviprone could potentially elevate from last-line treatment to first-line standard. By repairing the epigenetic breakdown caused by the H3K27M mutation, Dordaviprone not only opens the door to life for DMG patients but also has the potential to expand to other H3 K27M-related tumors. China Resources Sanjiu's five-year strategy is bringing Chinese patients to the forefront of global precision medicine—a groundbreaking journey that began in the laboratory and will ultimately make surgical no-go zones a thing of the past.

https://news.yaozh.com/archive/45882.html

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