BMS' Augtyro Gains FDA Accelerated Approval for NTRK-Positive Tumors

Bristol Myers Squibb (BMS) announced the FDA's accelerated approval of Augtyro (repotrectinib), a tyrosine kinase inhibitor, for treating NTRK-positive, locally advanced or metastatic solid tumors. 

The new approval allows Augtyro to be administered to patients aged 12 and older whose disease has progressed despite initial treatments. It can also serve patients without satisfactory alternative therapies or those at risk of severe morbidity from surgical procedures, according to BMS. 

This accelerated approval by the FDA requires BMS to conduct a confirmatory study to validate Augtyro's clinical benefit for this patient group and indication. Nick Botwood, BMS's senior vice president of medical oncology, referred to Augtyro’s approval as a significant milestone. Botwood emphasized that the drug addresses an unmet medical need and demonstrates clinical value across multiple genetic markers.

Previously, no FDA-approved treatment existed for NTRK-positive cancers that had been studied in both TKI-naïve and TKI-pretreated patients across various solid tumors. Augtyro now offers these patients an effective treatment option.

The FDA approval of Augtyro is supported by data from the Phase I/II TRIDENT-1 trial, which included patients previously treated with TKIs and those who were TKI-naïve. The study encompassed 15 different cancer types.

Results from the TRIDENT-1 trial revealed that Augtyro achieved a 58% confirmed objective response rate (cORR) in TKI-naïve patients, with 15% of these being complete responses (CR). For this subgroup, the median duration of response had not yet been reached at the time of analysis. In TKI-pretreated patients, Augtyro achieved a 50% cORR, with a median response duration of 9.9 months, although no complete responses were observed.

Regarding safety, 7% of patients in the TRIDENT-1 trial discontinued Augtyro due to side effects, and 3.5% experienced fatal adverse events including pneumonia, cardiac arrest, cardiac failure, and respiratory failure.

Augtyro is designed to be taken orally and functions by inhibiting the ROS1 proto-oncogene and various tyrosine kinases. This inhibition targets fusion proteins containing ROS1 domains, thereby disrupting signaling cascades that would otherwise promote the uncontrolled proliferation of cancer cells. 

This drug was initially approved in November 2023 for ROS1-positive advanced or metastatic non-small cell lung cancer.

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