FDA grants accelerated approval for Bristol Myers Squibb’s Augtyro for NTRK-positive tumors

Bristol Myers Squibb (NYSE: BMY) has received accelerated FDA approval for Augtyro (repotrectinib), a treatment designed for patients 12 years and older with NTRK gene fusion-positive solid tumors. This approval, pivotal for patients with advanced stages of cancer where surgical options could cause severe morbidity, marks a significant advancement in targeted cancer therapy.

Clinical Trial Insights and Efficacy

The approval of Augtyro is based on the outcomes of the Phase 1/2 TRIDENT-1 study, which demonstrated promising results in both TKI-naïve and TKI-pretreated patients. In the trial, TKI-naïve patients showed a confirmed objective response rate (cORR) of 58%, with 43% experiencing partial responses and 15% achieving complete responses. Notably, 83% of these responses were maintained at one year. For TKI-pretreated patients, the cORR was 50%, with a median duration of response (mDOR) of 9.9 months, highlighting the drug’s effectiveness even in previously treated populations.

This indication has been granted under accelerated approval, contingent upon further verification in confirmatory trials, focusing on the overall response rate and duration of response.

FDA grants accelerated approval for Bristol Myers Squibb’s Augtyro for the treatment of NTRK gene fusion-positive solid tumors.

Statements from Medical Experts and Advocates

Alexander Drilon, MD, the TRIDENT-1 global trial lead and Chief of the Early Drug Development Service at Memorial Sloan Kettering Cancer Center, emphasized the importance of this new treatment option: “NTRK fusion-positive tumors can present challenges in the clinical setting, which is why it is important that we have additional treatment options for these patients.”

Nick Botwood, senior vice president of Medical Oncology at Bristol Myers Squibb, reflected on the broader impact of this approval: “Today’s FDA approval of Augtyro for patients with NTRK-positive tumors adds to its indication in ROS1-positive NSCLC, showing its clinical value for more people across multiple genetic markers.”

Susan Spinosa, president and patient co-founder of NTRKers, a patient advocacy group, also commented on the emotional and practical benefits of this new treatment: “It’s exciting to know that there’s a new targeted therapy option for patients with NTRK-positive gene fusions, as this may offer hope to patients and their loved ones navigating this difficult journey.”

Safety Profile and Administration

Augtyro is administered orally, starting with a dose of 160 mg once daily for 14 days, followed by 160 mg twice daily. It’s associated with a range of side effects, including central nervous system effects, interstitial lung disease, hepatotoxicity, and others, necessitating careful monitoring and management.