Bristol-Myers Squibb Company — Augtyro FDA Review (NDA/BLA 218213, 2024)
Bristol-Myers Squibb Company · 2024 · FDA Review (Drugs@FDA) · company profile →
Report summary
The FDA granted accelerated approval to Bristol-Myers Squibb's Augtyro (repotrectinib) capsules on June 13, 2024, for adult and pediatric patients (12+) with locally advanced or metastatic NTRK gene fusion-positive solid tumors that have progressed or lack alternative therapy. This approval, contingent on confirmatory trials, is based on the TRIDENT-1 study, which demonstrated clinically meaningful overall response rates and durability in both TKI-naïve and TKI-pretreated patients. The drug's safety profile, detailed across multiple reports, highlights common adverse reactions like dizziness and peripheral neuropathy, and serious risks including CNS effects, ILD/pneumonitis, hepatotoxicity, and skeletal fractures. Postmarketing requirements include further safety and efficacy verification, particularly regarding long-term effects in pediatric patients. The FDA's review, including clinical pharmacology and statistical analyses, supported the recommended dosing regimen, concluding that benefits outweigh risks for this rare cancer.
Key findings — cited to the page
| Grade 3 myalgia | 0.7 % | TRIDENT-1 · p.20 |
| adverse reactions: dizziness (all grades) | 65 % | TRIDENT-1 · p.23 |
| Grade 4 mood disorders (mania) | 0.2 % | TRIDENT-1 · p.19 |
| Grade 3 or 4 increased AST | 2.9 % | TRIDENT-1 · p.19 |
| Grade 3 or 4 hyperuricemia | 0.7 % | TRIDENT-1 · p.20 |
| Grade 3 or 4 CNS adverse events | 4.5 % | TRIDENT-1 · p.18 |
| Grade 3 ataxia | 0.5 % | TRIDENT-1 · p.18 |
| Grade 3 cognitive impairment | 0.9 % | TRIDENT-1 · p.18 |
| Grade 3 ILD/pneumonitis | 1.2 % | TRIDENT-1 · p.19 |
| Grade 3 or 4 increased ALT | 3.3 % | TRIDENT-1 · p.19 |
| Grade 3 dizziness | 2.8 % | TRIDENT-1 · p.18 |
| adverse reactions: dizziness (Grade 3 or 4) | 2.8 % | TRIDENT-1 · p.23 |
Source: Bristol-Myers Squibb Company ↗ · License: US Government public domain. Summary and findings are extracted from the published document; every figure cites its page. Informational only — not investment advice. Explore the Oncology library →