Eli Lilly and Company — Retevmo FDA Review (NDA/BLA 213246, 2026)
Eli Lilly and Company · 2026 · FDA Review (Drugs@FDA) · company profile →
Report summary
Eli Lilly's Retevmo (selpercatinib) received accelerated FDA approval on September 21, 2022, for RET-fusion positive cancers, including NSCLC, medullary thyroid cancer, and other solid tumors. This approval, based on the LIBRETTO-001 trial, demonstrated significant overall response rates and duration of response, addressing an unmet medical need. The drug's safety profile, detailed across various populations, highlights key concerns like hepatotoxicity, ILD/pneumonitis, and hypertension, with management recommendations provided. Pharmacokinetic analyses support the proposed dosing regimen, and ongoing confirmatory trials are required to verify clinical benefit. The review also includes a new tumor-agnostic indication under accelerated approval and the transition of the NSCLC indication to traditional approval.
Key findings — cited to the page
| patients with Grade >=3 selected TEAEs | 29 (64.4%) | LIBRETTO-001 · p.136 |
| Grade >=3 TEAEs | 29 (64.4%) | LIBRETTO-001 · p.135 |
| Grade >=3 TEAEs | 572 (71.9%) | LIBRETTO-001 · p.135 |
| Grade >=3 TEAEs related to selpercatinib | 307 (38.6%) | LIBRETTO-001 · p.135 |
| patients with Grade >=3 selected TEAEs | 572 (71.9%) | LIBRETTO-001 · p.136 |
| Grade >=3 TEAEs related to selpercatinib | 17 (37.8%) | LIBRETTO-001 · p.135 |
| Most common Grade 3 or 4 laboratory abnormalities (≥5%) | decreased lymphocytes, increased alanine aminotransferase (ALT), increased aspartate aminotransferase (AST), decreased sodium, and decreased calcium | RETEVMO (selpercatinib) · p.11 |
| Grade 5 TEAEs | 3 (7%) | LIBRETTO-001 · p.129 |
| Decreased sodium (Grades 3-4) in patients receiving RETEVMO | 11 % | LIBRETTO-001 · p.144 |
| Increased creatinine (Grades 3-4) in patients receiving RETEVMO | 2.4 % | LIBRETTO-001 · p.144 |
| Decreased sodium (Grades 1-4) in patients receiving RETEVMO | 42 % | LIBRETTO-001 · p.144 |
| Increased alkaline phosphatase (Grades 1-4) in patients receiving RETEVMO | 40 % | LIBRETTO-001 · p.144 |
Source: Eli Lilly and 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 →