Eli Lilly and Company today announced that the European Medicines Agency (EMA) has granted marketing authorisation (MA) for Retsevmo® (selpercatinib 40 mg & 80 mg oral capsules) as a first-line monotherapy indicated for the treatment of adults and adolescents 12 years and older with advanced RET-mutant medullary thyroid cancer. Selpercatinib is a selective and potent RET kinase inhibitor with central nervous system activity that has previously been approved for the treatment of advanced medullary thyroid cancer in patients aged 12 years and older who had previously been treated with the cancer medicines cabozantinib or vandetanib or both. RET mutations in people with advanced medullary thyroid cancer are associated with more aggressive disease and poorer outcomes for patients.[1]

This approval is based on the Phase 1/2 LIBRETTO-001 trial, which is the largest clinical trial of patients with RET-driven cancers treated with a RET inhibitor. LIBRETTO-001 is a multi-centre (16 countries, 89 sites), open-label, single-arm clinical study and included a Phase 1 dose escalation and Phase 2 dose expansion. The primary efficacy outcome measure of the Phase 2 was overall response rate (ORR) according to RECIST v1.1 as evaluated by a blinded independent review committee (IRC). Secondary efficacy outcomes included duration of response (DOR), progression free survival (PFS) and overall survival (OS). Results demonstrated:

Abbreviations: n. number of patients; NE, not estimable; CI, confidence intervals

In the safety population (all LIBRETTO-001 patients that received at least one dose of selpercatinib, N=796), the most common serious adverse events were abdominal pain (2.5%), hypersensitivity (2.0%), diarrhoea (1.9%), alanine aminotransferase (ALT) increased (1.5%) and aspartate aminotransferase (AST) increased (1.5%). Eight percent of patients were discontinued due to an adverse event regardless of attribution.

Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials.

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[1] National Institute for Health and Care Excellence (NICE). Selpercatinib for treating advanced thyroid cancer with RET alterations. Technology Appraisal Guidance (TA742) published November 2021. https://www.nice.org.uk/guidance/ta742/chapter/3-Committee-discussion Last accessed 12 September 2022.