Eli Lilly's Tumor-Agnostic RET Inhibitor Retevmo Receives FDA Accelerated Approval for Pediatric Patients with RET-Altered Cancers

May 31, 2024 07:30 CST Updated 07:31

Eli Lilly

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▎WuXiEdited by Kant Content Team

The U.S. FDA recently announced the accelerated approval of Retevmo (selpercatinib), developed by Eli Lilly and Company, for the treatment of pediatric patients aged 2 years and older with indications including: those requiring systemic therapy.RETMutation-related advanced or metastatic medullary thyroid carcinoma (MTC); carryingRETAdvanced or metastatic thyroid cancer with gene fusion and ineffective radioactive iodine treatment; carryingRETGene fusion, locally advanced or metastatic solid tumors that have progressed after receiving systemic therapy or lack satisfactory alternative treatment options. The press release noted that this isFDA Approves for the First Time for Children Under 12RETTargeted Therapy for Pediatric Patients with Genetic Mutations.Retevmo had previously received accelerated approval for thyroid cancer indications in adults and children aged 12 and above, as well as for solid tumor indications in adults.

The clinical trial named LIBRETTO-121 evaluated the efficacy of Reztevmo in pediatric and adolescent patients. This trial was an international, single-arm, multi-cohort study. The primary efficacy population included 25 patients aged 2 to 20 years with locally advanced or metastatic RET-activated solid tumors who did not respond to existing therapies or had no standard treatment options.

The primary efficacy outcome measures are the confirmed Overall Response Rate (ORR) and Duration of Response (DOR), as assessed by a blinded independent review committee.The confirmed ORR (RECIST 1.1) was 48% (95% CI: 28, 69). The median DOR has not yet been reached, and 92% of patients who achieved response maintained the response at 12 months.InRETMutant MTC (n=14; ORR=43%) andRETDurable responses were observed in pediatric and adolescent patients with fusion thyroid cancer (n=10; ORR=60%).

The most common adverse reactions (≥25%) include musculoskeletal pain, diarrhea, headache, nausea, vomiting, COVID-19, abdominal pain, fatigue, fever, and bleeding. The most common Grade 3 or 4 laboratory abnormalities (≥5%) were decreased calcium, decreased hemoglobin, and decreased neutrophils.

Retevmo is a potent RET kinase inhibitor. It received accelerated approval from the U.S. FDA in 2020 for the treatment of three types of cancer: non-small cell lung cancer (NSCLC), medullary thyroid carcinoma (MTC), and thyroid cancer.These patients' tumorsRETGene fusion or mutation occurs.

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References:

[1] FDA grants accelerated approval to selpercatinib for pediatric patients two years and older with RET-altered metastatic thyroid cancer or solid tumors. Retrieved May 30, 2024, from https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-selpercatinib-pediatric-patients-two-years-and-older-ret-altered

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