In this article
FDA Approval for RET-Fused Solid Tumors
November 2022: Selpercatinib (Retevmo, Eli Lilly and Company) received accelerated approval from the Food and Drug Administration to treat adult patients with RET-fused solid tumours that are locally or metastatically advanced, have progressed on or after prior systemic therapy, or who have no other effective treatment options.
LIBRETTO-001 Clinical Trial and Study Design
In LIBRETTO-001 (NCT03157128), a multicenter, open-label, multi-cohort trial evaluating 41 patients with RET fusion-positive tumours (other than non-small cell lung cancer and thyroid cancer) who had disease progression during or after prior systemic treatment or who lacked adequate alternative treatment options, efficacy was shown. Data from 343 patients with RET fusion-positive NSCLC and thyroid cancer who were included in the same trial and previously mentioned in the product labelling provided support for the efficacy evaluation. Patients were given selpercatinib until the disease progressed or the side effects became intolerable.
Efficacy and Objective Response Results
A Blinded Independent Review Committee chose the overall response rate (ORR) and duration of response (DOR) as the major efficacy measures (BIRC). The ORR among the 41 individuals who could be evaluated was 44% (95% CI: 28, 60), and the DOR was 24.5 months (95% CI: 9.2, not estimable). Pancreatic adenocarcinoma, colorectal, salivary, unknown primary, breast, soft tissue sarcoma, bronchial carcinoid, ovarian, small intestine, and cholangiocarcinoma were among the tumour types that responded.
Patient Demographics and Tumor Types
The patients’ average age was 50. (range 21 to 85). 54% of the population was female, 68% were White, 24% were Asian, 4.9% were Black, 7% were Hispanic/Latino, 95% had an ECOG performance rating of 0 or 1, and 95% had metastatic disease. 37 patients (90%) had previously had systemic therapy (median 2; range 0–9; 32% had previously received 3 or more). Pancreatic (27%), colorectal (24%), salivary (10%), and unknown primary (7%) malignancies were the most prevalent. Using NGS, 97.6% of patients had RET fusion-positive status found, while 2.4% did so using FISH.
Adverse Reactions and Recommended Dosage
Edema, diarrhoea, exhaustion, dry mouth, hypertension, abdominal discomfort, constipation, rash, nausea, and headache were the most frequent side events among patients (25%).
Based on body weight, the suggested dose of selpercatinib is:
120 milligrammes twice daily if you weigh less than 50 kg.
more than 50 kg: 160 mg taken orally twice each day.
View full prescribing information for Retevmo.
How CancerFax Helps
CancerFax is a specialist cancer access and patient-navigation platform. We help patients and families understand their options, organise medical records, coordinate hospital communication, and support cross-border treatment planning where appropriate.
We help collect and organise reports, scans, pathology, biomarker results, and treatment history for structured case review.
We communicate with hospitals or trial teams to assess whether a case may be suitable for further screening.
We support appointment coordination, document submission, translation, and direct communication with international departments.
For international patients, we help with practical coordination — travel planning, hospital admission guidance, and local support.
If this option is not suitable, we help explore other relevant treatments, clinical trials, or advanced care pathways.
From inquiry through to follow-up, our coordinators provide a single point of contact for the family.
CancerFax does not guarantee treatment access, eligibility, or clinical outcome. Our role is to help patients access accurate information, structured review, and appropriate specialist pathways.
About Susan Hau
Susan Hau is a distinguished researcher in the field of cancer cell therapy, with a particular focus on T cell-based approaches and cancer vaccines. Her work spans several innovative treatment modalities, including CAR T-cell therapy, TIL (Tumor-Infiltrating Lymphocyte) therapy, and NK (Natural Killer) cell therapy. Hau's expertise lies in cancer cell biolo…
✓ Reviewed for medical accuracy by the CancerFax review panel.
Medical Disclaimer
This article is for educational purposes only and should not replace medical advice, diagnosis, or treatment from a qualified oncology specialist. Every patient's case is different. Treatment decisions should always be made after a review of complete medical records by the treating medical team.
Treatment availability, eligibility, timelines, and access can change. Any clinical trial participation depends on detailed review and approval by the trial hospital or investigator.