FDA Grants Full Approval to Retevmo for RET Fusion-Positive Metastatic Solid Tumors
Approval expands precision medicine option for adults and children with advanced cancers
The U.S. Food and Drug Administration (FDA) has granted traditional approval to Retevmo (selpercatinib) for the treatment of adults and children 2 years of age and older with locally advanced or metastatic RET fusion-positive solid tumors that have progressed following prior treatment or have no satisfactory alternative treatment options.
The approval converts the drug’s previous accelerated approvals—granted for adults in 2022 and pediatric patients in 2024—into full approval based on additional clinical evidence demonstrating durable responses across multiple tumor types.
A Precision Medicine Approach
Unlike treatments aimed at a specific type of cancer, Retevmo is a tissue-agnostic therapy, meaning eligibility is based on the presence of a RET gene fusion rather than where the cancer originated.
Patients must have a RET gene fusion confirmed through an FDA-approved diagnostic test before receiving treatment.
Clinical responses were observed in a broad range of cancers, including colorectal, pancreatic, cholangiocarcinoma (bile duct cancer), breast, ovarian, and salivary gland cancers, soft tissue sarcoma, neuroendocrine tumors, and several other rare cancers.
Clinical Trial Results
The approval is supported by results from the LIBRETTO-001 and LIBRETTO-121 clinical trials.
Among 75 patients with RET fusion-positive solid tumors other than lung and thyroid cancer there was a 47% overall response rate with a median response duration of 24.5 months.
Responses were also observed in pediatric patients with several rare RET fusion-positive tumors.
Important Safety Information
Healthcare providers should monitor patients for several potentially serious adverse reactions, including liver toxicity, interstitial lung disease/pneumonitis, hypertension, QT interval prolongation, hemorrhage, hypersensitivity reactions, tumor lysis syndrome, impaired wound healing, and hypothyroidism.
Additional precautions apply to pediatric patients because of the risk of slipped capital femoral epiphysis.
Regulatory Highlights
FDA completed its review two months ahead of the agency’s target decision date, using the Assessment Aid review process designed to streamline evaluation.
Retevmo also received Orphan Drug Designation for this tissue-agnostic indication, recognizing its potential benefit for patients with rare cancers.
Why It Matters
The approval represents another milestone in the shift toward precision oncology, where treatment decisions are increasingly guided by a tumor’s genetic characteristics rather than its location in the body.
For patients with rare RET fusion-positive cancers, many of whom have limited treatment options. The expanded approval provides access to a targeted therapy that has demonstrated durable responses across multiple tumor types.
As genomic testing becomes more widely integrated into cancer care, approvals like this underscore the growing importance of comprehensive molecular profiling in identifying patients who may benefit from precision medicines.
Regulatory Insight: When Cancer Treatment Is Based on Genetics, Not Location
Tissue-Agnostic Medicine Comes of Age
For decades, cancer drugs were approved to treat tumors based on where the cancer originated – lung, breast, colon, pancreas, and so on.
Retevmo reflects a different approach.
Patients qualify because their tumors contain a RET gene fusion, regardless of where the cancer began. This is known as a tissue-agnostic approval, one of the fastest-growing areas of oncology regulation.
Instead of asking Where is the cancer?
Regulators increasingly ask What genetic mutation is driving it?
As genomic testing becomes more common, FDA approvals are increasingly based on molecular biomarkers rather than tumor location.
Regulatory Lesson: Precision medicine is changing both oncology and how FDA evaluates new cancer therapies.
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