Repotrectinib isindicated for the treatment of adults with locally advanced or metastaticROS1-positive non-small cell lung cancer.[4][7]
In June 2024, the USFood and Drug Administration (FDA) expanded the indication to include the treatment of people twelve years of age and older with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion, are locally advanced or metastatic or where surgical resection is likely to result in severe morbidity, and that have progressed following treatment or have no satisfactory alternative therapy.[9][10]
The most common adverse reactions include dizziness, dysgeusia, peripheral neuropathy, constipation, dyspnea, fatigue, ataxia, cognitive impairment, muscular weakness, and nausea.[9]
Approval by the USFood and Drug Administration (FDA) was based on TRIDENT-1, a global, multicenter, single-arm, open-label, multi-cohort clinical trial (NCT03093116) which included participants with ROS1-positive locally advanced or metastatic non-small cell lung cancer.[7] Efficacy was evaluated in 71ROS1 tyrosine kinase inhibitor-naïve participants who received up to one prior line of platinum-based chemotherapy and/or immunotherapy and 56 participants who received one priorROS1 tyrosine kinase inhibitor with no prior platinum-based chemotherapy or immunotherapy.[7]
Efficacy was evaluated in TRIDENT-1 (NCT03093116), a multicenter, single-arm, open-label, multi-cohort trial in 88 adult participants with locally advanced or metastatic neurotrophic tyrosine receptor kinase gene fusion-positive solid tumors who had either received a prior TRK tyrosine kinase inhibitor (TKI) (n=48) or were TKI-naïve (n=40).[9][11] All participants were assessed for central nervous (CNS) lesions at baseline, and patients with symptomatic brain metastases were excluded. Tumor assessments were performed every eight weeks.[9]
In November 2024, theCommittee for Medicinal Products for Human Use of theEuropean Medicines Agency adopted a positive opinion, recommending the granting of a conditional marketing authorization for the medicinal product Augtyro, intended for the treatment of people whose solid tumors have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion, or people with ROS1-positive advanced non-small cell lung cancer (NSCLC).[5] The applicant for this medicinal product is Bristol-Myers Squibb Pharma EEIG.[5][12] Repotrectinib was authorized for medical use in the European Union in January 2025.[5][6]
^abcde"Augtyro EPAR".European Medicines Agency (EMA). 14 November 2024. Retrieved16 November 2024. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
^abc"Augtyro PI".Union Register of medicinal products. 14 January 2025. Retrieved16 January 2025.
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