In short
Larotrectinib is a targeted cancer medicine authorised for treating solid tumours with a specific genetic change called an NTRK gene fusion. It is taken by mouth as an oral solution. One registered clinical trial is currently authorised in the EU to compare molecularly tailored therapy including larotrectinib against standard care in 1,200 people with advanced pancreatic cancer.
Key points
- Larotrectinib is authorised as a medicine for solid tumours with NTRK gene fusions.
- One Phase 2 trial is authorised in the EU involving 1,200 participants with advanced pancreatic cancer.
- The trial compares molecularly tailored therapy including larotrectinib against standard of care.
- The primary outcome is progression-free survival, measuring how long people live without their cancer worsening.
- Larotrectinib is given orally at a dose of 200 mg in the trial.
What is larotrectinib?
Larotrectinib is a targeted cancer medicine, also known by the trade name Vitrakvi. It is a chemically synthesised substance that is authorised as a medicine. The US Food and Drug Administration (FDA) approved Vitrakvi as an oral solution, 20 mg/mL, for the treatment of adult and paediatric patients with solid tumours that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have no satisfactory alternative treatments or have progressed following treatment. In Europe, the European Medicines Agency has agreed a paediatric investigation plan for larotrectinib, covering its study in children. In the EU clinical trial registry, larotrectinib appears as a test substance in two trial records.
How does larotrectinib work?
Larotrectinib belongs to a group of medicines called multikinase inhibitors, which block signals that cancer cells use to grow. It targets tumours driven by NTRK gene fusions, which are genetic changes that cause abnormal proteins to form and drive cancer growth. The medicine is designed for use when the tumour is locally advanced or metastatic, or when surgery could cause severe health problems, and no satisfactory treatment options remain.
Which conditions is larotrectinib used or studied for?
- Solid tumours with NTRK gene fusions — larotrectinib is authorised for adult and paediatric patients whose tumours carry this specific genetic change and who have no satisfactory alternatives.
- Advanced pancreatic cancer — the authorised Phase 2 trial is studying larotrectinib as part of molecularly tailored therapy compared with standard care in people with advanced pancreatic cancer.
How is larotrectinib given?
Larotrectinib is taken by mouth. The approved form is an oral solution containing 20 mg of larotrectinib per millilitre. In the authorised clinical trial, larotrectinib is given orally at a dose of 200 mg.
What do the clinical trials show?
One trial involving larotrectinib is registered in the EU Clinical Trials Information System. It is a Phase 2 interventional trial with 1,200 participants, currently authorised to begin.
What is the trial measuring?
The primary outcome is progression-free survival, which measures how long people live without their cancer getting worse. The trial compares molecularly tailored therapy against standard of care in patients with pancreatic cancer who have actionable, reimbursed, druggable molecular alterations.
Who can take part?
The trial enrols patients with advanced pancreatic cancer. Larotrectinib is one of many targeted drugs in the molecularly tailored therapy arm, given to patients whose tumours carry the relevant molecular alteration.