This targeted therapy was developed to combat the progression of acute myeloid leukemia and cholangiocarcinoma.
The Servier pharmaceutical group has obtained approval for the use of ivosidenib in Europe as a treatment for i IDH1-mutated tumours. This targeted therapy was developed to combat the progression of acute myeloid leukemia and cholangiocarcinoma, two types of cancers that are often diagnosed late and have poor prognoses. Ivosidenib works by inhibiting isocitrate dehydrogenase-1 (IDH1), an enzyme responsible for the progression of these malignancies. The drug has already been approved in the United States, Australia and China, and has now also received the green light in Europe as an orphan drug.
In the case of the acute myeloid leukemia, the approval is based on results from the Phase 3 Agile study, which showed that the combination of ivosidenib and azacitidine led to a significant improvement in event-free survival and overall survival compared to placebo plus azacitidine. In cholangiocarcinoma, the approval is based on results from the Phase 3 Claridhy study, which showed a significant improvement in progression-free survival with the use of ivosidenib compared to placebo.
The most common side effects reported with the use of ivosidenib include vomiting, neutropenia, thrombocytopenia, electrocardiogram QT prolongation, and insomnia for acute myeloid leukemia, while for cholangiocarcinoma, fatigue, nausea, abdominal pain, diarrhea, decreased appetite, ascites, vomiting, anemia and rash.
This approval represents an important step forward in the treatment of IDH1-mutated cancers in Europe, offering new therapeutic options to improve the survival and quality of life of patients affected by these malignancies.
