2023-05-11 11:36:01
The European Commission (EC) has approved ivosidenib tablets as targeted therapy in two indications: in combination with azacitidine for the treatment of adult patients with newly diagnosed acute imeloid leukemia (AML) with an isocitrate dehydrogenase-1 (Idh1) mutation who are not eligible to receive standard induction chemotherapy; as monotherapy for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma (Cca) with an Idh1 mutation, who have been previously treated with at least one line of systemic therapy. The Servier pharmaceutical group announces it in a note. The first and only Idh1 inhibitor approved in Europe has received ivosidenib orphan drug designation, recognizing the significant benefit ivosidenib brings to patients over available therapies for both Cca and AML, two hard-to-treat cancers. deal with.
“The prognosis for patients diagnosed with acute myeloid leukemia or cholangiocarcinoma has always been poor, with treatment options very limited,” said Arnaud Lallouette, Executive Vice President, Global Medical & Patient Affairs at Servier. European Commission, ivosidenib is now the first targeted Idh1 inhibitor approved in Europe.This further confirms our scientific leadership in the setting of Idh1 mutations and our commitment to finding new therapeutic solutions for patients with difficult-to-treat cancers” .
“Idh1 mutations – explains Philippe Gonnard, Executive Vice President, Global Product Strategy of Servier – are the main factors of disease progression in acute myeloid leukemia and cholangiocarcinoma, diseases that are often diagnosed at an advanced stage and therefore need a targeted treatment option.The development of new therapies such as ivosidenib, with a mechanism of action different from traditional chemotherapy, offers patients new treatment options that can increase life expectancy and quality of life.”
AML – recalls the note – is a cancer of the blood and bone marrow characterized by a rapid progression of the disease. It is the most common acute leukemia in adults and affects 5 inhabitants out of 100,000 in Europe, i.e. more than 20,000 people every year. The 2-year survival rate of patients with 75-year AML is less than 10%. The approval of ivosidenib for acute myeloid leukemia is based on data from the Phase 3 Agile study, published in the New England Journal of Medicine. The results demonstrated a statistically significant improvement in event-free survival (Efs) (hazard ratio – Hr 0.33) and overall survival (Os) (Hr 0.44) in patients with Idh1-mutated AML ineligible for target chemotherapy , and who combined ivosidenib with azacitidine, compared to those who used only azacitidine plus placebo. Median overall survival was 24 months in the ivosidenib-only group compared with 7.9 months in the azacitidine-only group. In addition to the primary endpoint of EFS, the study met all key secondary endpoints. The most common adverse reactions were vomiting, neutropenia, thrombocytopenia, electrocardiogram Qt prolongation, and insomnia.
Cholangiocarcinoma is a rare and aggressive cancer of the bile ducts, often related to a medical history such as cirrhosis or a liver infection. CCA affects 1-3 out of 100,000 people in Europe, with about 10,000 new cases each year. The 5-year survival rate is 9%, but 0% in case of metastases. Only surgery can cure patients, but treatment is only possible for a limited number and the risk of recurrence remains high. Chemotherapy and immunotherapy are the standard therapies for patients with Cca who cannot undergo surgery or whose disease has progressed after surgery. The approval of ivosidenib in Cca is supported by data from the Claridhy study, the first and only randomized Phase 3 study for previously treated Idh1-mutated Cca. The results demonstrated a statistically significant improvement in the primary endpoint of progression-free survival (PFS). The median PFS for ivosidenib and placebo were 2.7 and 1.4 months, respectively. 32% and 22% of patients randomized to ivosidenib remained progression-free or death-free at 6 and 12 months, respectively, versus none in the placebo arm. The most common adverse reactions were fatigue, nausea, abdominal pain, diarrhea, decreased appetite, ascites, vomiting, anemia, and rash.
Ivosidenib is currently approved in the United States as monotherapy for the treatment of adults with relapsed or refractory Idh1-mutated AML and as monotherapy or in combination with azacitidine for adults with newly diagnosed Idh1-mutated AML 75 years of age or older or with comorbidities that preclude the use of intensive induction chemotherapy. The drug is also approved in the USA and Australia for previously treated patients with Idh1-mutated Cca. In China in adults with relapsed or refractory AML who have an Idh1-sensitive mutation. The marketing authorization covers the 27 countries of the European Union, as well as Iceland, Liechtenstein and Norway.
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