The Servier Group in Italy announces that ivosidenib has been awarded in the ‘Chemical synthesis drug’ category at the Prix Galien Italia 2024 ceremony: the most important recognition in the biomedical and pharmacological field in our country, organized by Springer Healthcare Italia. Established in 1970 and recognized internationally with editions in various countries, the Prix Galien is considered by professionals as the ‘Nobel’ of pharmaceuticals, which valorises innovative drugs and medical devices with a significant contribution to human health and rewards excellence among young researchers.
“I thank all the members of the jury who absolutely independently evaluated the numerous dossiers of innovative therapies, as well as the applications of the best Italian researchers in the field of pharmacological research (pre-clinical, translational and clinical) candidates for the 2024 edition of the Prix Galien Italia, a beacon of excellence for pharmacological research – declares Pier Luigi Canonico, president of the jury – For the ‘Chemical synthetic drug’ category, the prize was awarded to ivosidenib, a new target therapy indicated for two orphan neoplasms: the acute myeloid leukemia and cholangiocarcinoma with Idh1 mutation.
Ivosidenib is a new molecularly targeted oral therapy – reports a note – a powerful and selective inhibitor of the mutated isocitrate dehydrogenase 1 (Idh1) enzyme, responsible for blocking cellular differentiation and the development of haematological and non-haematological neoplasms. The first and only Idh1 inhibitor currently approved in Europe, it has obtained orphan drug designation from the EMA for the treatment in two indications: in monotherapy, of adult patients with locally advanced or metastatic cholangiocarcinoma (Cca) with Idh1 mutation, previously treated with at least one line of systemic therapy; in combination with azacitidine, of adult patients newly diagnosed with acute myeloid leukemia (AML) with an Idh1 mutation who are not eligible for standard induction chemotherapy.
Idh1 mutations – we read – are present in approximately 15% of cases of intrahepatic cholangiocarcinoma: a type of primary liver tumor, which records approximately 5,400 new diagnoses in Italy every year. 70% of patients present at diagnosis with an already advanced disease and 5-year survival is low, equal to 17% in men and 15% in women. “Ivosidenib is a highly innovative drug that acts with a completely new mechanism of action and is the only molecularly targeted drug available for patients with advanced or metastatic cholangiocarcinoma with Idh1 mutation – comments Lorenza Rimassa, associate professor of Oncology medical at Humanitas University and Irccs Humanitas Research Hospital, Rozzano (Milan) – The ClarIDHy phase 3 pivotal study has in fact demonstrated that in patients with Idh1 mutation, already treated with standard therapy, therapy with ivosidenib allowed for an improvement in progression-free and overall survival, associated with a good tolerability profile and a good quality of life”.
Molecular profiling is a fundamental part of the diagnosis also in acute myeloid leukemia, a blood cancer that affects around 2,100 people in Italy every year with a 5-year survival from diagnosis of around 30%. “Up to 50% of patients with acute myeloid leukemia have at least one gene mutation that potentially constitutes a target for targeted therapy. Among these, mutations in the Idh genes are those for which specific drugs have been developed. Mutations in the Idh1 gene they are present in approximately 10% of cases, those of Idh2 in 10-15% – underlines Adriano Venditti, director of Hematology at the Tor Vergata Polyclinic Foundation in Rome – Acute myeloid leukemia is an insidious hematological disease and still difficult to treat However, research is progressing rapidly and new drugs are available for the treatment of this disease. A paradigmatic example is that of the Agile study, published in the ‘New England Journal of Medicine’, in which a first-line therapy with ivosidenib (a drug that targeting the mutation in the Idh1 gene) in combination with azacitidine, tripled the median overall survival compared to placebo and azacitidine: 24 months versus 7.9 months”.
In recent years – the note reports – Servier has taken an important step forward in oncology and onco-hematology, which have become strategic priorities for the group. Thanks to its commitment, Servier now has a portfolio of innovative therapies for hard-to-treat tumors and a promising pipeline.
“We are honored and proud to receive this prestigious award: an important milestone in our journey towards innovation in oncology, which demonstrates the solidity of our R&D strategy. With 36 projects in clinical development and 25 in research, Servier is by concentrating its R&D efforts in all therapeutic areas where important clinical needs are found, today more than 50% of the Group’s research projects have the potential to become ‘first in class’ drugs, concludes Gilles Renacco, president of the Servier Group. in Italy.
