Type of information: Submission of a Market Application in the US
Product name: ivosidenib (AG-120)
Therapeutic area: Cancer - Oncology - Rare diseases
- enzyme inhibitor/isocitrate dehydrogenase inhibitor . AG-120 (ivosidenib) is a first-in-class, orally available, selective, potent inhibitor of the mutated IDH1 (Isocitrate dehydrogenase 1) protein, and is a highly targeted investigational medicine for the treatment of patients with cancers that harbor an IDH1 mutation. Isocitrate dehydrogenase (IDH) 1 and 2 are metabolic enzymes that are mutated in a wide range of hematologic and solid tumor malignancies, including acute myelogenous leukemia (AML) and glioma, a type of aggressive brain tumor with poor prognosis. Normally, IDH enzymes help to break down nutrients and generate energy for cells. When mutated, IDH creates a molecule that alters the cells’ genetic programming, and instead of maturing, the cells remain primitive and proliferate quickly. AG-120 is being developed in collaboration with Celgene.
Company: QRC Consultants (UK) - Agios Pharmaceuticals (USA - MA)
- acute myeloid leukaemia
- patients with relapsed or refractory acute myeloid leukemia (R/R AML) and an isocitrate dehydrogenase-1 (IDH1) mutation
- • On December 26, 2017, Agios Pharmaceuticals announced that it has submitted a New Drug Application (NDA) to the FDA for ivosidenib (AG-120), an investigational oral treatment for patients with relapsed or refractory acute myeloid leukemia (R/R AML) and an isocitrate dehydrogenase-1 (IDH1) mutation. Agios has requested priority review for the application, which, if granted, could result in a six-month review process.
- The NDA is supported by data from the ongoing Phase 1 dose-escalation and expansion study of ivosidenib in patients with advanced hematologic malignancies and an IDH1 mutation. Ivosidenib is wholly owned by Agios. The FDA has a 60-day filing review period to determine whether the NDA is complete and acceptable for filing.
- • On 3-4 November 2016, the Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending the approval of the orphan medicinal product designation for ivosidenib for treatment of acute myeloid leukaemia.
Submission of marketing authorization application USA : 2017-12-26
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA:
Orphan status UE: 2016-12-12
Pediatric exclusivit _USA:
Pediatric exclusivity UE: