The FDA on Tuesday approved Celgene and Agios Pharmaceuticals' Idhifa (enasidenib) for the treatment of adults with relapsed or refractory acute myeloid leukaemia (AML) who have a specific genetic mutation. The agency noted that the IDH2 inhibitor has been authorised for use with a companion diagnostic, Abbott's RealTime IDH2 Assay, which is used to detect specific mutations in the IDH2 gene in patients with AML.
"Idhifa is a targeted therapy that fills an unmet need for patients with relapsed or refractory AML who have an IDH2 mutation," remarked Richard Pazdur, director of the FDA's Oncology Center of Excellence. Pazdur added "the use of Idhifa was associated with a complete remission in some patients and a reduction in the need for both red cell and platelet transfusions."
The US agency noted that the approval was supported by safety and efficacy data from the Phase I/II AG221-C-001 trial, which included 199 patients, with study results showing that with a minimum of six months of treatment, 19 percent of patients experienced complete response for a median of 8.2 months, while 4 percent of patients experienced complete response with partial haematologic improvement for a median of 9.6 months. Further, of the 157 patients who required transfusions of blood or platelets due to AML at the start of the study, 34 percent no longer required transfusions after treatment with Idhifa.
Celgene CEO Mark Alles remarked "the FDA approval of Idhifa provides the first-ever treatment option for patients living with relapsed or refractory AML and an IDH2 mutation." The FDA awarded priority review status to the drug in March.
According to Celgene, the thereapy is expected to have a list price of nearly $25 000 per month. JP Morgan analyst Cory Kasimov remarked "while the product isn't expected to be a big needle mover for Celgene, it does represent the first approval for a partnered product."
Celgene and Agios co-developed Idhifa, formerly known as AG-221, under the terms of a collaboration for treatments targeting cancer metabolism that was expanded in 2013. The next year, Celgene exercised an option granting it an exclusive global license to the treatment.
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