Agios Pharmaceutical Inc. (NASDAQ:AGIO) has withdrawn its European Marketing Authorization Application (MAA) for TIBSOVO® in treating refractory or relapsed acute myeloid leukemia patients with isocitrate dehydrogenase-1 mutation.
Phase 1 clinical data didn’t support risk-benefit balance for TIBSOVO®
The company’s decision is based on the EMA’s Committee for Medicinal Products for Human Use feedback on its phase 1 clinical study data. According to the CHMP, the study data did not adequately support a positive risk-benefit balance for the intended indication.
Chris Bowden, the Chief Medical Officer of Agios, said that they are disappointed for not delivering TIBSOVO® to patients with AML in the EU based on the current data in refractory/relapsed settings. This is despite there being a significant need for a new treatment alternative for the devastating disease. Bowden said that the company believes in the benefit of TIBSOVO® for AML patients. Currently, Agios is advancing its two ongoing third phase randomized controlled trials assessing TIBSOVO® combinations in patients with newly diagnosed AML. Bowden added that the studies will be positive they plan to pursue approval for the US and EU indications.
Agios evaluating TIBSOVO® in newly diagnosed AML in two studies
The company is enrolling two Phase 3 combination studies in newly diagnosed AML, AGILE and HOVON150/AMLSG29. AGILE is the third phase TIBSOVO® study in combination with azacitidine for treating newly diagnosed AML patients that are not eligible for intensive chemotherapy. On the other hand, HOVIN150/AMLSG29 is an intergroup sponsored third phase trial studying TIBSOVO® or IDHIFA® combined with consolidation chemotherapy and standard induction in newly diagnosed AML patients qualifying for intensive chemotherapy.
Most patients with AML having IDH1 mutation qualify for non-intensive or intensive therapy. TIBSOVO® is currently indicated for treating patients with acute myeloid leukemia patients with isocitrate dehydrogenase-1 mutation, as detected through an FDA-approved test. This is in adult patients with refractory or relapsed AML and newly-diagnosed AML adult patients above 75 years. This can include patients with comorbidities precluding the use of intensive induction chemotherapy.