Takeda’s Iclusig (ponatinib) has been granted accelerated approval by the US Food and Drug Administration (FDA) to treat adults who are newly diagnosed with a rare form of leukaemia.
The kinase inhibitor, which has been authorised for use alongside chemotherapy, is now the first targeted therapy approved in the US as a frontline treatment for Philadelphia chromosome-positive acute lymphoblastic leukaemia (Ph-positive ALL).
Affecting approximately 25% of adult ALL patients in the US, Ph-positive ALL is characterised by the presence of an abnormal gene known as the Philadelphia chromosome.
Iclusig is already approved in the US to treat Ph-positive ALL patients with no other kinase inhibitor options, those with T315I-positive Ph-positive ALL, as well as certain patients with chronic myeloid leukaemia.
Takeda’s supplemental application for the drug was supported by positive results from the late-stage PhALLCON study, in which patients received either Iclusig or imatinib plus reduced-intensity chemotherapy.
The trial met its primary endpoint, with Iclusig-treated patients achieving a greater than two-fold improvement in the rate of minimal residual disease-negative complete remission at the end of induction.
The safety profile of Iclusig was comparable to that of imatinib and no new safety signals were identified.
Awny Farajallah, chief medical officer, oncology at Takeda, said: “This label expansion for Iclusig is an incredibly exciting milestone, allowing US adult patients with newly diagnosed Ph-positive ALL to have an approved, targeted treatment option in the frontline.
“We are thrilled that the FDA has recognised the potential of Iclusig to fill a large gap in care for these patients and look forward to seeing the impact this can have on people with this rare and aggressive form of cancer.”
Takeda gained access to Iclusig through its $5.2bn acquisition of Ariad Pharmaceuticals in 2017.
The approval comes just days after Takeda’s Eohilia (budesonide oral suspension) was approved by the FDA as the first and only oral therapy in the US for eosinophilic oesophagitis (EoE), a chronic, immune-mediated, inflammatory disease affecting around one in 2,000 people in the US.
The regulator’s decision means the corticosteroid can be used as a 12-week treatment in patients aged 11 years and older with EoE.
