USA – J&J files ‘breakthrough’ bladder cancer drug in US

Needed as most patients don't respond to checkpoint inhibitors. Johnson & Johnson has filed for approval of a new targeted bladder cancer therapy in the US, setting up a possible approval next year.

The new drug candidate – selective fibroblast growth factor receptor inhibitor erdafitinib – is an oral, once daily treatment for the second-line treatment of patients with locally advanced or metastatic urothelial cancer (UC) with certain FGFR mutations whose tumours have progressed after chemotherapy. FGFR mutations typically occur in around one in five UC patients.

Erdafitinib (JNJ-42756493) picked up a breakthrough designation (BTD) from the FDA earlier this year, so should benefit from a rapid review by the regulator, setting up a possible approval in the first half of 2019. The application is based on data from a phase 2 trial (BLC2001) which was presented at this year’s American Society of Clinical Oncology (ASCO) meeting.

The study was listed among the best-in-show studies, mainly because despite the emergence of immunotherapy with checkpoint inhibitors – which has transformed the prospects for some bladder cancer patients in recent years – the majority of patients don’t respond to this type of treatment.

The 99-patient study revealed a 40% overall response rate with erdafitinib, 3% complete responses and 37% partial responses – along with a median progression-free survival of 5.5 months and median overall survival of 13.8 months…