Quick Takes: FDA approves Takeda’s Exkivity for NSCLC with specific mutation

Almost six weeks ahead of the therapy’s PDUFA date, FDA granted accelerated approval to Exkivity mobocertinib from Takeda Pharmaceutical Co. Ltd. (Tokyo:4502; NYSE:TAK) to treat non-small cell lung cancer with an EGFR exon 20 insertion mutation, making it the first approved oral therapy in a competitive indication. The approval is based on data from a Phase I/II trial of the tyrosine kinase inhibitor, showing an overall response rate (ORR) of 28%, disease control rate of 78%, median duration of response of 17.5 months, and overall survival of 24 months. EGFR exon 20 insertions produce a conformational change in the receptor’s intracellular domain that renders tumors insensitive to traditional TKIs.

Signaling an apparent reversal of FDA’s thinking, Amylyx Pharmaceuticals Inc. said it intends to submit an NDA within the coming months for AMX0035 to treat amyotrophic lateral sclerosis, rather than wait for completion of a Phase III trial, based on interactions with FDA including a July pre-NDA meeting. The combination of sodium phenylbutyrate and tauroursodeoxycholic acid significantly slowed functional decline and led to a survival benefit in the 137-patient CENTAUR trial, leading foundations such as the ALS Association and I AM ALS to petition for the agency to approve AMX0035 without a longer, larger study. Until Wednesday, the company had guided for a submission after the receipt of additional data. The candidate is already under review by Health Canada, and Amylyx expects to submit an MAA to EMA this year. AMX0035 is one of dozens of clinical candidates in an expanding ALS pipeline...