We’re delighted to announce that a new drug, larotrectinib, has been approved for the treatment of certain sarcomas in England. ‘Sarcoma UK welcomes today’s approval of larotrectinib. Made available for use by the Cancer Drugs Fund and approved by NICE (National Institute for Health and Care Excellence) larotrectinib is a new type of ‘histologically-independent’ drug that targets tumours based on their genetic makeup. It works by targeting the specific NTKR genetic abnormality in tumours meaning patients are treated according to the genetics of their tumour rather than where it is in the body.

The ORR with larotrectinib in adult and pediatric patients with sarcoma harboring an NTRK fusion was 74% (95% CI 52–90) and 94% (95% CI 82–99), respectively….Of two patients with a bone sarcoma, one had a partial response and one had stable disease.

...adult subset of pts with TRK fusion cancer treated with larotrectinib. Cancer types included salivary gland (23%) and thyroid cancer (19%), soft tissue sarcoma (14%), lung cancer (13%), colon cancer and melanoma (7% each), GIST (5%), and bone sarcoma, cholangiocarcinoma, and appendiceal, breast, and pancreas cancer (≤2% each). In 74 pts evaluable per INV, the ORR was 76% with 9% CR, 57% confirmed PR, 9% PR pending confirmation, 12% SD, 11% PD, and 1% not determined...In 65 pts evaluable per IRC, the ORR was 68% with 17% CR, 51% PR, 15% SD, 12% PD, and 5% NE....Larotrectinib demonstrated robust tumor-agnostic efficacy and a favorable safety profile in adult pts with TRK fusion cancer.