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.

As of July 20, 2020, 18 patients with metastatic TRK fusion-positive GI cancer were enrolled...The other tumor types were cholangiocarcinoma (n=3), pancreatic (n=2), appendiceal (n=1), esophageal (n=1), and hepatocellular carcinoma (n=1)….In patients with TRK fusion-positive GI cancer, larotrectinib demonstrated rapid responses with high survival rates and a favorable safety profile.

...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.

...we enrolled 55 consecutive patients...The demographic characteristics of the patients, the tumor type, and fusion characteristics are summarized in Table 1,...Larotrectinib had marked and durable antitumor activity in patients with TRK fusion–positive cancer, regardless of the age of the patient or of the tumor type.