{"entity":{"id":"idea-bio2-anamorelin-access","kind":"idea","name":"Get the one approved appetite drug licensed beyond a single country","aka":[],"tldr":"A drug that improves appetite and lean weight in cancer wasting is approved in Japan but almost nowhere else. Reviewing the existing evidence could widen access quickly.","summary":"Anamorelin, a ghrelin receptor agonist, is approved in Japan for cancer cachexia in several tumour types, having shown gains in lean body mass with inconsistent effects on handgrip strength in the ROMANA programme. Other regulators declined largely on the function endpoint. A reappraisal using pooled data with a qualified function endpoint, or a confirmatory pragmatic trial in a stratified population, could resolve the question.","asOf":"2026-09-08","links":[{"label":"Bottleneck evidence (Cachexia, toxicity and the limits of the patient): Fearon et al., Definition and classification of cancer cachexia (Lancet Oncology 2011)","url":"https://doi.org/10.1016/S1470-2045(10)70218-7"}],"tags":[],"related":[],"cancers":["nsclc","pancreatic","gastric"],"sections":[],"technologies":["exercise-oncology"],"targets":[],"drugs":[],"companies":[],"institutions":["ncc-japan"],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":["b-cachexia-supportive","b-regulatory-fragmentation","b-global-access"],"keyPapers":["paper-fearon-lancet-oncol"],"journals":[],"dependsOn":[],"notes":[],"hypothesis":"A confirmatory trial in GDF-15-high or inflammation-high patients, using a qualified function endpoint and combined with exercise and protein, demonstrates a clinically meaningful functional benefit that earlier unselected trials missed.","rationale":"The lean mass effect is reproducible; what failed was the demonstration of functional translation, which is plausibly a design problem given that no anabolic stimulus was co-administered. Real-world Japanese experience provides safety and effectiveness data at scale.","test":"Publish a pooled analysis of existing trials plus Japanese real-world data; if the signal holds in stratified subgroups, run one confirmatory trial designed around the function endpoint.","maturity":"early-clinical","actor":"regulator","cost":"small","horizonYears":3},"route":"/ideas/idea-bio2-anamorelin-access/","neighbours":{"cancer":[{"id":"gastric","kind":"cancer","name":"Gastric & gastro-oesophageal junction cancer","route":"/cancers/gastric/"},{"id":"nsclc","kind":"cancer","name":"Non-small-cell lung cancer","route":"/cancers/nsclc/"},{"id":"pancreatic","kind":"cancer","name":"Pancreatic ductal adenocarcinoma","route":"/cancers/pancreatic/"}],"technology":[{"id":"cachexia-appetite-pharmacotherapy","kind":"technology","name":"Cachexia pharmacotherapy: GDF-15 blockade, anamorelin, olanzapine","route":"/technologies/cachexia-appetite-pharmacotherapy/"},{"id":"exercise-oncology","kind":"technology","name":"Exercise & lifestyle oncology","route":"/technologies/exercise-oncology/"}],"institution":[{"id":"ncc-japan","kind":"institution","name":"National Cancer Center Hospital","route":"/institutions/ncc-japan/"}],"bottleneck":[{"id":"b-cachexia-supportive","kind":"bottleneck","name":"Cachexia, toxicity and the limits of the patient","route":"/bottlenecks/b-cachexia-supportive/"},{"id":"b-global-access","kind":"bottleneck","name":"Most of the world has almost no cancer care","route":"/bottlenecks/b-global-access/"},{"id":"b-regulatory-fragmentation","kind":"bottleneck","name":"Regulatory divergence between regions","route":"/bottlenecks/b-regulatory-fragmentation/"}],"paper":[{"id":"paper-fearon-lancet-oncol","kind":"paper","name":"Definition and classification of cancer cachexia: an international consensus","route":"/key-papers/paper-fearon-lancet-oncol/"}],"trial":[{"id":"romana-1-2","kind":"trial","name":"ROMANA 1 and ROMANA 2","route":"/trials/romana-1-2/"}]}}