OnCo
ideasIdea

Get the one approved appetite drug licensed beyond a single country

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.

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.

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.
What would test it
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
Who has to act
regulator
Cost to try
Small (under $1M)
Years to first evidence
3
Bottlenecks it attacks

Connected

10top

Pages like this

not linked directly; found by shared links