Anamorelin

updated

Anamorelin is an oral ghrelin receptor agonist approved in Japan as Adlumiz for cancer cachexia (company statement of 22 January 2021). The EMA refused authorization in 2017, and no FDA record exists. In four placebo-controlled phase 3 trials it added about 1 to 2 kg of weight or lean mass over 12 weeks but did not improve handgrip strength, and a PubMed search on 10 October 2026 found no trial testing muscle gain in healthy people or athletes.

Ghrelin receptor agonist (small molecule)

  • Ono Pharmaceutical (with Helsinn) said in a company statement dated 22 January 2021 that it received manufacturing and marketing approval in Japan for Adlumiz (anamorelin hydrochloride) for cancer cachexia in non-small cell lung, gastric, pancreatic and colorectal cancer.
  • The EMA refused marketing authorization on 16 November 2017 after a negative CHMP opinion on 18 May 2017, confirmed on re-examination on 14 September 2017. openFDA Drugs@FDA and DailyMed returned no US record or label on 10 October 2026.
  • In ROMANA 1 and 2 (484 and 495 patients with advanced non-small-cell lung cancer and cachexia), lean body mass rose over 12 weeks, but handgrip strength, the other co-primary endpoint, did not differ (p=0.15 and p=0.65).
  • In two later US-led phase 3 trials (318 randomized each, sponsor-posted registry results), weight rose more than on placebo but the anorexia-symptom co-primary did not differ, and about half of each arm did not complete.
  • A PubMed search on 10 October 2026 for anamorelin in healthy people, athletes or older adults, excluding cancer, returned 26 records and no trial testing muscle gain.
  • A Japanese adverse event database analysis (537 reports) found an adjusted reporting odds ratio of 20.00 (95% CI 14.86 to 26.91) for conduction defects, which measures disproportionate reporting, not incidence.

What is anamorelin, and where is it approved?

Anamorelin is a small, orally active non-peptide ghrelin receptor agonist. Ono Pharmaceutical said in a company statement dated 22 January 2021 that it received approval in Japan for Adlumiz for cancer cachexia in four cancer types. The EMA refused authorization in 2017, and openFDA and DailyMed returned no US record or label on 10 October 2026.

Anamorelin is a small, orally active non-peptide molecule. PubChem lists the free base as CID 9828911 (C31H42N6O3, molecular weight 546.7, CAS 249921-19-5; synonyms RC-1291, ONO-7643 and ST-1291) and anamorelin hydrochloride, the form in the approved tablet, as CID 16072155 (C31H43ClN6O3, molecular weight 583.2, CAS 861998-00-7). A vendor product may be either form, and doses in the trials are for the hydrochloride.

In a company statement dated 22 January 2021, Ono Pharmaceutical (with Helsinn) said it received manufacturing and marketing approval in Japan for Adlumiz (anamorelin hydrochloride) tablets for cancer cachexia in non-small cell lung cancer, gastric cancer, pancreatic cancer and colorectal cancer, for adults. The basis named was a randomized, placebo-controlled phase 2 trial in lung cancer (ONO-7643-04) and an open-label, uncontrolled phase 3 study in gastrointestinal cancers (ONO-7643-05). A 2023 review gives the same approval date. A December 2020 editorial gave 11 December 2020, which is likely a pre-approval step and is not used here. No PMDA document was read, and the Japanese label's cardiac contraindications and warnings were not read.

On 10 October 2026, openFDA Drugs@FDA returned no record for anamorelin and DailyMed returned no label, so no US approval appears. The EU outcome is set out in the next section.

PubChem: anamorelin and anamorelin hydrochloride identity

Chemical identity record

National Library of Medicine. PubChem compound records CID 9828911 (anamorelin) and CID 16072155 (anamorelin hydrochloride).

Anamorelin is a small, orally active non-peptide molecule. PubChem lists the free base as CID 9828911, C31H42N6O3, molecular weight 546.7, CAS 249921-19-5 (synonyms RC-1291, ONO-7643, ST-1291), and anamorelin hydrochloride, the form in the approved tablet, as CID 16072155, C31H43ClN6O3, molecular weight 583.2, CAS 861998-00-7.

Participants / model
Chemical identity, not a clinical population
Treatment
Defined small molecule and hydrochloride salt
Follow-up
Not applicable
Study design
NLM chemical record
Funding
not stated in the source

A vendor product may be either form. Doses in the trials are for the hydrochloride.

Read the original source
Company statement, 22 January 2021: Adlumiz approved in Japan for cancer cachexia in four cancer types

Regulatory record

Ono Pharmaceutical Co., Ltd., with Helsinn. News release dated 22 January 2021 on manufacturing and marketing approval of Adlumiz (anamorelin hydrochloride) in Japan.

The company said Ono received manufacturing and marketing approval for Adlumiz (anamorelin hydrochloride) tablets for cancer cachexia in non-small cell lung cancer, gastric cancer, pancreatic cancer and colorectal cancer, for adults. The basis named was a randomized, placebo-controlled phase 2 trial in lung cancer (ONO-7643-04) and an open-label, uncontrolled phase 3 study in gastrointestinal cancers (ONO-7643-05). A 2023 review gives the same approval date.

Participants / model
Adults in Japan with cachexia from these four cancers
Treatment
Oral tablet
Follow-up
Statement dated 22 January 2021
Study design
Company statement
Funding
not stated in the source

A December 2020 editorial gave 11 December 2020, which is likely a pre-approval step and is not used here. No PMDA document was read, and the Japanese label's cardiac contraindications and warnings were not read.

Read the original source
FDA record searches, 10 October 2026: no record and no label

Regulatory record

U.S. Food and Drug Administration. openFDA Drugs@FDA query and DailyMed label search for anamorelin, run on 10 October 2026.

openFDA Drugs@FDA returned no record for anamorelin, and DailyMed returned no label.

Participants / model
Records reviewed, not a population
Treatment
Not applicable
Follow-up
Searches run on 10 October 2026
Study design
Dated regulator database search
Funding
not stated in the source

A search result shows what the databases returned on that date. It is US-scoped.

Read the original source

Why did the EMA refuse anamorelin?

The CHMP gave a negative opinion on 18 May 2017, confirmed it on re-examination on 14 September 2017, and marketing authorization was refused on 16 November 2017. It cited a marginal effect on lean body mass, no proven effect on handgrip strength or quality of life, and safety data that had not been recorded adequately at inspected trial sites.

The EMA page for Adlumiz (applicant Helsinn Birex Pharmaceuticals) says the medicine has been refused authorisation. The CHMP gave a negative opinion on 18 May 2017, the refusal was confirmed on re-examination on 14 September 2017, and marketing authorisation was refused on 16 November 2017.

The CHMP cited a marginal effect on lean body mass, no proven effect on handgrip strength or quality of life, and safety data that had not been recorded adequately at inspected trial sites. These dates and reasons come from the EMA summary page, not from the full refusal assessment report.

EMA: negative CHMP opinion 18 May 2017, authorization refused 16 November 2017

Regulatory record

European Medicines Agency. Adlumiz (anamorelin), applicant Helsinn Birex Pharmaceuticals, European public assessment report summary page.

The EMA page says the medicine has been refused authorisation. The CHMP gave a negative opinion on 18 May 2017, the refusal was confirmed on re-examination on 14 September 2017, and marketing authorisation was refused on 16 November 2017. The CHMP cited a marginal effect on lean body mass, no proven effect on handgrip strength or quality of life, and safety data that had not been recorded adequately at inspected trial sites.

Participants / model
Not a clinical population
Treatment
Oral anamorelin hydrochloride
Follow-up
Opinion 18 May 2017; refusal 16 November 2017
Study design
Regulator record
Funding
not stated in the source

The dates come from the EPAR summary page, not from the full refusal assessment report.

Read the original source

What did the lung cancer trials find?

In ROMANA 1 and 2 (484 and 495 patients), lean body mass rose over 12 weeks (median 0.99 versus -0.47 kg and 0.65 versus -0.98 kg), but handgrip strength, the other co-primary endpoint, did not differ (p=0.15 and p=0.65). A pooled analysis of 6 randomized trials found 1.78 kg higher body weight and no difference in survival or handgrip strength.

ROMANA 1 and ROMANA 2 were double-blind trials at 93 sites in 19 countries. They randomized 484 and 495 patients with inoperable stage III or IV non-small-cell lung cancer and cachexia 2:1 to anamorelin 100 mg daily or placebo for 12 weeks. Lean body mass rose (median 0.99 versus -0.47 kg in ROMANA 1; 0.65 versus -0.98 kg in ROMANA 2), but handgrip strength, the other co-primary endpoint, did not differ (p=0.15 and p=0.65). Grade 3 to 4 treatment-related events did not differ. Helsinn funded the trials, registered as NCT01387269 and NCT01387282.

In the ROMANA 3 safety extension, 513 of 703 ROMANA completers with preserved performance status continued anamorelin (345) or placebo (168) for weeks 12 to 24. Body weight stayed higher on anamorelin through 24 weeks, with no handgrip improvement. The trial is registered as NCT01395914.

A meta-analysis of 6 randomized trials (1,641 patients with non-small cell lung cancer) found higher total body weight (mean difference 1.78 kg, 95% CI 1.28 to 2.28) and lean body mass (1.10 kg, 95% CI 0.35 to 1.85) on anamorelin, and a small quality-of-life gain (standardized mean difference 0.19). There was no difference in overall survival (HR 0.99) or handgrip strength.

ROMANA 1 and 2: 484 and 495 patients, 12 weeks, lean body mass up but handgrip strength unchanged

Randomized controlled trial

Temel JS et al. Lancet Oncol. 2016;17(4):519-531. PMID 26906526.

Lean body mass rose (median 0.99 versus -0.47 kg in ROMANA 1; 0.65 versus -0.98 kg in ROMANA 2), but handgrip strength, the other co-primary endpoint, did not differ (p=0.15 and p=0.65). Grade 3 to 4 treatment-related events did not differ, and hyperglycaemia was the most common grade 3 to 4 event (under 1% and 1%).

Participants / model
484 and 495 adults with inoperable stage III or IV non-small-cell lung cancer and cachexia, at 93 sites in 19 countries
Treatment
Oral anamorelin 100 mg daily or placebo, randomized 2:1
Follow-up
12 weeks
Study design
Two double-blind randomized phase 3 trials (NCT01387269 and NCT01387282)
Funding
Funded by Helsinn

Lean body mass and handgrip strength were the co-primary endpoints; only lean body mass improved.

Read the original source
ROMANA 3: 513 completers, weeks 12 to 24, similar adverse events, no handgrip gain

Randomized controlled trial

Currow D et al. Ann Oncol. 2017;28(8):1949-1956. PMID 28472437.

Treatment-emergent adverse events (52.2% versus 55.7%), grade 3 or higher events (22.4% versus 21.6%) and serious events (12.8% versus 12.6%) were similar, and 36 and 23 deaths were judged unrelated. Body weight stayed higher on anamorelin through 24 weeks, with no handgrip improvement.

Participants / model
513 of 703 ROMANA completers with preserved performance status
Treatment
Oral anamorelin 100 mg daily (345) or placebo (168) for weeks 12 to 24
Follow-up
Weeks 12 to 24 of ROMANA
Study design
Randomized safety extension (NCT01395914)
Funding
not stated in the source

Only participants who completed ROMANA 1 or 2 with preserved performance status continued.

Read the original source
Meta-analysis: 6 trials, 1,641 patients, weight +1.78 kg, no survival or handgrip difference

Systematic review and meta-analysis

Nishie K et al. Lung Cancer. 2017;112:25-34. PMID 29191597.

Anamorelin gave higher total body weight (mean difference 1.78 kg, 95% CI 1.28 to 2.28) and lean body mass (1.10 kg, 95% CI 0.35 to 1.85), and a small quality-of-life gain (standardized mean difference 0.19). There was no difference in overall survival (HR 0.99) or handgrip strength, and adverse events did not differ.

Participants / model
1,641 patients with advanced non-small cell lung cancer and cachexia in 6 pooled randomized trials
Treatment
Oral anamorelin
Follow-up
Pooled trials
Study design
Systematic review and meta-analysis of 6 randomized trials
Funding
not stated in the source

Pooled outcomes were weight, lean mass, survival, handgrip strength and quality of life.

Read the original source

What did the Japanese studies find?

In ONO-7643-04, a double-blind trial in 174 Japanese patients with lung cancer cachexia, lean body mass changed 1.38 versus -0.17 kg over 12 weeks. ONO-7643-05, the other approval basis, was a single-arm study in 50 patients with no control group.

ONO-7643-04 randomized 174 Japanese patients with unresectable stage III or IV non-small cell lung cancer and cachexia to anamorelin 100 mg daily or placebo for 12 weeks. Least-squares mean lean body mass change was 1.38 versus -0.17 kg. Weight and anorexia symptoms improved, but handgrip strength and 6-minute walk did not. This randomized phase 2 trial is one of the two trials named as the basis of the Japan approval.

ONO-7643-05, the other approval basis, was an open-label single-arm phase 3 study in 50 Japanese patients with advanced colorectal, gastric or pancreatic cancer and cachexia who took anamorelin 100 mg daily for 12 weeks. 63.3% maintained or gained lean body mass, with mean changes of +1.89 kg lean mass and +1.41 kg weight. There was no control group.

In an open-label randomized trial at 10 Japanese hospitals, 198 analyzed patients with gastric cancer cachexia on chemotherapy had lean body mass at 8 weeks that rose 0.99 kg on anamorelin versus 0.14 kg without it, a difference that missed significance (p=0.063). Ono funded the trial.

ONO-7643-04: 174 Japanese patients, 12 weeks, lean body mass +1.38 versus -0.17 kg

Randomized controlled trial

Katakami N et al. Cancer. 2018;124(3):606-616. PMID 29205286.

Least-squares mean lean body mass change was 1.38 versus -0.17 kg. Weight and anorexia symptoms improved, but handgrip strength and 6-minute walk did not.

Participants / model
174 Japanese patients with unresectable stage III or IV non-small cell lung cancer and cachexia
Treatment
Oral anamorelin 100 mg daily or placebo
Follow-up
12 weeks
Study design
Double-blind randomized phase 2 trial in Japan (approval basis)
Funding
not stated in the source

This randomized phase 2 trial is one of the two trials named as the basis of the Japan approval.

Read the original source
ONO-7643-05: 50 Japanese patients, 12 weeks, single-arm, no control group

Clinical trial

Hamauchi S et al. Cancer. 2019;125(23):4294-4302. PMID 31415709.

63.3% maintained or gained lean body mass, with mean changes of +1.89 kg lean mass and +1.41 kg weight. Adverse events occurred in 79.6%, and treatment-related events included raised GGT (8.2%), diabetes mellitus (6.1%), hyperglycemia (6.1%) and prolonged QRS (6.1%).

Participants / model
50 Japanese patients with advanced colorectal, gastric or pancreatic cancer and cachexia
Treatment
Oral anamorelin 100 mg daily
Follow-up
12 weeks
Study design
Open-label single-arm phase 3 study (approval basis)
Funding
not stated in the source

No control group. This uncontrolled study is one of the two trials named as the basis of the Japan approval.

Read the original source
Gastric cancer cachexia: 198 analyzed patients, 8 weeks, lean body mass +0.99 versus +0.14 kg, p=0.063

Randomized controlled trial

Yamamoto K et al. EClinicalMedicine. 2025;88:103500. PMID 41181834.

Lean body mass at 8 weeks rose 0.99 kg on anamorelin versus 0.14 kg without it, a difference that missed significance (p=0.063). Hyperglycemia occurred only on anamorelin (grade 1 to 2 in 4%, grade 3 in 1%).

Participants / model
198 analyzed patients with unresectable or recurrent gastric cancer and cachexia on chemotherapy, at 10 Japanese hospitals
Treatment
Oral anamorelin 100 mg daily for 12 weeks, or no anamorelin
Follow-up
Lean body mass assessed at 8 weeks; treatment 12 weeks
Study design
Open-label randomized trial
Funding
Funded by Ono

Open-label, with no placebo. The lean body mass difference missed significance.

Read the original source

What did the later US phase 3 trials report?

Two later US-led phase 3 trials (318 randomized each) posted results on ClinicalTrials.gov. The weight difference over placebo was 1.345 kg in one and 1.284 kg in the other, but the anorexia-symptom co-primary did not differ in either, and about half of each arm did not complete. These results are sponsor-posted and not found in PubMed.

In ANAM-17-20 (NCT03743051, 318 randomized), weight rose 1.938 versus 0.594 kg over 12 weeks (difference 1.345 kg, 95% CI 0.718 to 1.971), but the anorexia-symptom co-primary did not differ (difference 0.622, p=0.1514). ANAM-17-21 (NCT03743064, 318 randomized) matched it: the weight difference was 1.284 kg (95% CI 0.718 to 1.851) and there was no anorexia-symptom difference (p=0.7241). The participants were adults with advanced non-small cell lung cancer and weight loss with anorexia.

About half of each arm did not complete. The results were posted by the sponsor on ClinicalTrials.gov, and a peer-reviewed report was not found in PubMed.

US phase 3 registry results: 318 randomized each, weight difference 1.345 and 1.284 kg, anorexia score unchanged

Trial registry record

ClinicalTrials.gov. NCT03743051 (ANAM-17-20), with the matching record NCT03743064 (ANAM-17-21). Results posted by the sponsor; not found in PubMed.

In ANAM-17-20 (NCT03743051, 318 randomized), weight rose 1.938 versus 0.594 kg over 12 weeks (difference 1.345 kg, 95% CI 0.718 to 1.971), but the anorexia-symptom co-primary did not differ (difference 0.622, p=0.1514). ANAM-17-21 (NCT03743064, 318 randomized) matched it: weight difference 1.284 kg (95% CI 0.718 to 1.851) and no anorexia-symptom difference (p=0.7241). About half of each arm did not complete.

Participants / model
Adults with advanced non-small cell lung cancer and weight loss with anorexia
Treatment
Oral anamorelin 100 mg daily or placebo
Follow-up
12 weeks
Study design
Two randomized phase 3 trials with sponsor-posted registry results
Funding
not stated in the source

Sponsor-submitted registry results, not peer reviewed and not found in PubMed. Weight and the 5-item anorexia symptom score were the co-primary endpoints.

Read the original source

Does the supplier description hold up, and what is known outside cancer?

Mostly. The Japan approval (Adlumiz, 22 January 2021, four cancer types) and the ROMANA results are accurately described, and the EMA refused authorization in 2017. Any claim of muscle, strength or growth hormone benefit in healthy people has no trial behind it.

The supplier description is of an oral ghrelin receptor agonist approved in Japan as Adlumiz for cancer cachexia, supported by the ROMANA phase 3 trials, with an EMA decision to confirm. The Japan approval and the ROMANA results are accurately described. The EMA decision is a refusal: authorization was refused on 16 November 2017.

Use outside cancer is thin. In healthy young men, single doses of 25 and 50 mg raised hunger and increased caloric intake by 18.4% versus placebo in a 9-person crossover study. In a 1-year pilot trial of 26 adults aged 50 and older with osteosarcopenia, anamorelin raised IGF-1 by 50% but did not change muscle mass or lean body mass.

A PubMed search on 10 October 2026 for anamorelin with healthy, volunteers, athletes, resistance training, sarcopenia or older adults, excluding cancer in the title, returned 26 records. None was a trial in healthy people or athletes testing muscle gain. Any claim of muscle, strength or growth hormone benefit in healthy people therefore has no trial behind it.

Company statement, 22 January 2021: Adlumiz approved in Japan for cancer cachexia in four cancer types

Regulatory record

Ono Pharmaceutical Co., Ltd., with Helsinn. News release dated 22 January 2021 on manufacturing and marketing approval of Adlumiz (anamorelin hydrochloride) in Japan.

The company said Ono received manufacturing and marketing approval for Adlumiz (anamorelin hydrochloride) tablets for cancer cachexia in non-small cell lung cancer, gastric cancer, pancreatic cancer and colorectal cancer, for adults. The basis named was a randomized, placebo-controlled phase 2 trial in lung cancer (ONO-7643-04) and an open-label, uncontrolled phase 3 study in gastrointestinal cancers (ONO-7643-05). A 2023 review gives the same approval date.

Participants / model
Adults in Japan with cachexia from these four cancers
Treatment
Oral tablet
Follow-up
Statement dated 22 January 2021
Study design
Company statement
Funding
not stated in the source

A December 2020 editorial gave 11 December 2020, which is likely a pre-approval step and is not used here. No PMDA document was read, and the Japanese label's cardiac contraindications and warnings were not read.

Read the original source
EMA: negative CHMP opinion 18 May 2017, authorization refused 16 November 2017

Regulatory record

European Medicines Agency. Adlumiz (anamorelin), applicant Helsinn Birex Pharmaceuticals, European public assessment report summary page.

The EMA page says the medicine has been refused authorisation. The CHMP gave a negative opinion on 18 May 2017, the refusal was confirmed on re-examination on 14 September 2017, and marketing authorisation was refused on 16 November 2017. The CHMP cited a marginal effect on lean body mass, no proven effect on handgrip strength or quality of life, and safety data that had not been recorded adequately at inspected trial sites.

Participants / model
Not a clinical population
Treatment
Oral anamorelin hydrochloride
Follow-up
Opinion 18 May 2017; refusal 16 November 2017
Study design
Regulator record
Funding
not stated in the source

The dates come from the EPAR summary page, not from the full refusal assessment report.

Read the original source
ROMANA 1 and 2: 484 and 495 patients, 12 weeks, lean body mass up but handgrip strength unchanged

Randomized controlled trial

Temel JS et al. Lancet Oncol. 2016;17(4):519-531. PMID 26906526.

Lean body mass rose (median 0.99 versus -0.47 kg in ROMANA 1; 0.65 versus -0.98 kg in ROMANA 2), but handgrip strength, the other co-primary endpoint, did not differ (p=0.15 and p=0.65). Grade 3 to 4 treatment-related events did not differ, and hyperglycaemia was the most common grade 3 to 4 event (under 1% and 1%).

Participants / model
484 and 495 adults with inoperable stage III or IV non-small-cell lung cancer and cachexia, at 93 sites in 19 countries
Treatment
Oral anamorelin 100 mg daily or placebo, randomized 2:1
Follow-up
12 weeks
Study design
Two double-blind randomized phase 3 trials (NCT01387269 and NCT01387282)
Funding
Funded by Helsinn

Lean body mass and handgrip strength were the co-primary endpoints; only lean body mass improved.

Read the original source
Outside cancer: 9-person crossover, 26-adult pilot, and a PubMed search with no muscle-building trial

PubMed search record

Dawson-Hughes B et al. J Clin Endocrinol Metab. 2024;109(3):e945-e955. PMID 38057159.

In healthy young men, single doses of 25 and 50 mg raised hunger and increased caloric intake by 18.4% versus placebo in a 9-person crossover study. In a 1-year pilot trial of 26 adults aged 50 and older with osteosarcopenia, anamorelin raised IGF-1 by 50% but did not change muscle mass or lean body mass. No trial in healthy people or athletes testing muscle gain was found in the PubMed search, which returned 26 records.

Participants / model
16 and 9 healthy young men in two phase 1 trials; 26 adults with osteosarcopenia; records reviewed
Treatment
Oral anamorelin, single doses or daily for 1 year
Follow-up
Single doses; 1 year; search run on 10 October 2026
Study design
Phase 1 trials, a pilot randomized trial and a dated literature search
Funding
not stated in the source

A search result shows what PubMed returned on that date. It is not proof that no unindexed study exists.

Read the original source

How does anamorelin work, and what is known about its exposure?

Anamorelin is described as an oral ghrelin receptor agonist. In trials it raised weight and lean body mass, and in healthy young men it raised hunger and caloric intake. No half-life, Tmax or other pharmacokinetic value appears in the sources reviewed.

Anamorelin is a small, orally active non-peptide molecule described as an oral ghrelin receptor agonist. The effects in the trials are on weight and lean body mass in cancer cachexia, with no gain in handgrip strength. In a 9-person crossover in healthy young men, single doses of 25 and 50 mg raised hunger and increased caloric intake by 18.4% versus placebo, and in a 1-year pilot trial of 26 adults with osteosarcopenia it raised IGF-1 by 50% without changing muscle mass.

No half-life, Tmax or other pharmacokinetic value appears in the sources reviewed. The approved tablet contains the hydrochloride, and a vendor product may be the free base or the hydrochloride.

PubChem: anamorelin and anamorelin hydrochloride identity

Chemical identity record

National Library of Medicine. PubChem compound records CID 9828911 (anamorelin) and CID 16072155 (anamorelin hydrochloride).

Anamorelin is a small, orally active non-peptide molecule. PubChem lists the free base as CID 9828911, C31H42N6O3, molecular weight 546.7, CAS 249921-19-5 (synonyms RC-1291, ONO-7643, ST-1291), and anamorelin hydrochloride, the form in the approved tablet, as CID 16072155, C31H43ClN6O3, molecular weight 583.2, CAS 861998-00-7.

Participants / model
Chemical identity, not a clinical population
Treatment
Defined small molecule and hydrochloride salt
Follow-up
Not applicable
Study design
NLM chemical record
Funding
not stated in the source

A vendor product may be either form. Doses in the trials are for the hydrochloride.

Read the original source
Outside cancer: 9-person crossover, 26-adult pilot, and a PubMed search with no muscle-building trial

PubMed search record

Dawson-Hughes B et al. J Clin Endocrinol Metab. 2024;109(3):e945-e955. PMID 38057159.

In healthy young men, single doses of 25 and 50 mg raised hunger and increased caloric intake by 18.4% versus placebo in a 9-person crossover study. In a 1-year pilot trial of 26 adults aged 50 and older with osteosarcopenia, anamorelin raised IGF-1 by 50% but did not change muscle mass or lean body mass. No trial in healthy people or athletes testing muscle gain was found in the PubMed search, which returned 26 records.

Participants / model
16 and 9 healthy young men in two phase 1 trials; 26 adults with osteosarcopenia; records reviewed
Treatment
Oral anamorelin, single doses or daily for 1 year
Follow-up
Single doses; 1 year; search run on 10 October 2026
Study design
Phase 1 trials, a pilot randomized trial and a dated literature search
Funding
not stated in the source

A search result shows what PubMed returned on that date. It is not proof that no unindexed study exists.

Read the original source

What adverse events and warnings are reported?

Hyperglycemia was the most common grade 3 to 4 event in ROMANA 1 and 2 and occurred only on anamorelin in a Japanese gastric cancer trial. A Japanese adverse event database analysis (537 reports) found an adjusted reporting odds ratio of 20.00 for conduction defects, a measure of disproportionate reporting and not incidence. The EMA cited safety data not recorded adequately at inspected trial sites.

In ROMANA 1 and 2, grade 3 to 4 treatment-related events did not differ between groups, and hyperglycaemia was the most common grade 3 to 4 event (under 1% and 1%). In the ROMANA 3 extension, treatment-emergent adverse events (52.2% versus 55.7%), grade 3 or higher events (22.4% versus 21.6%) and serious events (12.8% versus 12.6%) were similar, and 36 and 23 deaths were judged unrelated. In the meta-analysis, adverse events did not differ.

In the 50-patient single-arm study ONO-7643-05, adverse events occurred in 79.6%, and treatment-related events included raised GGT (8.2%), diabetes mellitus (6.1%), hyperglycemia (6.1%) and prolonged QRS (6.1%). In the Japanese gastric cancer trial, hyperglycemia occurred only on anamorelin (grade 1 to 2 in 4%, grade 3 in 1%).

In the Japanese adverse event database (JADER, 537 anamorelin reports through December 2023), the adjusted reporting odds ratio for conduction defects was 20.00 (95% CI 14.86 to 26.91), with a median onset of 13 days. The literature review found seven cases, all within about three weeks of starting and all recovered. Reporting odds ratios measure disproportionate reporting, not incidence. The Japanese label's cardiac contraindications and warnings were not read for this page.

The EMA cited safety data that had not been recorded adequately at inspected trial sites. About half of each arm in the two US phase 3 trials did not complete.

ROMANA 1 and 2: 484 and 495 patients, 12 weeks, lean body mass up but handgrip strength unchanged

Randomized controlled trial

Temel JS et al. Lancet Oncol. 2016;17(4):519-531. PMID 26906526.

Lean body mass rose (median 0.99 versus -0.47 kg in ROMANA 1; 0.65 versus -0.98 kg in ROMANA 2), but handgrip strength, the other co-primary endpoint, did not differ (p=0.15 and p=0.65). Grade 3 to 4 treatment-related events did not differ, and hyperglycaemia was the most common grade 3 to 4 event (under 1% and 1%).

Participants / model
484 and 495 adults with inoperable stage III or IV non-small-cell lung cancer and cachexia, at 93 sites in 19 countries
Treatment
Oral anamorelin 100 mg daily or placebo, randomized 2:1
Follow-up
12 weeks
Study design
Two double-blind randomized phase 3 trials (NCT01387269 and NCT01387282)
Funding
Funded by Helsinn

Lean body mass and handgrip strength were the co-primary endpoints; only lean body mass improved.

Read the original source
ROMANA 3: 513 completers, weeks 12 to 24, similar adverse events, no handgrip gain

Randomized controlled trial

Currow D et al. Ann Oncol. 2017;28(8):1949-1956. PMID 28472437.

Treatment-emergent adverse events (52.2% versus 55.7%), grade 3 or higher events (22.4% versus 21.6%) and serious events (12.8% versus 12.6%) were similar, and 36 and 23 deaths were judged unrelated. Body weight stayed higher on anamorelin through 24 weeks, with no handgrip improvement.

Participants / model
513 of 703 ROMANA completers with preserved performance status
Treatment
Oral anamorelin 100 mg daily (345) or placebo (168) for weeks 12 to 24
Follow-up
Weeks 12 to 24 of ROMANA
Study design
Randomized safety extension (NCT01395914)
Funding
not stated in the source

Only participants who completed ROMANA 1 or 2 with preserved performance status continued.

Read the original source
Meta-analysis: 6 trials, 1,641 patients, weight +1.78 kg, no survival or handgrip difference

Systematic review and meta-analysis

Nishie K et al. Lung Cancer. 2017;112:25-34. PMID 29191597.

Anamorelin gave higher total body weight (mean difference 1.78 kg, 95% CI 1.28 to 2.28) and lean body mass (1.10 kg, 95% CI 0.35 to 1.85), and a small quality-of-life gain (standardized mean difference 0.19). There was no difference in overall survival (HR 0.99) or handgrip strength, and adverse events did not differ.

Participants / model
1,641 patients with advanced non-small cell lung cancer and cachexia in 6 pooled randomized trials
Treatment
Oral anamorelin
Follow-up
Pooled trials
Study design
Systematic review and meta-analysis of 6 randomized trials
Funding
not stated in the source

Pooled outcomes were weight, lean mass, survival, handgrip strength and quality of life.

Read the original source
ONO-7643-05: 50 Japanese patients, 12 weeks, single-arm, no control group

Clinical trial

Hamauchi S et al. Cancer. 2019;125(23):4294-4302. PMID 31415709.

63.3% maintained or gained lean body mass, with mean changes of +1.89 kg lean mass and +1.41 kg weight. Adverse events occurred in 79.6%, and treatment-related events included raised GGT (8.2%), diabetes mellitus (6.1%), hyperglycemia (6.1%) and prolonged QRS (6.1%).

Participants / model
50 Japanese patients with advanced colorectal, gastric or pancreatic cancer and cachexia
Treatment
Oral anamorelin 100 mg daily
Follow-up
12 weeks
Study design
Open-label single-arm phase 3 study (approval basis)
Funding
not stated in the source

No control group. This uncontrolled study is one of the two trials named as the basis of the Japan approval.

Read the original source
Gastric cancer cachexia: 198 analyzed patients, 8 weeks, lean body mass +0.99 versus +0.14 kg, p=0.063

Randomized controlled trial

Yamamoto K et al. EClinicalMedicine. 2025;88:103500. PMID 41181834.

Lean body mass at 8 weeks rose 0.99 kg on anamorelin versus 0.14 kg without it, a difference that missed significance (p=0.063). Hyperglycemia occurred only on anamorelin (grade 1 to 2 in 4%, grade 3 in 1%).

Participants / model
198 analyzed patients with unresectable or recurrent gastric cancer and cachexia on chemotherapy, at 10 Japanese hospitals
Treatment
Oral anamorelin 100 mg daily for 12 weeks, or no anamorelin
Follow-up
Lean body mass assessed at 8 weeks; treatment 12 weeks
Study design
Open-label randomized trial
Funding
Funded by Ono

Open-label, with no placebo. The lean body mass difference missed significance.

Read the original source
JADER: 537 reports, reporting odds ratio 20.00 for conduction defects, median onset 13 days

Human study

Yabuki N et al. In Vivo. 2025;39(1):404-410. PMID 39740881.

In the Japanese adverse event database (JADER, 537 anamorelin reports through December 2023), the adjusted reporting odds ratio for conduction defects was 20.00 (95% CI 14.86 to 26.91), with a median onset of 13 days. The literature review found seven cases, all within about three weeks of starting and all recovered.

Participants / model
Japanese patients in spontaneous reports and published case reports
Treatment
Oral anamorelin in routine care
Follow-up
Reports through December 2023
Study design
Pharmacovigilance analysis and literature review
Funding
not stated in the source

Reporting odds ratios measure disproportionate reporting, not incidence.

Read the original source
EMA: negative CHMP opinion 18 May 2017, authorization refused 16 November 2017

Regulatory record

European Medicines Agency. Adlumiz (anamorelin), applicant Helsinn Birex Pharmaceuticals, European public assessment report summary page.

The EMA page says the medicine has been refused authorisation. The CHMP gave a negative opinion on 18 May 2017, the refusal was confirmed on re-examination on 14 September 2017, and marketing authorisation was refused on 16 November 2017. The CHMP cited a marginal effect on lean body mass, no proven effect on handgrip strength or quality of life, and safety data that had not been recorded adequately at inspected trial sites.

Participants / model
Not a clinical population
Treatment
Oral anamorelin hydrochloride
Follow-up
Opinion 18 May 2017; refusal 16 November 2017
Study design
Regulator record
Funding
not stated in the source

The dates come from the EPAR summary page, not from the full refusal assessment report.

Read the original source
US phase 3 registry results: 318 randomized each, weight difference 1.345 and 1.284 kg, anorexia score unchanged

Trial registry record

ClinicalTrials.gov. NCT03743051 (ANAM-17-20), with the matching record NCT03743064 (ANAM-17-21). Results posted by the sponsor; not found in PubMed.

In ANAM-17-20 (NCT03743051, 318 randomized), weight rose 1.938 versus 0.594 kg over 12 weeks (difference 1.345 kg, 95% CI 0.718 to 1.971), but the anorexia-symptom co-primary did not differ (difference 0.622, p=0.1514). ANAM-17-21 (NCT03743064, 318 randomized) matched it: weight difference 1.284 kg (95% CI 0.718 to 1.851) and no anorexia-symptom difference (p=0.7241). About half of each arm did not complete.

Participants / model
Adults with advanced non-small cell lung cancer and weight loss with anorexia
Treatment
Oral anamorelin 100 mg daily or placebo
Follow-up
12 weeks
Study design
Two randomized phase 3 trials with sponsor-posted registry results
Funding
not stated in the source

Sponsor-submitted registry results, not peer reviewed and not found in PubMed. Weight and the 5-item anorexia symptom score were the co-primary endpoints.

Read the original source

Why does anamorelin sit in B tier?

Across four placebo-controlled phase 3 trials, anamorelin added about 1 to 2 kg of weight or lean mass in 12 weeks, but it never improved handgrip strength, missed the anorexia-symptom co-primary in both later US trials, did not improve survival, and was refused by the EMA. It is approved only in Japan, where the main approval trials were a phase 2 and an uncontrolled study.

The B reflects a drug with a consistent mechanism description and mixed outcomes. The weight and lean mass gains are reproducible across trials, but handgrip strength never improved, the anorexia-symptom co-primary was missed in both later US trials, and pooled survival did not differ.

It is not the footing of an FDA-approved drug: there is no US record, the EMA refused authorization in 2017, and Japan's approval rested mainly on a randomized phase 2 trial and an uncontrolled study. The grade does not cover muscle, strength or growth hormone claims in healthy people, which no trial found tests.

ROMANA 1 and 2: 484 and 495 patients, 12 weeks, lean body mass up but handgrip strength unchanged

Randomized controlled trial

Temel JS et al. Lancet Oncol. 2016;17(4):519-531. PMID 26906526.

Lean body mass rose (median 0.99 versus -0.47 kg in ROMANA 1; 0.65 versus -0.98 kg in ROMANA 2), but handgrip strength, the other co-primary endpoint, did not differ (p=0.15 and p=0.65). Grade 3 to 4 treatment-related events did not differ, and hyperglycaemia was the most common grade 3 to 4 event (under 1% and 1%).

Participants / model
484 and 495 adults with inoperable stage III or IV non-small-cell lung cancer and cachexia, at 93 sites in 19 countries
Treatment
Oral anamorelin 100 mg daily or placebo, randomized 2:1
Follow-up
12 weeks
Study design
Two double-blind randomized phase 3 trials (NCT01387269 and NCT01387282)
Funding
Funded by Helsinn

Lean body mass and handgrip strength were the co-primary endpoints; only lean body mass improved.

Read the original source
US phase 3 registry results: 318 randomized each, weight difference 1.345 and 1.284 kg, anorexia score unchanged

Trial registry record

ClinicalTrials.gov. NCT03743051 (ANAM-17-20), with the matching record NCT03743064 (ANAM-17-21). Results posted by the sponsor; not found in PubMed.

In ANAM-17-20 (NCT03743051, 318 randomized), weight rose 1.938 versus 0.594 kg over 12 weeks (difference 1.345 kg, 95% CI 0.718 to 1.971), but the anorexia-symptom co-primary did not differ (difference 0.622, p=0.1514). ANAM-17-21 (NCT03743064, 318 randomized) matched it: weight difference 1.284 kg (95% CI 0.718 to 1.851) and no anorexia-symptom difference (p=0.7241). About half of each arm did not complete.

Participants / model
Adults with advanced non-small cell lung cancer and weight loss with anorexia
Treatment
Oral anamorelin 100 mg daily or placebo
Follow-up
12 weeks
Study design
Two randomized phase 3 trials with sponsor-posted registry results
Funding
not stated in the source

Sponsor-submitted registry results, not peer reviewed and not found in PubMed. Weight and the 5-item anorexia symptom score were the co-primary endpoints.

Read the original source
Meta-analysis: 6 trials, 1,641 patients, weight +1.78 kg, no survival or handgrip difference

Systematic review and meta-analysis

Nishie K et al. Lung Cancer. 2017;112:25-34. PMID 29191597.

Anamorelin gave higher total body weight (mean difference 1.78 kg, 95% CI 1.28 to 2.28) and lean body mass (1.10 kg, 95% CI 0.35 to 1.85), and a small quality-of-life gain (standardized mean difference 0.19). There was no difference in overall survival (HR 0.99) or handgrip strength, and adverse events did not differ.

Participants / model
1,641 patients with advanced non-small cell lung cancer and cachexia in 6 pooled randomized trials
Treatment
Oral anamorelin
Follow-up
Pooled trials
Study design
Systematic review and meta-analysis of 6 randomized trials
Funding
not stated in the source

Pooled outcomes were weight, lean mass, survival, handgrip strength and quality of life.

Read the original source
EMA: negative CHMP opinion 18 May 2017, authorization refused 16 November 2017

Regulatory record

European Medicines Agency. Adlumiz (anamorelin), applicant Helsinn Birex Pharmaceuticals, European public assessment report summary page.

The EMA page says the medicine has been refused authorisation. The CHMP gave a negative opinion on 18 May 2017, the refusal was confirmed on re-examination on 14 September 2017, and marketing authorisation was refused on 16 November 2017. The CHMP cited a marginal effect on lean body mass, no proven effect on handgrip strength or quality of life, and safety data that had not been recorded adequately at inspected trial sites.

Participants / model
Not a clinical population
Treatment
Oral anamorelin hydrochloride
Follow-up
Opinion 18 May 2017; refusal 16 November 2017
Study design
Regulator record
Funding
not stated in the source

The dates come from the EPAR summary page, not from the full refusal assessment report.

Read the original source
Company statement, 22 January 2021: Adlumiz approved in Japan for cancer cachexia in four cancer types

Regulatory record

Ono Pharmaceutical Co., Ltd., with Helsinn. News release dated 22 January 2021 on manufacturing and marketing approval of Adlumiz (anamorelin hydrochloride) in Japan.

The company said Ono received manufacturing and marketing approval for Adlumiz (anamorelin hydrochloride) tablets for cancer cachexia in non-small cell lung cancer, gastric cancer, pancreatic cancer and colorectal cancer, for adults. The basis named was a randomized, placebo-controlled phase 2 trial in lung cancer (ONO-7643-04) and an open-label, uncontrolled phase 3 study in gastrointestinal cancers (ONO-7643-05). A 2023 review gives the same approval date.

Participants / model
Adults in Japan with cachexia from these four cancers
Treatment
Oral tablet
Follow-up
Statement dated 22 January 2021
Study design
Company statement
Funding
not stated in the source

A December 2020 editorial gave 11 December 2020, which is likely a pre-approval step and is not used here. No PMDA document was read, and the Japanese label's cardiac contraindications and warnings were not read.

Read the original source

Studies and sources

PubChem: anamorelin and anamorelin hydrochloride identity

Chemical identity record

National Library of Medicine. PubChem compound records CID 9828911 (anamorelin) and CID 16072155 (anamorelin hydrochloride).

Anamorelin is a small, orally active non-peptide molecule. PubChem lists the free base as CID 9828911, C31H42N6O3, molecular weight 546.7, CAS 249921-19-5 (synonyms RC-1291, ONO-7643, ST-1291), and anamorelin hydrochloride, the form in the approved tablet, as CID 16072155, C31H43ClN6O3, molecular weight 583.2, CAS 861998-00-7.

Participants / model
Chemical identity, not a clinical population
Treatment
Defined small molecule and hydrochloride salt
Follow-up
Not applicable
Study design
NLM chemical record
Funding
not stated in the source

A vendor product may be either form. Doses in the trials are for the hydrochloride.

Read the original source
Company statement, 22 January 2021: Adlumiz approved in Japan for cancer cachexia in four cancer types

Regulatory record

Ono Pharmaceutical Co., Ltd., with Helsinn. News release dated 22 January 2021 on manufacturing and marketing approval of Adlumiz (anamorelin hydrochloride) in Japan.

The company said Ono received manufacturing and marketing approval for Adlumiz (anamorelin hydrochloride) tablets for cancer cachexia in non-small cell lung cancer, gastric cancer, pancreatic cancer and colorectal cancer, for adults. The basis named was a randomized, placebo-controlled phase 2 trial in lung cancer (ONO-7643-04) and an open-label, uncontrolled phase 3 study in gastrointestinal cancers (ONO-7643-05). A 2023 review gives the same approval date.

Participants / model
Adults in Japan with cachexia from these four cancers
Treatment
Oral tablet
Follow-up
Statement dated 22 January 2021
Study design
Company statement
Funding
not stated in the source

A December 2020 editorial gave 11 December 2020, which is likely a pre-approval step and is not used here. No PMDA document was read, and the Japanese label's cardiac contraindications and warnings were not read.

Read the original source
EMA: negative CHMP opinion 18 May 2017, authorization refused 16 November 2017

Regulatory record

European Medicines Agency. Adlumiz (anamorelin), applicant Helsinn Birex Pharmaceuticals, European public assessment report summary page.

The EMA page says the medicine has been refused authorisation. The CHMP gave a negative opinion on 18 May 2017, the refusal was confirmed on re-examination on 14 September 2017, and marketing authorisation was refused on 16 November 2017. The CHMP cited a marginal effect on lean body mass, no proven effect on handgrip strength or quality of life, and safety data that had not been recorded adequately at inspected trial sites.

Participants / model
Not a clinical population
Treatment
Oral anamorelin hydrochloride
Follow-up
Opinion 18 May 2017; refusal 16 November 2017
Study design
Regulator record
Funding
not stated in the source

The dates come from the EPAR summary page, not from the full refusal assessment report.

Read the original source
ROMANA 1 and 2: 484 and 495 patients, 12 weeks, lean body mass up but handgrip strength unchanged

Randomized controlled trial

Temel JS et al. Lancet Oncol. 2016;17(4):519-531. PMID 26906526.

Lean body mass rose (median 0.99 versus -0.47 kg in ROMANA 1; 0.65 versus -0.98 kg in ROMANA 2), but handgrip strength, the other co-primary endpoint, did not differ (p=0.15 and p=0.65). Grade 3 to 4 treatment-related events did not differ, and hyperglycaemia was the most common grade 3 to 4 event (under 1% and 1%).

Participants / model
484 and 495 adults with inoperable stage III or IV non-small-cell lung cancer and cachexia, at 93 sites in 19 countries
Treatment
Oral anamorelin 100 mg daily or placebo, randomized 2:1
Follow-up
12 weeks
Study design
Two double-blind randomized phase 3 trials (NCT01387269 and NCT01387282)
Funding
Funded by Helsinn

Lean body mass and handgrip strength were the co-primary endpoints; only lean body mass improved.

Read the original source
ROMANA 3: 513 completers, weeks 12 to 24, similar adverse events, no handgrip gain

Randomized controlled trial

Currow D et al. Ann Oncol. 2017;28(8):1949-1956. PMID 28472437.

Treatment-emergent adverse events (52.2% versus 55.7%), grade 3 or higher events (22.4% versus 21.6%) and serious events (12.8% versus 12.6%) were similar, and 36 and 23 deaths were judged unrelated. Body weight stayed higher on anamorelin through 24 weeks, with no handgrip improvement.

Participants / model
513 of 703 ROMANA completers with preserved performance status
Treatment
Oral anamorelin 100 mg daily (345) or placebo (168) for weeks 12 to 24
Follow-up
Weeks 12 to 24 of ROMANA
Study design
Randomized safety extension (NCT01395914)
Funding
not stated in the source

Only participants who completed ROMANA 1 or 2 with preserved performance status continued.

Read the original source
ONO-7643-04: 174 Japanese patients, 12 weeks, lean body mass +1.38 versus -0.17 kg

Randomized controlled trial

Katakami N et al. Cancer. 2018;124(3):606-616. PMID 29205286.

Least-squares mean lean body mass change was 1.38 versus -0.17 kg. Weight and anorexia symptoms improved, but handgrip strength and 6-minute walk did not.

Participants / model
174 Japanese patients with unresectable stage III or IV non-small cell lung cancer and cachexia
Treatment
Oral anamorelin 100 mg daily or placebo
Follow-up
12 weeks
Study design
Double-blind randomized phase 2 trial in Japan (approval basis)
Funding
not stated in the source

This randomized phase 2 trial is one of the two trials named as the basis of the Japan approval.

Read the original source
ONO-7643-05: 50 Japanese patients, 12 weeks, single-arm, no control group

Clinical trial

Hamauchi S et al. Cancer. 2019;125(23):4294-4302. PMID 31415709.

63.3% maintained or gained lean body mass, with mean changes of +1.89 kg lean mass and +1.41 kg weight. Adverse events occurred in 79.6%, and treatment-related events included raised GGT (8.2%), diabetes mellitus (6.1%), hyperglycemia (6.1%) and prolonged QRS (6.1%).

Participants / model
50 Japanese patients with advanced colorectal, gastric or pancreatic cancer and cachexia
Treatment
Oral anamorelin 100 mg daily
Follow-up
12 weeks
Study design
Open-label single-arm phase 3 study (approval basis)
Funding
not stated in the source

No control group. This uncontrolled study is one of the two trials named as the basis of the Japan approval.

Read the original source
US phase 3 registry results: 318 randomized each, weight difference 1.345 and 1.284 kg, anorexia score unchanged

Trial registry record

ClinicalTrials.gov. NCT03743051 (ANAM-17-20), with the matching record NCT03743064 (ANAM-17-21). Results posted by the sponsor; not found in PubMed.

In ANAM-17-20 (NCT03743051, 318 randomized), weight rose 1.938 versus 0.594 kg over 12 weeks (difference 1.345 kg, 95% CI 0.718 to 1.971), but the anorexia-symptom co-primary did not differ (difference 0.622, p=0.1514). ANAM-17-21 (NCT03743064, 318 randomized) matched it: weight difference 1.284 kg (95% CI 0.718 to 1.851) and no anorexia-symptom difference (p=0.7241). About half of each arm did not complete.

Participants / model
Adults with advanced non-small cell lung cancer and weight loss with anorexia
Treatment
Oral anamorelin 100 mg daily or placebo
Follow-up
12 weeks
Study design
Two randomized phase 3 trials with sponsor-posted registry results
Funding
not stated in the source

Sponsor-submitted registry results, not peer reviewed and not found in PubMed. Weight and the 5-item anorexia symptom score were the co-primary endpoints.

Read the original source
Meta-analysis: 6 trials, 1,641 patients, weight +1.78 kg, no survival or handgrip difference

Systematic review and meta-analysis

Nishie K et al. Lung Cancer. 2017;112:25-34. PMID 29191597.

Anamorelin gave higher total body weight (mean difference 1.78 kg, 95% CI 1.28 to 2.28) and lean body mass (1.10 kg, 95% CI 0.35 to 1.85), and a small quality-of-life gain (standardized mean difference 0.19). There was no difference in overall survival (HR 0.99) or handgrip strength, and adverse events did not differ.

Participants / model
1,641 patients with advanced non-small cell lung cancer and cachexia in 6 pooled randomized trials
Treatment
Oral anamorelin
Follow-up
Pooled trials
Study design
Systematic review and meta-analysis of 6 randomized trials
Funding
not stated in the source

Pooled outcomes were weight, lean mass, survival, handgrip strength and quality of life.

Read the original source
Gastric cancer cachexia: 198 analyzed patients, 8 weeks, lean body mass +0.99 versus +0.14 kg, p=0.063

Randomized controlled trial

Yamamoto K et al. EClinicalMedicine. 2025;88:103500. PMID 41181834.

Lean body mass at 8 weeks rose 0.99 kg on anamorelin versus 0.14 kg without it, a difference that missed significance (p=0.063). Hyperglycemia occurred only on anamorelin (grade 1 to 2 in 4%, grade 3 in 1%).

Participants / model
198 analyzed patients with unresectable or recurrent gastric cancer and cachexia on chemotherapy, at 10 Japanese hospitals
Treatment
Oral anamorelin 100 mg daily for 12 weeks, or no anamorelin
Follow-up
Lean body mass assessed at 8 weeks; treatment 12 weeks
Study design
Open-label randomized trial
Funding
Funded by Ono

Open-label, with no placebo. The lean body mass difference missed significance.

Read the original source
JADER: 537 reports, reporting odds ratio 20.00 for conduction defects, median onset 13 days

Human study

Yabuki N et al. In Vivo. 2025;39(1):404-410. PMID 39740881.

In the Japanese adverse event database (JADER, 537 anamorelin reports through December 2023), the adjusted reporting odds ratio for conduction defects was 20.00 (95% CI 14.86 to 26.91), with a median onset of 13 days. The literature review found seven cases, all within about three weeks of starting and all recovered.

Participants / model
Japanese patients in spontaneous reports and published case reports
Treatment
Oral anamorelin in routine care
Follow-up
Reports through December 2023
Study design
Pharmacovigilance analysis and literature review
Funding
not stated in the source

Reporting odds ratios measure disproportionate reporting, not incidence.

Read the original source
Outside cancer: 9-person crossover, 26-adult pilot, and a PubMed search with no muscle-building trial

PubMed search record

Dawson-Hughes B et al. J Clin Endocrinol Metab. 2024;109(3):e945-e955. PMID 38057159.

In healthy young men, single doses of 25 and 50 mg raised hunger and increased caloric intake by 18.4% versus placebo in a 9-person crossover study. In a 1-year pilot trial of 26 adults aged 50 and older with osteosarcopenia, anamorelin raised IGF-1 by 50% but did not change muscle mass or lean body mass. No trial in healthy people or athletes testing muscle gain was found in the PubMed search, which returned 26 records.

Participants / model
16 and 9 healthy young men in two phase 1 trials; 26 adults with osteosarcopenia; records reviewed
Treatment
Oral anamorelin, single doses or daily for 1 year
Follow-up
Single doses; 1 year; search run on 10 October 2026
Study design
Phase 1 trials, a pilot randomized trial and a dated literature search
Funding
not stated in the source

A search result shows what PubMed returned on that date. It is not proof that no unindexed study exists.

Read the original source

Why is Anamorelin in B tier?

B fits a drug approved in one country on mixed evidence. Across four placebo-controlled phase 3 trials, anamorelin added about 1 to 2 kg of weight or lean mass over 12 weeks, but it never improved handgrip strength, missed the anorexia-symptom co-primary in both later US trials, did not improve survival, and was refused by the EMA. The Japan approval rested mainly on a randomized phase 2 trial and an uncontrolled study, and the grade does not cover muscle or growth hormone claims in healthy people.