This page shows what was measured, who it was measured in, and what that does not settle.
What Metreleptin does in the body
MYALEPT is a leptin analog indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy.
From the FDA-approved label: Adipocytes store lipids to meet the fuel requirements of non-adipose tissues during fasting. In patients with generalized lipodystrophy, the deficiency of adipose tissue leads to hypertriglyceridemia and ectopic deposition of fat in non-adipose tissues such as liver and muscle, contributing to metabolic abnormalities including insulin resistance. Native leptin is a hormone predominantly secreted by adipose tissue that informs the central nervous system of the status of energy stores in the body. In patients with generalized lipodystrophy, leptin deficiency, resulting from the loss of adipose tissue, contributes to excess caloric intake, which exacerbates the metabolic abnormalities.
Why people take it. MYALEPT is a leptin analog indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy.
What happened in people
RNAWiki has not yet published a reviewed conclusion for this use.
§ A fixed RNAWiki sentence
Where this came from
Wording RNAWiki always uses, not a finding about this substance.
No reviewed claim names a result for any goal on this record.
No source is stored against this line.
The limit that matters most
Not recorded.
Where it acts
Not recorded.
Kind of result
No result is published, so no kind of result applies yet
Supervision
Professional supervision is normally required. A regulator classifies this substance in a way that restricts how it is supplied.
What the registries record it as
The substance registry classes this as protein.
FDA substance registry · TL60C27RLH · read 2026-08-29
Where each sentence above came from
The use the label states, quoted from it. No plain-language version of this sentence has been written.
The use the label states, quoted from it. It is written for a clinician, not for a reader without medical training.
No statement of the main limit is recorded.
The four opening statements run to 168 words.
Words this page uses
Four words worth knowing first
Chosen from what this page shows, with each one explained before the word it depends on.
Biomarker
A biomarker is a number from a test that stands in for something about health.
A picture of it, and where the picture fails
A biomarker is like a fuel gauge.
Where that stops being true. A gauge is wired to the tank. Many biomarkers are only loosely tied to health.
What people get wrong. A better number is read as a better life. Several medicines improved a number and helped nobody.
A measurable indicator used as a substitute for a clinical outcome of interest.
Stand-in result
A stand-in result is a number measured because the real result takes too long.
A picture of it, and where the picture fails
It is like judging a journey by the speedometer rather than by arriving.
Where that stops being true. Speed does predict arrival. Many stand-in results do not predict the real one.
What people get wrong. A stand-in result is often reported as the result itself.
A surrogate endpoint substituted for a clinical endpoint, valid only where the substitution has been shown to hold.
What happened in people◇Read from sources, not yet reviewed
What was measured, goal by goal
One row for each goal a registered study measured something for. One column for each kind of thing that could be measured.
Registered studies list 10 outcome measures that RNAWiki could read. A registered study says what someone planned to measure. It does not say what they found. 0 of the matched studies tested this substance, and 0 posted a result.
There is no single score. A strong test result and a weak life result are different facts.
Goals down the side, kinds of measurement across the top. Each cell says what kind of thing was registered, not what was found.
Goal
Life outcome
What a body can do
How a person feels
A test result
A step in the body
Harms
How long
Who was studied
Blood sugar
∅Nothing in the sources checkedNo registered study lists a life outcome for this goal.
∅Nothing in the sources checkedNo registered study lists a performance measure for this goal.
∅Nothing in the sources checkedNo registered study lists a symptom measure for this goal.
△Only a number moved2 registered test measure.
∅Nothing in the sources checkedNo registered study lists a body-step measure for this goal.
—Not recordedHarms were not a registered measure for this goal.
—Not recordedNo finished study window is recorded.
…Waiting for a reviewerWho was studied is listed further down the page.
Cholesterol
∅Nothing in the sources checkedNo registered study lists a life outcome for this goal.
∅Nothing in the sources checkedNo registered study lists a performance measure for this goal.
∅Nothing in the sources checkedNo registered study lists a symptom measure for this goal.
△Only a number moved2 registered test measure.
∅Nothing in the sources checkedNo registered study lists a body-step measure for this goal.
—Not recordedHarms were not a registered measure for this goal.
—Not recordedNo finished study window is recorded.
…Waiting for a reviewerWho was studied is listed further down the page.
Body weight
∅Nothing in the sources checkedNo registered study lists a life outcome for this goal.
∅Nothing in the sources checkedNo registered study lists a performance measure for this goal.
∅Nothing in the sources checkedNo registered study lists a symptom measure for this goal.
△Only a number moved2 registered test measure.
∅Nothing in the sources checkedNo registered study lists a body-step measure for this goal.
—Not recordedHarms were not a registered measure for this goal.
—Not recordedNo finished study window is recorded.
…Waiting for a reviewerWho was studied is listed further down the page.
Energy
∅Nothing in the sources checkedNo registered study lists a life outcome for this goal.
∅Nothing in the sources checkedNo registered study lists a performance measure for this goal.
∅Nothing in the sources checkedNo registered study lists a symptom measure for this goal.
∅Nothing in the sources checkedNo registered study lists a test result for this goal.
∅Nothing in the sources checkedNo registered study lists a body-step measure for this goal.
—Not recordedHarms were not a registered measure for this goal.
—Not recordedNo finished study window is recorded.
…Waiting for a reviewerWho was studied is listed further down the page.
Which registered measures put each goal on this table
Blood sugar
hba1c; insulin sensitivity
Cholesterol
fasting serum triglycerides; serum triglycerides
Body weight
body weight at 6 months; body composition
Energy
total energy expenditure
Sorted by fixed word lists, version v1. A name the rules do not recognise stays unsorted rather than moving to the nearest column.
What each mark on this table means
∅ Nothing in the sources checked
No registered study lists a life outcome for this goal.
△ Only a number moved
2 registered test measure.
— Not recorded
Harms were not a registered measure for this goal.
… Waiting for a reviewer
Who was studied is listed further down the page.
What happened in people◇Read from sources, not yet reviewed
What happened in people
Each card is one study: the exact question it asked, who was in it, and whether it showed what it set out to show.
Least Squares (LS) Mean Percent Change in Body Weight From Baseline to Week 28 - Evaluable Population
✗ The study did not show it
Who was studied
NCT00673387
How many people
636
Study design
Phase 2
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated
What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.
The limits of this study, and where it came from
Main limit. No limitation is recorded for this study.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Injection
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
openFDA Drugs@FDA · a recorded source, not a stored snapshot
openFDA NDC Directory · a recorded source, not a stored snapshot
openFDA SPL label · a recorded source, not a stored snapshot
FDA label (openFDA SPL) · a recorded source, not a stored snapshot
LS Mean Percent Change in Body Weight From Original Study DFA102 (NCT00673387) Baseline (Day 1) at Week 52 in Extension Study DFA102E - Evaluable Treatment Stable Population
✗ The study did not show it
Who was studied
NCT00819234
How many people
274
Study design
Phase 2
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated
What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.
The limits of this study, and where it came from
Main limit. No limitation is recorded for this study.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Injection
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
openFDA Drugs@FDA · a recorded source, not a stored snapshot
openFDA NDC Directory · a recorded source, not a stored snapshot
openFDA SPL label · a recorded source, not a stored snapshot
FDA label (openFDA SPL) · a recorded source, not a stored snapshot
Number of Participants With Treatment Emergent Adverse Events and Number With Post Treatment Adverse Events - Intent to Treat Population
✗ The study did not show it
Who was studied
NCT01235741
How many people
213
Study design
Phase 2
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated
What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.
The limits of this study, and where it came from
Main limit. No limitation is recorded for this study.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Injection
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
openFDA Drugs@FDA · a recorded source, not a stored snapshot
openFDA NDC Directory · a recorded source, not a stored snapshot
openFDA SPL label · a recorded source, not a stored snapshot
FDA label (openFDA SPL) · a recorded source, not a stored snapshot
Mean Absolute Change From Baseline to Week 16 in Body Weight - Evaluable Population
✗ The study did not show it
Who was studied
NCT00392925
How many people
177
Study design
Phase 2
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated
What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.
The limits of this study, and where it came from
Main limit. No limitation is recorded for this study.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Injection
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
openFDA Drugs@FDA · a recorded source, not a stored snapshot
openFDA NDC Directory · a recorded source, not a stored snapshot
openFDA SPL label · a recorded source, not a stored snapshot
FDA label (openFDA SPL) · a recorded source, not a stored snapshot
Percentage of Glycosylated Hemoglobin at Baseline, 6 Months, and 12 Months on Treatment With Metreleptin
✗ The study did not show it
Who was studied
NCT00025883
How many people
103
Study design
Phase 2
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated
What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.
The limits of this study, and where it came from
Main limit. No limitation is recorded for this study.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Injection
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
openFDA Drugs@FDA · a recorded source, not a stored snapshot
openFDA NDC Directory · a recorded source, not a stored snapshot
openFDA SPL label · a recorded source, not a stored snapshot
FDA label (openFDA SPL) · a recorded source, not a stored snapshot
Change from baseline to month 6 in HbA1c in subjects with partial lipodystrophy (PL)
✗ The study did not show it
Who was studied
NCT05164341
How many people
69
Study design
Phase 3
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated
What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.
The limits of this study, and where it came from
Main limit. No limitation is recorded for this study.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Injection
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
openFDA Drugs@FDA · a recorded source, not a stored snapshot
openFDA NDC Directory · a recorded source, not a stored snapshot
openFDA SPL label · a recorded source, not a stored snapshot
FDA label (openFDA SPL) · a recorded source, not a stored snapshot
What we know
RNAWiki holds 6 written-up human studies for this substance, each with the question it asked.
How we know it
Each card names the study, the number of people and what the study set out to measure.
What this does not prove
These summaries were written by a person and have not been signed off as reviewed claims.
Why it matters
A study that failed is as informative as one that worked, and both are here.
What we still do not know
No reviewed claim exists, so no exact population, effect size or interval is published yet.
What happened in people◇Read from sources, not yet reviewed
How close this is to real life
The top step is something a person would feel or care about. The bottom is a guess from software. Filled steps are where evidence exists for this substance.
□Living longer, or avoiding a major eventNo evidence recorded. Death, a heart attack, a stroke, a hospital stay.No registered study measures this.
□What a body can do day to dayNo evidence recorded. Walking, dressing, breathing, recovering.No registered study measures this.
□Measured performanceNo evidence recorded. How much was lifted, how far was run, how fast.No registered study measures this.
□Symptoms and quality of lifeNo evidence recorded. Pain, tiredness, mood, sleep, as the person rated it.No registered study measures this.
■A number that stands in for healthEvidence recorded. Cholesterol, blood sugar, bone density. A stand-in, not the thing itself.6 registered measures of this kind.
■A step measured inside a personEvidence recorded. Something measured in human tissue or human cells.A human record sits on the stored organism ladder.
□AnimalsNo evidence recorded. Mice, rats, dogs. Useful for ideas, not proof about people.No animal record is stored.
■Cells in a dishEvidence recorded. Cells or chemistry on a bench, far from a whole body.Stored mechanism abstracts describe steps measured in cells or tissue.
□A guess from softwareNo evidence recorded. Nobody measured it. RNAWiki never publishes one as a finding.RNAWiki stores no prediction for this record. Software can suggest a link. RNAWiki does not publish one as a finding.
Higher on these steps means closer to something a person would feel. It does not mean better done.
What it would be like to take◇Read from sources, not yet reviewed
Felt, measured, or meaningful
Three different things that get called the same word. Registered studies measured all three, and mixing them is how a blood test becomes a health claim.
Felt
Things a person could notice without a test.
No registered study measured anything of this kind.
Measured
Things only a test, a scale or a device shows.
hba1c
fasting serum triglycerides
body weight at 6 months
serum triglycerides
insulin sensitivity
body composition
Meaningful
Things that change how a life goes, not only a number.
No registered study measured anything of this kind.
A registered study says what someone planned to measure. It does not say what they found. A number moving is not the same as a life going better. The two are shown apart here.
Felt, but not shown to help. A person notices a change. No study showed it leads anywhere good.
Measured, but not felt. A number moves. The person notices nothing. Both can be true.
Measured, but not shown to matter. A number moves in the good direction. Nobody showed that lives went better.
Matters, but takes years. The result that counts may take longer than anyone would keep watching.
Names that fit none of the three (4)
cortisol
total energy expenditure
weight
hepatic steatosis
These are harms, study-process measures, or names the rules did not recognise. They are shown rather than dropped.
What it would be like to take◇Read from sources, not yet reviewed
How long anything takes
Nine different lengths of time that get confused with each other. None of them is worked out from another.
Before anything is noticed.RNAWiki does not store this separately, and never works it out from another figure on this page.
Before a test result moves.RNAWiki does not store this separately, and never works it out from another figure on this page.
Before performance moves.RNAWiki does not store this separately, and never works it out from another figure on this page.
How long the result was watched.No finished study window is recorded for a study that tested this substance.
How long people took it.How long people actually took it is not stored. The study window is not the same thing.
How long people were followed.Follow-up length is not stored separately. It is never read off the study window, which would be a different thing.
How fast the body clears it. 3.8 to 4.7 hours hours
Read from the label, which states: “Following single subcutaneous doses of 0.01 to 0.3 mg/mL metreleptin in healthy subjects, the half-life was 3.8 to 4.7 hours.”
How long effects linger.RNAWiki does not store this separately, and never works it out from another figure on this page.
Beyond the studies. Nothing is recorded about the long term.
The longest finished study sets the edge of what anyone measured.
A study window is not how long people took it, and neither is how long they were followed. Where RNAWiki holds only one of the three, it shows one.
What is missing or unclear◇Read from sources, not yet reviewed
Were people like you studied?
RNAWiki cannot tell whether a study fits you. It can show who was in it, and where the result stops carrying.
Who was studied
People similar to this profile were included.
MYALEPT is a leptin analog indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy. ( 1 ) Limitations of Use The safety and effectiveness of MYALEPT for the treatment of complications of partial lipodystrophy have not been established.
Who is missing from the studies
Which groups were under-represented in the studies has not been recorded for this substance. Pregnancy, breastfeeding, children, older adults, and people with liver or kidney conditions are commonly excluded from trials; nothing here says whether they were.
What the label states about particular groups
On pediatric, the label states: “The MYALEPT study included a total of 35 pediatric patients (73%) with an age range from 1 to 17 years [see Clinical Studies (14.1) ].”
US prescribing information · d3b50bbd-140b-425c-b5ec-d4682aad62bc · read 2026-08-30
On older people, the label states: “Clinical trials of MYALEPT did not include sufficient numbers of subjects aged 65 and over (n=1) to determine whether they respond differently from younger subjects.”
US prescribing information · d3b50bbd-140b-425c-b5ec-d4682aad62bc · read 2026-08-30
On people who are pregnant, the label states: “Exposure Registry There is a program that monitors outcomes in women exposed to MYALEPT during pregnancy.”
US prescribing information · d3b50bbd-140b-425c-b5ec-d4682aad62bc · read 2026-08-30
On people who are breastfeeding, the label states: “There are no available data on the presence of metreleptin in human milk; however, endogenous leptin is present in human milk.”
US prescribing information · d3b50bbd-140b-425c-b5ec-d4682aad62bc · read 2026-08-30
Where the result stopped carrying
This is a scope explorer, not a diagnosis engine.
It shows who was studied so you can see whether people similar to you were included.
RNAWiki cannot tell whether a study fits you. It can show who was in it.
What it would be like to take◇Read from sources, not yet reviewed
Why it might seem to do nothing
A real effect and a noticed effect are different things. These are the recorded reasons the two come apart.
It was studied for a different goal
The studies measured something else entirely.
On this record: Some registered studies measured things that match no goal on this page.
It was studied in different people
The people in the studies were not much like the reader.
On this record: Which groups were under-represented in the studies has not been recorded for this substance. Pregnancy, breastfeeding, children, older adults, and people with liver or kidney conditions are commonly excluded from trials; nothing here says whether they were.
The evidence may simply be wrong
Small early studies often do not hold up.
On this record: RNAWiki has not yet published a reviewed conclusion for this use.
Other reasons RNAWiki checked and found nothing for (10)
A different form was studied
The studied form is not the form on the shelf. Nothing in this record points to this reason.
There was nothing to correct
Where a level is already normal, topping it up may change nothing. Nothing in this record points to this reason.
Something else had to happen too
In the studies it was paired with training, a diet or another treatment. Nothing in this record points to this reason.
The change is too small to feel
A real change can still sit below what a person notices. Nothing in this record points to this reason.
Day-to-day swing hides it
Sleep, food, stress and time of day move most numbers more than this would. Nothing in this record points to this reason.
Not enough time yet
The studies ran longer than the person has waited. Nothing in this record points to this reason.
It was not taken as studied
Missed days change the result, and studies count them. Nothing in this record points to this reason.
Something else changed at the same time
Two changes at once cannot be told apart afterwards. Nothing in this record points to this reason.
The product may not be what it says
Contents of a sold product are not always what the label states. Nothing in this record points to this reason.
No recorded reason
RNAWiki has nothing stored that would explain it. Nothing in this record points to this reason.
None of these is a reason to take more. Taking more is not a step this page ever suggests.
What it would be like to take◇Read from sources, not yet reviewed
What taking it involves
The recorded facts about getting hold of it and using it. Nothing here is a suggestion about what you should do.
How it is supplied
Prescription only
❝ Quoted from a source
Where this came from
Copied from a source RNAWiki stored, with the source named.
Read from the recorded approval status.
No source is stored against this line.
Form and route
Injection
✎ Source-linked record
Where this came from
A person wrote this into the record with the study named beside it. No reviewer has signed it off.
The form and route recorded on this substance.
No source is stored against this line.
Who oversees it
Professional supervision is normally required. A regulator classifies this substance in a way that restricts how it is supplied.
❝ Quoted from a source
Where this came from
Copied from a source RNAWiki stored, with the source named.
Suppression classes recorded: S3, S5, S6.
No source is stored against this line.
What is in the pack
Sold as injectable, injection, powder, lyophilized, for solution, given by the injection, subcutaneous route.
✎ Source-linked record
Where this came from
A person wrote this into the record with the study named beside it. No reviewer has signed it off.
Recorded notes on how this is sold and what that means for what is in the pack.
No source is stored against this line.
Where it is registered
Regulatory records are listed in the technical disclosure at the foot of this page.
# Counted from records
Where this came from
A count of rows RNAWiki holds. It describes our records, not your body.
Register entries are stored per jurisdiction and shown with their dates.
No source is stored against this line.
Why people stop
Not recorded.
∅ Nothing recorded
Where this came from
RNAWiki holds nothing here and says so rather than guessing.
No record of why people stopped taking it is stored for this substance.
No source is stored against this line.
What it would be like to take∅Nothing found in the sources checked
What can go wrong
Sorted by where each line came from. A regulator's label and a report somebody sent in are not the same kind of fact.
∅Nothing found in the sources checked
No harm is recorded against this substance in the sources RNAWiki checked. Finding nothing is not the same as showing there is nothing.
The sources listed were searched and held nothing. That is not the same as nothing existing.
Reports sent to a regulator
These are reports people sent to a regulator. They do not show the medicine caused the reaction.
Nobody counted how many people took the medicine and reported nothing.
The same event can be reported more than once, and many reports are incomplete.
News coverage, lawsuits and new warnings change how often people report.
A count is not a rate and not a risk.
Metreleptin appears in spontaneous reports to regulators. Across the 10 most-reported reaction terms, 244 reaction mentions were counted. One report can name several reactions.
open-targets-adr · recorded 2026-06-24 · a recorded source, not a stored snapshot
Over a long time. No completed tested study window is recorded, so long-term effects are not established by the registry record.
Groups the studies covered thinly. Which groups were under-represented in the studies has not been recorded for this substance. Pregnancy, breastfeeding, children, older adults, and people with liver or kidney conditions are commonly excluded from trials; nothing here says whether they were.
What is missing or unclear◇Read from sources, not yet reviewed
Does the exact form matter?
Evidence belongs to the exact thing that was tested. A different salt, a different mixture or a different preparation is a different question.
The form that was studied
Injection
✎ Source-linked record
Where this came from
A person wrote this into the record with the study named beside it. No reviewer has signed it off.
The form used in the studies cited on this page.
No source is stored against this line.
What is sold
Not recorded.
∅ Nothing recorded
Where this came from
RNAWiki holds nothing here and says so rather than guessing.
Nothing further is recorded about which forms are sold.
No source is stored against this line.
What is recorded as being sold
2 products list this as an active ingredient in the United States drug directory. 2 of them contain it and nothing else.
FDA National Drug Code directory · 10122-210 · read 2026-08-29
They are sold as injection, powder, lyophilized, for solution and liquid, taken subcutaneous.
FDA National Drug Code directory · 10122-210 · read 2026-08-29
The regulator's established pharmacologic class for it is analogs/derivatives [chemical/ingredient], leptin analog [epc] and leptin [chemical/ingredient].
FDA National Drug Code directory · 10122-210 · read 2026-08-29
1 published label names it as an active ingredient. 1 of them describe this substance alone, which is where its own label text on this page comes from.
US prescribing information · d3b50bbd-140b-425c-b5ec-d4682aad62bc · read 2026-08-29
Those labels are classed as human prescription drug.
US prescribing information · d3b50bbd-140b-425c-b5ec-d4682aad62bc · read 2026-08-29
Myalept is subcutaneous at 3 DOSAGE FORMS AND STRENGTHS For Injection: 11.3 mg of metreleptin supplied in a vial as a sterile, white, solid, lyophilized cake (delivers 5 mg per mL of metreleptin when reconstituted with 2.2 mL of BWFI or WFI)., recorded as fda label in effect 2024-03-25 in the United States.
US prescribing information · d3b50bbd-140b-425c-b5ec-d4682aad62bc · read 2026-08-30
Evidence carries across two names for one substance. It does not carry across a salt, a mirror form or a mixture.
What it would be like to take◇Read from sources, not yet reviewed
What you could measure
This one is decided with a clinician, so this section holds questions to ask rather than a plan to run.
Self-experiment planning is not offered for a prescription or clinician-supervised medicine. The questions below are for a clinician or pharmacist.
Questions worth asking
Which of the trials of Metreleptin studied people like me?
What was measured, and for how long?
Was the result a laboratory value or a health outcome?
What would we watch for, and when would we stop?
Tracking can show whether something changed for you. It cannot show what caused it.
RNAWiki records evidence. It does not say whether this substance is right for you.
What is missing or unclear◇Read from sources, not yet reviewed
What nobody knows yet
Open questions, each with why it is open and what would close it.
Missing populations
Which groups were under-represented in the studies has not been recorded.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
Missing long-term data
No completed tested study window is recorded.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
No reviewed conclusion
RNAWiki has not yet published a reviewed conclusion for this use.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
Missing interaction studies
No interaction was found in the registers checked. Not finding one is not the same as showing there is none.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
Formulation uncertainty
Several salts, forms or products of Metreleptin are recorded. Results from one form may not transfer to another.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
Mechanism not reviewed
No reviewed mechanism story exists for this substance.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
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FDA substance identifier (UNII)
TL60C27RLH
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1491629
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1 approved application covers products containing this substance. The earliest was BLA125390, approved 20140224 to CHIESI FARMACEUTICI SPA.
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5 questions this page could not answer
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Recorded evidence blocks (13)
Q2
What did Metreleptin's largest trial (636 people) and its longest (26 years) measure?
636 people in Metreleptin's largest registered study, 26 years in its longest registered window, measuring Percentage of Glycosylated Hemoglobin at Baseline, 6 Months, and 12 Months on Treatment With Metreleptin. ClinicalTrials.gov · 2026-09-01
20 phase2, 5 phase3, 4 phase1, 3 na or unstated, 2 na, 2 phase4; NCT00085982; 2030-01-01; no ageing endpoint recorded. Last human test completed 2024, NCT04026178.
Interpretation These counts include studies where Metreleptin was a comparison treatment, a background treatment or an exposure in an observational study, and the longest window may be a planned end date. Trial roles have not yet been classified for this record.
Show the evidence
phase2
20
phase3
5
phase1
4
na or unstated
3
na
2
phase4
2
1 more recorded row
Last recorded human testNCT04026178
2024-10-31
recorded 2026-09-01 · last checked 2026-09-04
Q3
Metreleptin was tested only in human — what did it show?
1 recorded entry; human; also "placebo-metreleptin 1 mL"
Show the evidence
humanNCT00392925
placebo-metreleptin 1 mL
recorded 2026-09-01 · last checked 2026-09-04
Q6
Metreleptin's half-life is 3.8 to 4.7 hours — which schedules were studied?
3.8 to 4.7 hours, the half-life Metreleptin's label states. openfda-label · d3b50bbd-140b-425c-b5ec-d4682aad62bc · 2026-08-30
Show the evidence
half life
3.8 to 4.7 hours hours; Following single subcutaneous doses of 0.01 to 0.3 mg/mL metreleptin in healthy subjects, the half-life was 3.8 to 4.7 hours.
tmax
In a supportive trial in lipodystrophy patients, the median T max of metreleptin was 4 hours (range: 2 to 8 hours; N=5) following single-dose administration of metreleptin.
metabolism
Metabolism and Elimination No formal metabolism studies have been conducted with metreleptin.
recorded 2026-08-30 · last checked 2026-09-04
Q7
Which of body composition, body weight at 6 months and cortisol did Metreleptin's trials measure?
body composition, body weight at 6 months and cortisol lead 10 outcome terms across Metreleptin's trials. ClinicalTrials.gov · 2026-09-01
fasting serum triglycerides, weight, body weight at 6 months, serum triglycerides, hepatic steatosis and insulin sensitivity follow.
Show the evidence
hba1c
1
cortisol
1
total energy expenditure
1
fasting serum triglycerides
1
weight
1
body weight at 6 months
1
4 more recorded rows
serum triglycerides
1
hepatic steatosis
1
insulin sensitivity
1
body composition
1
recorded 2026-09-01 · last checked 2026-09-04
Q8
Which of Metreleptin's 9 ongoing trials reports first?
Change in HbA1C; Serum hemoglobin A1C; latest 2031-10-31
Show the evidence
Trial
NCT00085982
"Effect of Metreleptin Therapy in the Treatment of Severe Insulin Resistance"; n 11; "Change in HbA1C"; 2030-01-01
NCT02262806
"Compassionate Use of Metreleptin in Previously Treated People With Partial Lipodystrophy"; n 29; "Serum hemoglobin A1C"; 2027-07-31
NCT02262832
"Compassionate Use of Metreleptin in Previously Treated People With Generalized Lipodystrophy"; n 24; "Serum triglycerides"; 2027-07-31
NCT02325674
"MEASuRE: Metreleptin Effectiveness And Safety Registry"; n 100; "The severity and incidence of the following safety events in patients prescribed Metreleptin in routine clinical practice"; 2031-10-31
NCT05164341
"Study to Evaluate the Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects With PL"; n 69; "Change from baseline to month 6 in HbA1c in subjects with partial lipodystrophy (PL)"; 2026-10-31
NCT05351164
"Evaluation of Safety and Efficiency of Metreleptin Treatment for Patients With Multiple Symmetric Lipomatosis (MSL)"; n 4; "Change in Truncal Adiposity"; 2028-10
3 further recorded trials
NCT06305182
"Metreleptin in Anorexia Nervosa"; n 50; "Clinician-rated depression on the 17 point Hamilton Depression Scale (HAMD-17) in the metreleptin-assisted therapy group compared to placebo-therapy group between Baseline, Post Treatment and 5 weeks Follow Up"; 2026-12-31
NCT06484868
"Open-label Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy"; n 12; "Number of patients with decrease of at least 0.5% in glycated haemoglobin (HbA1c) at Month 12 compared to Baseline or HbA1c <6.5 % at Month 12, in patients with baseline HbA1c ≥6.5%."; 2028-02
NCT06679270
"Open-label Extension Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy"; n 24; "Evaluate the Incidence and Frequency of Treatment-Emergent Adverse Events (Safety and Tolerability)"; 2028-12
recorded 2026-09-01 · last checked 2026-09-04
Q9
Which one trial of Metreleptin posted no result?
Posted no result
1 of 1 completed trials
Registrations
NCT04063488
Completion dates
oldest 2021-08-13
Show the evidence
TrialNCT04063488
2021-08-13
Q10
At the median, Metreleptin's trials enrolled 21 people — anything larger?
Median enrolment
21
Largest enrolment
636
Registered trials counted
32
Q11
What do 244 spontaneous reports say about Metreleptin — and not say?
These are reports people sent to a regulator. They do not show the medicine caused the reaction. Nobody counted how many people took the medicine and reported nothing. The same event can be reported more than once, and many reports are incomplete. News coverage, lawsuits and new warnings change how often people report. A count is not a rate and not a risk.
Metreleptin appears in spontaneous reports to regulators. Across the 10 most-reported reaction terms, 244 reaction mentions were counted: blood glucose increased 50; weight decreased 39; injection site pain 24; product dose omission issue 24. open-targets-adr · CHEMBL2107857 · 2026-06-24
Show the evidence
blood glucose increased
50
weight decreased
39
injection site pain
24
product dose omission issue
24
weight increased
20
blood triglycerides increased
19
4 more recorded rows
blood glucose decreased
18
hypoglycaemia
17
pancreatitis
17
hypertriglyceridaemia
16
recorded 2026-06-24 · last checked 2026-09-04
Q12
Metreleptin and CYP450 and cytochrome P450: shared by which compounds?
CYP450 and cytochrome P450 appear in Metreleptin's recorded interaction sentences, 3 in all. openfda-label+europepmc · 2024-03-25
Interpretation drug_interactions
Show the evidence
cytochrome P450drug_interactions
Leptin is a cytokine and may have the potential to alter the formation of cytochrome P450 (CYP450) enzymes.
CYP450
drug_interactions
This should be taken into account when prescribing concomitant drugs metabolized by CYP450 (e.g., oral contraceptives and drugs with a narrow therapeutic index).
drug_interactions
The effect of metreleptin on CYP450 enzymes may be clinically relevant for CYP450 substrates with narrow therapeutic index, where the dose is individually adjusted.
recorded 2024-03-25 · last checked 2026-09-04
Q13
Was Metreleptin studied with fasting and exercise?
fasting and exercise are named in Metreleptin's label sentences: "We conducted a randomized, placebo-controlled, double-blinded, crossover study from 2002 to 2004 in seven HIV+ men with HAART-induced lipoatrophy, serum leptin level less than 3 ng/ml, and fasting triglyceride level greater than 300 mg/dl, who were administered placebo for 2 months before or after…" openfda-label+europepmc · 2024-03-25
2 recorded statements; fasting, exercise
Show the evidence
fasting
We conducted a randomized, placebo-controlled, double-blinded, crossover study from 2002 to 2004 in seven HIV+ men with HAART-induced lipoatrophy, serum leptin level less than 3 ng/ml, and fasting triglyceride level greater than 300 mg/dl, who were administered placebo for 2 months before or after administration of r-metHuLeptin at physiological doses for an additional 2 months.
exercise
Study 1: 15 lean subjects received placebo or physiologic replacement-dose recombinant human leptin (metreleptin) following short term complete caloric deprivation to induce severe hypoleptinemia; Study 2: 7 women with relative leptin deficiency due to strenuous exercise or low weight received 3 months of metreleptin; Study 3: 17 women with relative leptin deficiency were randomized to receive…
recorded 2024-03-25 · last checked 2026-09-04
Q14
What is recorded about Metreleptin and mTOR?
"Acute in vivo, ex vivo, and/or in vitro metreleptin administration similarly activated STAT3, AMPK, ERK1/2, Akt, mTOR, NF-κB, and/or IKKα/β without any differences between male versus female and obese versus lean subjects." — where Metreleptin and mTOR appear together. Europe PMC · pathway abstract search · 2014-09-23
mTOR, AMPK; PMID 25249580, 23382191
Show the evidence
mTORPMID 25249580
"Acute in vivo, ex vivo, and/or in vitro metreleptin administration similarly activated STAT3, AMPK, ERK1/2, Akt, mTOR, NF-κB, and/or IKKα/β without any differences between male versus female and obese versus lean subjects."
AMPKPMID 25249580
"Acute in vivo, ex vivo, and/or in vitro metreleptin administration similarly activated STAT3, AMPK, ERK1/2, Akt, mTOR, NF-κB, and/or IKKα/β without any differences between male versus female and obese versus lean subjects."
mTORPMID 23382191
"We also observed that signaling pathways involved in cell growth/survival/proliferation, such as the STAT3, AMPK, mTOR, ERK1/2, and Akt pathways, were activated directly by acute in vivo metreleptin administration in peripheral blood mononuclear cells and CD4(+) T-cells both from subjects with chronic hypoleptinemia and from normoleptinemic, lean female subjects."
AMPKPMID 23382191
"We also observed that signaling pathways involved in cell growth/survival/proliferation, such as the STAT3, AMPK, mTOR, ERK1/2, and Akt pathways, were activated directly by acute in vivo metreleptin administration in peripheral blood mononuclear cells and CD4(+) T-cells both from subjects with chronic hypoleptinemia and from normoleptinemic, lean female subjects."
recorded 2014-09-23 · last checked 2026-09-04
Where it is registeredIdentifiers, relations and other names
ChEMBL ATC CC BY-SA · ClinicalTrials.gov — US Government work · Europe PMC — metadata only, under the recorded legal gate · Open Targets 26.06 — CC0 · derived from this page's own recorded fields; no external licence · mixed register set; per-register licences in docs/specs/corpus-20k-sources.md · openFDA / DailyMed — US Government work
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