Vosoritide dosage and evidence
Written by Aaron CuhaReviewed Sep 2026
The short answer
Approved for achondroplasia, a genetic cause of short height, on a final-stage trial. Children grew about 1.6 cm more a year. Nobody knows yet whether they end up taller as adults.
Evidence grade for Vosoritide: Limited. The rule: people have taken it in a published study, but fewer than 100 of them. The sum on this page: 1 completed trial, none stating how many people. A grade counts how much has been done to people, not whether it works.
- People given it
- 0
- Human studies
- 1
- Found nothing
- 0
Dose
Approved medicine. No human dose established for unlabelled uses
Labeled amounts sit on the prescribing information. This page records what trials measured, not a personal dose.
FDA-approved as Voxzogo to help children with achondroplasia, a form of dwarfism, grow taller while their growth plates are still open. The approval rests on how fast they grew, not on their final adult height.
4 effects reported, 3 unwanted, 1 wanted, by people using it, not a trial
In plain English
In plain English, from Aaron
This is how I would explain it to a friend. The evidence, with the numbers, is further down the page.
Vosoritide is a daily shot for children with achondroplasia, a genetic cause of short height. It pushes back against the faulty gene's effect on the growth plates. It is an approved prescription drug.
What people take it for
- Helping children with achondroplasia grow more.
- Nobody takes it for ageing. It is here because it shows what a well-aimed peptide looks like.
What studies in people showed
In 121 children aged five and up, a daily shot for a year made them grow about 1.57 cm more than children on a dummy shot. That is how fast they grew, not how tall they ended up. Nobody knows yet if they end up taller as adults. It needs a shot every day for years.
What people report
Reports, not study results
The good
- Children grew about 1.57 cm more per year in the main trial.
The bad
- Reactions where the needle goes in.
- Short drops in blood pressure.
- A daily shot for years is hard on a child and a family.
Where these come from: From the main trial and drug notes on this page. We have no user reports.
My bottom line
This is what a well-aimed peptide looks like. It still only proved faster growth, not a taller adult. It is a call for families and their doctors.
An opinion, not a finding. I am a coach, not a doctor.
What it has been measured to do
Nothing indexed yet
The 1 citation on this page do not report a result for any of the 36 goals indexed here. Most of what sits here is something else: a listing of a planned study, a study of how the body handles the drug, a safety report or a government record. None of those tests whether it works.
Show full evidence
What is Vosoritide?
Approved for achondroplasia, a genetic cause of short height, on a final-stage trial. Children grew about 1.6 cm more a year. Nobody knows yet whether they end up taller as adults.
Vosoritide is the clearest example on this site of a peptide designed from exact knowledge of one gene fault. Achondroplasia is caused by a change in the FGFR3 gene that leaves it switched on too hard. That over-brakes the growth plates, the parts of bone where growing happens. A natural peptide called CNP signals through a different receptor. It pushes back on that same pathway further down the line. So giving a longer-lasting version of CNP counters the gene fault right where it acts.
In the final-stage trial, 121 children had a daily shot or a dummy shot for a year. Treated children grew about 1.57 cm more a year, and it was approved on that basis.
The honest limit matters, and it is why this page reads the way it does. The trial measured how fast children grew over a year, not their final adult height. Those are different things. A child who grows faster for a year has not necessarily ended up taller as an adult.
How does Vosoritide work?
Vosoritide is a 39 amino acid copy of C-type natriuretic peptide (CNP). It is changed so an enzyme that normally destroys CNP within minutes (neutral endopeptidase) cannot break it down as easily. It binds a receptor called NPR-B. That raises a messenger inside the cell (cyclic GMP) and switches on an enzyme (protein kinase G), which blocks a growth-braking chain of signals (RAF, MEK and ERK) at an early link. In achondroplasia, the faulty FGFR3 gene drives that same chain too hard, and growth plate cells stop multiplying. So vosoritide brakes the pathway the gene fault speeds up. It pushes back further down the line. It does not fix the fault itself.
What have studies in people found about Vosoritide?
A final-stage trial, with a dummy shot for comparison and nobody knowing who got what, showed children grew faster. There is also follow-on data. There is no result for final adult height.
- 121 children aged five and up with achondroplasia and still-growing bones, in seven countries, got a daily shot or a dummy shot for a year. Nobody knew who got which.
- Treated children grew about 1.57 cm more per year than the dummy shot group. That is what the approval rests on.
- It needs a daily shot under the skin, in children, for years. That is a real burden to keep up.
- Reactions where the needle goes in, and short drops in blood pressure, are the expected side effects. The blood pressure drop comes straight from how the drug works.
- Final adult height was not what the trial measured, and it is still unknown.
What this does not tell you: Growing faster over one year stands in for what families actually care about, which is adult height and how well a person gets around. Faster growth for a while could mean a child ends up taller. It could also mean they just get there sooner. Telling those apart means following children until their bones stop growing. It is also worth saying that many people with achondroplasia do not see it as a disease. The medical problems worth treating, such as a narrow opening at the base of the skull and sleep apnoea, are not the same thing as height.
The state of the research
This page is good to read next to the growth hormone boosters elsewhere on this site, because it shows what a precisely aimed peptide looks like. The gene fault is known. The pathway is known. The receptor that pushes back is known. The drug was built to hit it. That is a different kind of evidence from a peptide sold because it raises a hormone that falls with age.
The stand-in marker problem still applies here, as strongly as anywhere. Growth speed is not adult height. This site's position is that a drug approved on a stand-in marker is exactly that, a drug approved on a stand-in marker.
The evidence, in charts
Fig. 1a · how many people
1study in people cited, but none says how many people took part
number of people not stated
We do not draw a total, because none of the studies in people on this page gives one. Read the sources below for what each study measured.
We read the number of people from the source lines on this page. Where a line gives no number, we count nothing for it. We do not guess.
Fig. 1b · evidence mix
All 1 source here is a study in people.
- Study in people · done in people1100%
Counted from the list of sources on this page. It counts only what we cite, so there may be more studies out there. A review counts once, however many studies it sums up.
Fig. 2 · evidence over time
Every source here was published in 2020.
Too few different years on this page to draw a timeline. The newest source on file is from 2020, more than five years ago. Research on it may have gone quiet.
Fig. 3 · legal status at a glance
US
Approved
UK
Approved
AU
Approved
CA
Approved
Approved in all four countries shown. This is what the law says, not a verdict on the science. See Legal status below for the full text and any notes.
Fig. 5 · what the molecule is
- Kind of compound
- Peptide
- Molecule weight
- Roughly 4.1 kDa. A 39-amino-acid peptide.
- Half-life
- About 25 minutesUltra-short, minutes
- Chain length
- None on file
Half-life as stated on file: Roughly 20 to 30 minutes, very short, which is why it requires daily subcutaneous injection despite being engineered for stability against neutral endopeptidase.
No amino acid sequence is on file for Vosoritide, so none is drawn rather than guessed from a name.
The studies we cite
- Study in people2020Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial
Vosoritide made children with achondroplasia grow faster, and its approval rests on this trial. 121 children aged five and older were split by chance into two groups across seven countries. One group had a daily shot under the skin for a year. The others had a dummy shot, and nobody involved knew which. Treated children gained about 1.57 cm more height per year. The trial measured growth speed over one year, not final adult height. BioMarin, which sells the drug, paid for it.
The pivotal randomised double-blind placebo-controlled phase 3 in children with achondroplasia, showing increased annualised growth velocity on daily subcutaneous vosoritide. This is the trial the approval rests on, and its endpoint is growth velocity rather than final adult height.
Where to buy Vosoritide
Vosoritide is an approved medicine. The approved product comes from a pharmacy, with a prescription. The vials below are sold “for research” and are not the approved drug.
No seller we track lists Vosoritide yet. We read sellers’ public product lists every week, and this section fills in as soon as one does. Every seller, graded
Common questions
Has Vosoritide been tested in people?
Yes. 0 people given it across 1 completed trial cited here, 0 of which found nothing. Approved for achondroplasia, a genetic cause of short height, on a final-stage trial. Children grew about 1.6 cm more a year. Nobody knows yet whether they end up taller as adults.
What is the Vosoritide dosage?
Approved medicine. No human dose established for unlabelled uses. Labeled amounts sit on the prescribing information. This page records what trials measured, not a personal dose. FDA-approved as Voxzogo to help children with achondroplasia, a form of dwarfism, grow taller while their growth plates are still open. The approval rests on how fast they grew, not on their final adult height.
Is Vosoritide FDA-approved?
FDA-approved as Voxzogo to help children with achondroplasia, a form of dwarfism, grow taller while their growth plates are still open. The approval rests on how fast they grew, not on their final adult height.
Does vosoritide make children with achondroplasia taller as adults?
Unknown. The final-stage trial measured how fast children grew over a year, and they grew faster. That is what the approval rests on. Final adult height means following children until their bones stop growing, and it was not known at approval.
How does it work when the problem is a genetic mutation?
It does not fix the gene fault. The FGFR3 fault over-drives a chain of signals that holds back growth. CNP signalling blocks that same chain further down. So the drug pushes back against what the fault does, instead of fixing its cause.
Why does it need a daily injection?
Because it only lasts about 20 to 30 minutes in the blood, even after being built to resist the enzyme that destroys natural CNP. That is a big practical burden when the patient is a child and treatment runs for years.