Avalglucosidase Alfa-Ngpt
- Biologic
- Given by a clinician
- Identity checked
- Sources last checked 2026-09-04
This page shows what was measured, who it was measured in, and what that does not settle.
RNAWiki holds very little about this substance. The page shows what there is.
What Avalglucosidase Alfa-Ngpt does in the body
NEXVIAZYME is indicated for the treatment of patients 1 year of age and older with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency).
Pompe disease (also known as glycogen storage disease type II, acid maltase deficiency, and glycogenosis type II) is an inherited disorder of glycogen metabolism caused by a deficiency of the lysosomal enzyme acid α-glucosidase (GAA) that degrades glycogen to glucose in the lysosome. GAA deficiency results in intralysosomal accumulation of glycogen in various tissues. Avalglucosidase alfa-ngpt provides an exogenous source of GAA. The M6P on avalglucosidase alfa-ngpt mediates binding to M6P receptors on the cell surface with high affinity.
Why people take it. NEXVIAZYME is indicated for the treatment of patients 1 year of age and older with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency).
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 · EO144CP0X9 · 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 136 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.
Formulation
A formulation is the exact made-up form a substance comes in.
A picture of it, and where the picture fails
It is like the difference between a whole bean and instant coffee.
Where that stops being true. Coffee tastes different. A formulation can change how much reaches the blood.
What people get wrong. Two products with the same name are assumed to behave the same. They often do not.
The specific composition and physical form of a product, including salt, excipients and release profile.
What is missing or unclearRead 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.
- Patients 1 year of age and older with late-onset Pompe disease
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: “Use of NEXVIAZYME for this indication is supported by evidence from two clinical studies which included adults with LOPD, and 1 pediatric patient with LOPD (16 years of age) and from safety experience in 19 pediatric patients with infantile-onset Pompe disease (IOPD) (1 to 12 years of age) treated with NEXVIAZYME [see Clinical Studies (14.1) ] .”
US prescribing information · 80eb3c9c-01d3-44fe-90ef-81df101b954d · read 2026-08-30
On older people, the label states: “Clinical studies with NEXVIAZYME included 13 patients 65 to 74 years of age and 4 patients 75 years of age and older.”
US prescribing information · 80eb3c9c-01d3-44fe-90ef-81df101b954d · read 2026-08-30
On people who are pregnant, the label states: “The effects of NEXVIAZYME were evaluated based on comparison with a control group treated with DPH alone.”
US prescribing information · 80eb3c9c-01d3-44fe-90ef-81df101b954d · read 2026-08-30
On people who are breastfeeding, the label states: “Risk Summary There are no data on the presence of avalglucosidase alfa-ngpt in either human or animal milk, the effects on the breastfed infant, or the effects on milk production.”
US prescribing information · 80eb3c9c-01d3-44fe-90ef-81df101b954d · 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 takeRead 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.
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 studied form is not the form on the shelf.
On this record: This record is linked to 1 related forms. Evidence does not carry across all of them.
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)
- It was studied for a different goal
- The studies measured something else entirely. 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 takeRead 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
Given by a clinician
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
Not recorded.
Nothing recorded
Where this came from
RNAWiki holds nothing here and says so rather than guessing.
No form or route is recorded.
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: S7.
No source is stored against this line.
What is in the pack
Not recorded.
Nothing recorded
Where this came from
RNAWiki holds nothing here and says so rather than guessing.
Nothing is recorded about product quality.
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 takeNothing 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.
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 unclearRead 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
Not recorded.
Nothing recorded
Where this came from
RNAWiki holds nothing here and says so rather than guessing.
The exact form studied is not recorded.
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
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 · 80eb3c9c-01d3-44fe-90ef-81df101b954d · read 2026-08-29
Those labels are classed as human prescription drug.
US prescribing information · 80eb3c9c-01d3-44fe-90ef-81df101b954d · read 2026-08-29
Nexviazyme is intravenous at 3 DOSAGE FORMS AND STRENGTHS For injection: 100 mg of avalglucosidase alfa-ngpt as a white to pale-yellow lyophilized powder in a single-dose vial for reconstitution., recorded as fda label in effect 2025-06-05 in the United States.
US prescribing information · 80eb3c9c-01d3-44fe-90ef-81df101b954d · 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.
Names and forms linked to this record
Biosimilar of
Evidence on this page does not automatically apply to this one.
What it would be like to takeRead 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 Avalglucosidase Alfa-Ngpt 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 unclearRead from sources, not yet reviewed
What nobody knows yet
Open questions, each with why it is open and what would close it.
How much did people take in the studies?
The sources RNAWiki checked hold nothing for this field.
Why it matters. A result belongs to an amount. Without the amount the result floats free.
What would answer it
A stored source that records 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 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 Avalglucosidase Alfa-Ngpt 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.
What happened in peopleRead from sources, not yet reviewed
Check any of this yourself
Every line above traced back to the study it came from. This is the technical layer, and the vocabulary changes here.
Where else this substance is registered
- FDA substance identifier (UNII)
- EO144CP0X9
- RxNorm concept
- 2565818
Checks this page had to pass
Passed
Identity resolved
no open identity hold
Passed
No unresolved merge across substance families
no quarantine open
Passed
Every public sentence names a source
The opening statement carries the origin: Quoted from a stored source.
Not passed
Trial roles classified for highlighted evidence
No registered study is classified as testing this substance.
Passed
No internal keys in reader text
enforced by the copy-contract test over the rendered page
Passed
Safety mode resolved
Suppression classes recorded: S7.
Passed
Canonical metadata present
slug and display name present
What is missing or unclearRead from sources, not yet reviewed
How this medicine reached us
Kept near the foot of the page. A historical event moves up only when it changes something about this substance today.
What the approval register records
1 approved application covers products containing this substance. The earliest was BLA761194, approved 20210806 to GENZYME CORP.
Drugs@FDA application register · BLA761194 · read 2026-08-29
Marketing status on the register: prescription.
Drugs@FDA application register · BLA761194 · read 2026-08-29
What is missing or unclearRead from sources, not yet reviewed
What to learn next
Ordered by what would most change how you read this page, starting with what is easiest to misunderstand.
- Were people like me actually studied?
- How close is this evidence to something I would notice?
- What does nobody know about this yet?
- What is a formulation?
This order is fixed in code and does not count clicks or time on the page.
What is not here
10 questions this page could not answer
These sections were prepared and left out because there was nothing to put in them. They are listed rather than hidden, so the gaps in this record are visible.
- What was measured, goal by goal — found nothing in the sources checked.
- What happened in people — found nothing in the sources checked.
- How close this is to real life — found nothing in the sources checked.
- The path through the body — found nothing in the sources checked.
- Felt, measured, or meaningful — found nothing in the sources checked.
- How long anything takes — found nothing in the sources checked.
- What it may clash with — found nothing in the sources checked.
- Other ways to the same goal — found nothing in the sources checked.
- Claims that go past the evidence — found nothing in the sources checked.
- What changed on this page — found nothing in the sources checked.
The record as stored
The full record, for auditing
Everything above is built from what is below. This layer keeps the technical vocabulary, record identifiers and every stored row, so a reader who wants to check the page can.
Recorded evidence blocks (5)
On the Avalglucosidase Alfa-Ngpt label: indicated for what?
"NEXVIAZYME is indicated for the treatment of patients 1 year of age and older with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency). NEXVIAZYME is a hydrolytic lysosomal glycogen-specific enzyme indicated for the treatment of patients 1 year of age and older with late-onset Pompe disease…": indications and usage on Avalglucosidase Alfa-Ngpt's label. DailyMed label · 80eb3c9c-01d3-44fe-90ef-81df101b954d · 2025-06-05
6 registered trials of Avalglucosidase Alfa-Ngpt — at which phases?
- Registered studies posting no result
- 4 of 6
6 registered studies of Avalglucosidase Alfa-Ngpt: 2 phase2, 1 na or unstated, 1 phase1, 1 phase3, 1 phase4. CLINICALTRIALS_SNAPSHOT · 2026-09-01
0 with a PubMed record
Show the evidence
- phase22
- na or unstated1
- phase11
- phase31
- phase41
- active not recruiting2
2 more recorded rows
- completed2
- recruiting2
recorded 2026-09-01 · last checked 2026-09-04
Avalglucosidase Alfa-Ngpt's half-life is 1.6 hours — which schedules were studied?
1.6 hours, the half-life Avalglucosidase Alfa-Ngpt's label states: "Elimination The mean total body clearance of avalglucosidase alfa-ngpt was 0.9 L/hour and the mean plasma elimination half-life of avalglucosidase alfa-ngpt was 1.6 hours in LOPD patients." DailyMed label · 80eb3c9c-01d3-44fe-90ef-81df101b954d · 2025-06-05
Show the evidence
- half life pharmacokinetics1.6 hours; Elimination The mean total body clearance of avalglucosidase alfa-ngpt was 0.9 L/hour and the mean plasma elimination half-life of avalglucosidase alfa-ngpt was 1.6 hours in LOPD patients.
- metabolism pharmacokineticsMetabolism The protein portion of avalglucosidase alfa-ngpt is expected to be metabolized into small peptides by catabolic pathways.
recorded 2025-06-05 · last checked 2026-09-04
Which one trial of Avalglucosidase Alfa-Ngpt posted no result?
- Posted no result
- 1 of 1 completed trials
- Registrations
- NCT01898364
- Completion dates
- oldest 2015-02-25
Show the evidence
- Trial NCT018983642015-02-25
At the median, Avalglucosidase Alfa-Ngpt's trials enrolled 20.5 people — anything larger?
- Median enrolment
- 20.5
- Largest enrolment
- 100
- Registered trials counted
- 6
Where it is registered
Identifiers, relations and other names
The exact record
- UNII
- EO144CP0X9
- CAS number
- 1802558-87-7
- RxCUI
- 2565818
Relations
- Biosimilar of Avalglucosidase alfa
- Trade name
- Nexviazyme
- Also called
- AVALGLUCOSIDASE ALFA, AVALGLUCOSIDASE ALFA [JAN], AVALGLUCOSIDASE ALFA [USAN], Avalglucosidase alfa [MI], Avalglucosidase alfa [WHO-DD], Avalglucosidase alfa ngpt [WHO-DD], NEO-RECOMBINANT HUMAN ACID ALPHA-GLUCOSIDASE, NEOGAA
- Development code
- GZ-402666, GZ402666
Sources (4)
Sources
- CLINICALTRIALS_SNAPSHOT K1:EO144CP0X9#ngpt ·
- drugsfda drugsfda ·
- DailyMed label 80eb3c9c-01d3-44fe-90ef-81df101b954d ·
- Drugs@FDA K1:EO144CP0X9#ngpt ·
ClinicalTrials.gov — US Government work · mixed register set; per-register licences in docs/specs/corpus-20k-sources.md · openFDA / DailyMed — US Government work
How these records are assembled · Which registers were checked
Index-quality checks
- identity passed: no open identity hold
- required summary fields resolved: 3 required field(s) not terminal: Best-supported result, Biggest unanswered question, Human evidence
- public claims reviewed: 0 reviewed claim(s); drafts are never rendered
- source coverage passed: 4 source rows
- no critical contamination: no quarantine open
- canonical metadata passed: slug and display name present
- no raw internal fields: enforced by the copy-contract test over the rendered page
This is a record of evidence. It is not medical advice, and it does not say this substance suits you. Nothing here says any substance on RNAWiki is appropriate for a child.