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Efalizumab

  • Withdrawn substance
  • Withdrawn
  • 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.

What Efalizumab does in the body

Formerly used as a weekly injection for severe plaque psoriasis.

White blood cells stick to the inside of blood vessels before they squeeze through the wall into tissue. That sticking uses a surface protein pair, and efalizumab is an antibody that covers one half of that pair. T cells then cannot grip the vessel wall properly, so far fewer of them reach the skin and the psoriasis plaques settle. The same grip is used by T cells that patrol the brain for a common dormant virus, and blocking it long enough allowed that virus to reactivate.

What happened in people

About one quarter of treated people had a major skin improvement, compared with one in twenty given a dummy treatment.

Reviewed first-read answer

Where this came from

A person wrote this and a reviewer approved it against this exact record. It carries no effect size.

A reviewer approved this against this record. The reviewed-claim record carrying the exact population and effect size does not exist yet.

No source is stored against this line.

The limit that matters most

Four fatal brain infections were reported, but the risk per long-term user could not be calculated.

Where it acts
Circulating T lymphocytes and the dermal vascular endothelium; the toxicity site is central nervous system white matter
Kind of result
The kind of result is not recorded
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 · XX2MN88N5D · read 2026-08-29

Where each sentence above came from

No recorded sentence describes the change this makes in a way that stands on its own. The page opens with what it is taken for instead, and the recorded explanation follows below.

Shown as the opening line on this page.

The limit a reviewer approved as the one that matters most here.

The four opening statements run to 118 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.

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.

Enzyme

An enzyme is a protein that speeds up one chemical change.

A picture of it, and where the picture fails

An enzyme is like a machine on a production line doing one cut.

Where that stops being true. A machine is switched on and off by a person. Enzymes are controlled by the cell.

What people get wrong. Enzymes are thought to be used up. They are not; they work again and again.

A catalytic protein that lowers the activation energy of a specific reaction.

Receptor

A receptor is a part of a cell that a signal fits into.

A picture of it, and where the picture fails

A receptor is like a lock waiting for one key.

Where that stops being true. A lock either opens or does not. A receptor can be half-triggered, or worn out.

What people get wrong. Fitting a receptor is read as causing a benefit. It causes a step, and nothing more.

A protein that binds a specific ligand and converts that binding into a cellular response.

Pathway

A pathway is a chain of steps inside a cell, each one setting off the next.

A picture of it, and where the picture fails

A pathway is like a row of dominoes.

Where that stops being true. Dominoes fall one way. Pathways loop back and can switch themselves off.

What people get wrong. Changing one step is read as changing the outcome. Other steps often absorb it.

An ordered series of molecular interactions producing a defined cellular change.

What happened in peopleRead 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 5 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.
GoalLife outcomeWhat a body can doHow a person feelsA test resultA step in the bodyHarmsHow longWho was studied
Blood sugarNothing 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 moved1 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.
Which registered measures put each goal on this table
Blood sugar
insulin independent full islet graft function

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
1 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 peopleRead 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.

Improvement of 75 per cent or more in the Psoriasis Area and Severity Index at week 12

The study showed what it set out to show

Who was studied
Phase 3 placebo-controlled trial of efalizumab in plaque psoriasis (Lebwohl et al.)
How many people
597
Study design
Phase 3
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
PASI-75 in 22 per cent at 1 mg/kg/week and 28 per cent at 2 mg/kg/week against 5 per cent on placebo, P < 0.001 for both
Repeated elsewhere
Replicated

What this does not prove. Meeting one endpoint in one population does not carry to other goals or other people.

The limits of this study, and where it came from

Main limit. The trial ran 12 weeks with 24 weeks of treatment in extension. Every PML case reported after approval occurred at three years or more of continuous exposure, a duration the registration programme could not have observed.

How far it carries. This study is recorded from the curated record, not from the registry match.

Form and route. Subcutaneous injection, once weekly

Interval reported. Not recorded in this summary

Written into the record, not signed off as a reviewed claim.

Confirmed PML cases by biologic agent approved for psoriasis, from approval to 30 January 2009

The study showed what it set out to show

Who was studied
FDA Adverse Event Reporting System review of PML with psoriasis biologics
How many people
12
Study design
Post-marketing pharmacovigilance case series
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Efalizumab 4 cases, all in psoriasis, all fatal, all at three or more years of treatment; adalimumab 1, etanercept 3, infliximab 4, none in psoriasis and all confounded or unconfirmed
Repeated elsewhere
Partially Replicated

What this does not prove. Meeting one endpoint in one population does not carry to other goals or other people.

The limits of this study, and where it came from

Main limit. Spontaneous reporting has no denominator and is subject to underreporting, so a per-patient incidence rate was never established.

How far it carries. This study is recorded from the curated record, not from the registry match.

Form and route. Subcutaneous injection, once weekly

Interval reported. Not recorded in this summary

Written into the record, not signed off as a reviewed claim.

What we know
RNAWiki holds 2 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 peopleRead 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.

  1. Living longer, or avoiding a major event Evidence recorded. Death, a heart attack, a stroke, a hospital stay.1 registered measure of this kind. No reviewed result.
  2. What a body can do day to day No evidence recorded. Walking, dressing, breathing, recovering.No registered study measures this.
  3. Measured performance No evidence recorded. How much was lifted, how far was run, how fast.No registered study measures this.
  4. Symptoms and quality of life No evidence recorded. Pain, tiredness, mood, sleep, as the person rated it.No registered study measures this.
  5. A number that stands in for health Evidence recorded. Cholesterol, blood sugar, bone density. A stand-in, not the thing itself.1 registered measure of this kind.
  6. A step measured inside a person Evidence recorded. Something measured in human tissue or human cells.A human record sits on the stored organism ladder.
  7. Animals Evidence recorded. Mice, rats, dogs. Useful for ideas, not proof about people.Stored records in Mouse, Rat. A result in animals says what to test next. It does not say what happens in people.
  8. Cells in a dish No evidence recorded. Cells or chemistry on a bench, far from a whole body.No cell or bench record is stored.
  9. A guess from software No 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 changes in the bodyRead from sources, not yet reviewed

The path through the body

From what a person takes to what changes. A solid line is a step measured in people. A dashed line is a step nobody has measured that way.

  1. Start

    Efalizumab

    What a person takes: Subcutaneous injection, once weekly.

    The measurement behind this step

    Weekly subcutaneous humanised IgG1 antibody with a conditioning first dose. Target-mediated clearance means exposure rises non-linearly once CD11a occupancy saturates, and surface CD11a downmodulation means immune function recovers more slowly than drug concentrations fall.

  2. Getting in

    A weekly injection under the skin

    Given once a week as a subcutaneous injection, often self-administered.

    Described, with no measurement named. No measurement is named for this step, so it is drawn as an unconfirmed link.

    The measurement behind this step

    Subcutaneous efalizumab, a conditioning dose followed by weekly maintenance dosing at 1 mg/kg. Clearance is target-mediated and saturable, so steady-state exposure rises disproportionately once CD11a is fully occupied.

  3. What it acts on

    Binds CD11a on circulating T cells

    In the bloodstream it coats a specific protein on the surface of T cells and pulls some of it off the surface entirely.

    Described, with no measurement named. No measurement is named for this step, so it is drawn as an unconfirmed link.

    The measurement behind this step

    Binds the alpha-L subunit of LFA-1 on circulating lymphocytes, producing both steric blockade and downmodulation of surface CD11a, which is why lymphocyte counts rise and functional recovery lags the drug's clearance.

  4. Reaching the cell

    T cells lose their grip on the vessel wall

    Without that protein working, T cells cannot hold on to the inside of blood vessels long enough to squeeze through into tissue.

    Described, with no measurement named. No measurement is named for this step, so it is drawn as an unconfirmed link.

    The measurement behind this step

    LFA-1 can no longer engage ICAM-1 on activated endothelium, so firm adhesion and transendothelial migration fail. The same blockade impairs the LFA-1/ICAM-1 immunological synapse required for T-cell activation by antigen-presenting cells.

  5. The change it makes

    Fewer T cells reach the plaque — and fewer reach the brain

    Far fewer T cells enter the skin, so the psoriasis inflammatory loop breaks. The same block applies to the T cells that keep a dormant brain virus in check.

    Described, with no measurement named. No measurement is named for this step, so it is drawn as an unconfirmed link.

    The measurement behind this step

    Reduced T-cell trafficking into dermis and epidermis interrupts the IL-23/Th17 keratinocyte loop. The same reduction in transendothelial migration applies at the blood-brain barrier, permitting reactivation of JC polyomavirus in oligodendrocytes.

  6. What that does for a person

    Plaques clear in a quarter of patients; four developed fatal PML

    About a quarter of patients had a major improvement in their skin. Four patients developed a fatal brain infection after three or more years of treatment.

    Measured in people. Written by a person from the study named beside the step. Not signed off as a reviewed claim.

    The measurement behind this step

    Measured: PASI-75 in 22 to 28 per cent at 12 weeks against 5 per cent on placebo. Measured: four confirmed PML cases in psoriasis patients in the FDA adverse event database, all fatal, all after three or more years of treatment.

No suggested links are held for this record, so nothing is hidden from this path.

What we know
The record describes 5 steps from taking it to an effect in a person.
How we know it
Each step names the study behind it in the technical detail beside it.
What this does not prove
A change inside the body is a reason to look. It is not a result in a person.
Why it matters
A reader can see exactly where the chain stops being measured in people.
What we still do not know
No reviewed claim binds any of these steps to a named population.

What it would be like to takeRead 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.

  • insulin independent full islet graft function

Meaningful

Things that change how a life goes, not only a number.

  • renal allograft survival

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 (3)
  • pasi psoriasis area and severity index
  • experiencing adverse events
  • psoriasis area and severity index

These are harms, study-process measures, or names the rules did not recognise. They are shown rather than dropped.

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.

  • Nobody. The product is withdrawn worldwide. Patients on it in 2009 were transitioned to other systemic agents, which by then included the TNF inhibitors and ustekinumab.

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.

Where the result stopped carrying

  • Marketing authorisation suspended in the European Union in February 2009 and voluntarily withdrawn in the United States the same year
  • The registration programme observed 12 to 24 weeks; the harm appeared at three years and later
  • Discontinuation itself carried rebound and severe flare risk, so the withdrawal required managed transition rather than a stop order
  • 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.

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 change is too small to feel

A real change can still sit below what a person notices.

On this record: Natalizumab, blocking the same functional step through a different integrin, returned under a monitoring programme; efalizumab did not

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 (9)
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.
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

Withdrawn

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

Subcutaneous injection, once weekly

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: S1, S8.

No source is stored against this line.

What is in the pack

Weekly subcutaneous humanised IgG1 antibody with a conditioning first dose. Target-mediated clearance means exposure rises non-linearly once CD11a occupancy saturates, and surface CD11a downmodulation means immune function recovers more slowly than drug concentrations fall.

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 takeRead from sources, not yet reviewed

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.

Worth asking a clinician aboutWritten into the record from the studies named on this page

Withdrawn worldwide in 2009. The decisive harm was progressive multifocal leukoencephalopathy: four confirmed cases in psoriasis patients in the FDA adverse event database, all fatal, all after three or more years of continuous treatment. Other recognised effects were first-dose flu-like reactions, thrombocytopenia, haemolytic anaemia, immune-mediated arthritis, and rebound or severe flare of psoriasis on discontinuation, including erythrodermic and pustular presentations.

Nobody counted how many people took this and were fine, so this cannot be turned into a rate.

Where this came from

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.

Efalizumab appears in spontaneous reports to regulators. Across the 10 most-reported reaction terms, 558 reaction mentions were counted. One report can name several reactions.

The recorded terms (10)
  • psoriasis — 264 reaction mentions
  • white blood cell count increased — 60 reaction mentions
  • myalgia — 47 reaction mentions
  • psoriatic arthropathy — 36 reaction mentions
  • influenza like illness — 34 reaction mentions
  • arthritis — 33 reaction mentions
  • skin exfoliation — 28 reaction mentions
  • lymphocyte count increased — 21 reaction mentions
  • lymphocytosis — 18 reaction mentions
  • dermatitis exfoliative — 17 reaction mentions
  • 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 unclearNothing found in the sources checked

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

Subcutaneous injection, once weekly

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

Target-mediated clearance means exposure rises non-linearly once CD11a occupancy saturates, and surface CD11a downmodulation means immune function recovers more slowly than drug concentrations fall.

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 which forms are sold and how they compare.

No source is stored against this line.

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 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, withdrawn medicine. The questions below are for a clinician or pharmacist.

Questions worth asking

  • Which of the trials of Efalizumab 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

Claims that go past the evidence

Things said about this substance that sound reasonable, and the exact step that is missing between the evidence and the claim.

Goes past the evidence

That a per-patient PML incidence rate is known — the analysis has a numerator of four and no denominator of long-duration exposures

Why it sounds right. It follows from something real on this page: a body step, an animal result or a related finding.

The missing step. Nobody measured this in people for this use, or the study that did measure it did not show it.

What would change this

A study in people, for this exact use, measuring this exact thing, that a reviewer signs off.

RNAWiki has not yet published a reviewed conclusion for this use.

Goes past the evidence

That a safe treatment duration exists below three years; the authors state explicitly that none has been defined

Why it sounds right. It follows from something real on this page: a body step, an animal result or a related finding.

The missing step. Nobody measured this in people for this use, or the study that did measure it did not show it.

What would change this

A study in people, for this exact use, measuring this exact thing, that a reviewer signs off.

RNAWiki has not yet published a reviewed conclusion for this use.

Goes past the evidence

That the PML cases reported with anti-TNF agents in the same review are comparable — those occurred in other conditions with confounding immunosuppression

Why it sounds right. It follows from something real on this page: a body step, an animal result or a related finding.

The missing step. Nobody measured this in people for this use, or the study that did measure it did not show it.

What would change this

A study in people, for this exact use, measuring this exact thing, that a reviewer signs off.

RNAWiki has not yet published a reviewed conclusion for this use.

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.

How fast does the body clear it?

The sources RNAWiki checked hold nothing for this field.

Why it matters. Without this, nothing on this page can say how long anything lasts.

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 Efalizumab 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.

Every line above can be traced to the study named beside it. Follow the link and read it.

PASI-75 in 22 to 28 per cent at 12 weeks against 5 per cent on placebo
In plain words
In the pivotal trial of 597 patients, about a quarter of those on efalizumab had their psoriasis score fall by three quarters at 12 weeks, against one in twenty on placebo.
What was measured
PASI-75 response rate at 12 weeks, efalizumab versus placebo
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Phase 3 multicentre randomised placebo-controlled double-blind trial in 597 subjects with moderate to severe plaque psoriasis, randomised to subcutaneous efalizumab 1 mg/kg/week, 2 mg/kg/week, or placebo for 12 weeks. At week 12, an improvement of 75 per cent or more in the Psoriasis Area and Severity Index occurred in 22 per cent at 1 mg/kg and 28 per cent at 2 mg/kg, against 5 per cent on placebo (P < 0.001 for both). Separation from placebo was evident by week 4 (P < 0.001). Among efalizumab-treated responders at week 12, response was maintained through week 24 in 77 per cent of those who continued against 20 per cent of those switched to placebo. After discontinuation at week 24, PASI-50 was maintained in roughly 30 per cent over the following 12 weeks.
Source
Lebwohl M et al. N Engl J Med 2003;349:2004-2013
Role in the trial
Not matched to a registered study
Identity check
no open identity hold
Audit mark
verified
Review state
Written into the record, not signed off as a reviewed claim
Four PML cases in psoriasis patients, all fatal, all after three or more years
In plain words
The FDA searched its adverse-event database for brain infections reported with every psoriasis biologic. Efalizumab was the only one with cases in psoriasis patients. All of them died.
What was measured
Confirmed PML cases by psoriasis biologic in the FDA Adverse Event Reporting System through 30 January 2009
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
FDA Office of Surveillance and Epidemiology searched the Adverse Event Reporting System for post-marketing reports of progressive multifocal leukoencephalopathy associated with the biologics approved for psoriasis — adalimumab, alefacept, efalizumab, etanercept and infliximab — from approval to 30 January 2009. Twelve cases suggestive of PML were identified: adalimumab 1, efalizumab 4, etanercept 3, infliximab 4. Efalizumab was the only drug with cases occurring in the setting of psoriasis. All four efalizumab cases presented three years or more after treatment initiation, and all resulted in death. The cases attributed to the other agents occurred in conditions other than psoriasis and were confounded by concurrent immunosuppression or were not confirmed PML.
Source
Kothary N, Diak IL, Brinker A, Bezabeh S, Avigan M, Dal Pan G. J Am Acad Dermatol 2011;65:546-551
Role in the trial
Not matched to a registered study
Identity check
no open identity hold
Audit mark
verified
Review state
Written into the record, not signed off as a reviewed claim
The mechanism was demonstrated directly in a treated patient
In plain words
In one patient, researchers measured how well T cells could cross a vessel wall while on the drug, and again after it was removed. Migration recovered as the blocked protein reappeared.
What was measured
T-cell transendothelial migration against LFA-1 surface expression, before and after plasma exchange
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Two patients with severe psoriasis treated for three years or more developed fatal PML with JC virus identified in cerebrospinal fluid; both died two and six months after onset despite plasma exchange and signs of immune reconstitution, with PML confirmed neuropathologically. Serial studies in one patient showed that efalizumab treatment was associated with reduced transendothelial migration by peripheral T cells in vitro, and that as LFA-1 expression on peripheral T cells rose after plasma exchange, in vitro migration increased with it. That is a direct dose-response measurement of the proposed mechanism in the affected patient rather than an inference from the drug's target.
Source
Schwab N et al. Neurology 2012;78:458-467
Role in the trial
Not matched to a registered study
Identity check
no open identity hold
Audit mark
verified
Review state
Written into the record, not signed off as a reviewed claim
The same mechanism, the opposite regulatory outcome, in the same years
In plain words
Natalizumab blocks a closely related adhesion protein, also caused PML, and was brought back under a monitoring programme. Efalizumab was not. The difference is what the alternatives looked like.
What was measured
Not recorded
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Natalizumab, an anti-alpha-4-integrin antibody, was withdrawn in February 2005 after three PML cases and returned in June 2006 under a restricted distribution and monitoring programme. Efalizumab, an anti-CD11a antibody blocking the same functional step in a different integrin, was withdrawn in 2009 and never returned. The distinguishing facts are not mechanistic. Natalizumab reduced the relapse rate by 68 per cent in relapsing multiple sclerosis, a progressive neurological disease with limited alternatives. Efalizumab produced PASI-75 in a quarter of psoriasis patients at a time when ustekinumab was being approved and the TNF inhibitors were established. When the benefit is modest and substitutes exist, a monitoring programme does not change the arithmetic.
Source
Kothary N et al. J Am Acad Dermatol 2011;65:546-551; Polman CH et al. N Engl J Med 2006;354:899-910
Role in the trial
Not matched to a registered study
Identity check
no open identity hold
Audit mark
verified
Review state
Written into the record, not signed off as a reviewed claim
No incidence rate was ever established, only a numerator
In plain words
Four cases were reported. Nobody knows how many people had taken the drug for three or more years, so the actual risk per patient was never calculated.
What was measured
That a per-patient PML incidence rate for efalizumab is known
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
The FDA analysis rests on spontaneous post-marketing reports, which the authors identify as limited by underreporting and by variable quality of information. There is a numerator — four confirmed cases in psoriasis — and no reliable denominator of patients treated for three or more years, which is the exposure window in which every case occurred. Statements of the form "the PML risk with efalizumab was one in N" therefore do not derive from the evidence base that caused the withdrawal. The authors were explicit that a treatment duration which does not place patients at risk has not been defined. The decision was made on four fatal cases with a plausible and later demonstrated mechanism, not on a rate.
Source
Kothary N et al. J Am Acad Dermatol 2011;65:546-551
Role in the trial
Not matched to a registered study
Identity check
no open identity hold
Audit mark
caution
Review state
Written into the record, not signed off as a reviewed claim
Withdrawal came from Europe first
In plain words
The European Medicines Agency recommended suspension in February 2009. The United States withdrawal followed in the same year.
What was measured
Not recorded
Effect estimate
No reviewed effect estimate exists for this record.
Limits
This entry records a failure.
From the source
The European Medicines Agency's Committee for Medicinal Products for Human Use recommended suspension of the Raptiva marketing authorisation on 19 February 2009, concluding that the benefits no longer outweighed the risks given the reports of PML. Genentech announced a phased voluntary withdrawal from the United States market, completed in 2009. The sequence matters for reading the record: the European suspension is a formal regulatory act with a published assessment, while the United States exit was a sponsor decision, so the two jurisdictions leave different kinds of document behind for the same event.
Source
European Medicines Agency, Raptiva (efalizumab): withdrawn medicine, marketing authorisation withdrawn 2009; Kothary N et al. J Am Acad Dermatol 2011;65:546-551
Role in the trial
Not matched to a registered study
Identity check
no open identity hold
Audit mark
verified
Review state
Written into the record, not signed off as a reviewed claim
Rebound psoriasis on withdrawal was a documented problem of stopping it
In plain words
Stopping the drug abruptly could make psoriasis flare worse than before, which complicated moving thousands of patients off it at once.
What was measured
Maintenance of PASI-75 on continued treatment versus switch to placebo, and PASI-50 maintenance after discontinuation
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
The pivotal trial measured what happens on discontinuation: among subjects who achieved PASI-75 at week 12 and were then switched to placebo, response was maintained in 20 per cent against 77 per cent of those who continued, and after treatment stopped at week 24 only about 30 per cent maintained even PASI-50 over 12 weeks of follow-up. Rebound and severe flares including erythrodermic and pustular presentations were recognised on discontinuation. The 2009 withdrawal therefore required a supervised transition rather than a stop order, which is a class of harm that a withdrawal itself creates and that a withdrawal notice does not usually count.
Source
Lebwohl M et al. N Engl J Med 2003;349:2004-2013
Role in the trial
Not matched to a registered study
Identity check
no open identity hold
Audit mark
verified
Review state
Written into the record, not signed off as a reviewed claim

Where else this substance is registered

FDA substance identifier (UNII)
XX2MN88N5D
CAS registry number
214745-43-4
ChEMBL
CHEMBL1201575
WHO international nonproprietary name list entry
8122
RxNorm concept
356988
EMA substance identifier
100000089542
DrugBank
DB00095

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A humanised anti-CD11a antibody that produced PASI-75 in 22 to 28 per cent of patients against 5 per cent on placebo at 12 weeks, withdrawn in 2009 after four reports of progressive multifocal leukoencephalopathy in psoriasis patients — every one of them fatal, and every one after three or more years of continuous treatment.

Recorded evidence blocks (7)

What did Efalizumab's largest trial (1200 people) and its longest (9 years) measure?


1200 people in Efalizumab's largest registered study, 9 years in its longest registered window, measuring Mean concentrations of anti-<phi>X174 antibodies in the FU period 2 weeks after the FU Day 70 vaccinations in Group A compared with mean antibody concentrations 2 weeks after the Treatment Day 63… ClinicalTrials.gov · 2026-09-01

11 phase2, 10 phase1, 8 phase3, 7 phase4, 2 na, 1 na or unstated; NCT00276250; 2014-12; no ageing endpoint recorded. Last human test completed 2014, NCT00276250.

Interpretation These counts include studies where Efalizumab 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
    11
  • phase1
    10
  • phase3
    8
  • phase4
    7
  • na
    2
  • na or unstated
    1
1 more recorded row
  • Last recorded human test NCT00276250
    2014-12

recorded 2026-09-01 · last checked 2026-09-04

From mouse to human: where has Efalizumab shown lifespan?


mouse: mechanism-only, rat: lifespan and human: biomarker (34): the rungs where Efalizumab has a recorded finding. Europe PMC and ClinicalTrials.gov · organism ladder · 2026-09-01

Mean concentrations of anti-<phi>X174 antibodies in the FU period 2 weeks after the FU Day 70 vaccinations in Group A compared with mean antibody… — the recorded outcome words.

Yeast C. elegans Drosophila Mouse mechanism-onlyRat lifespanDog Non-human primate Human biomarker
Show the evidence
  • mouse
    mechanism-only
  • rat
    lifespan
  • human NCT00382512
    biomarker; Mean concentrations of anti-<phi>X174 antibodies in the FU period 2 weeks after the FU Day 70 vaccinations in Group A compared with mean antibody concentrations 2 weeks after the Treatment Day 63 vaccination in Group C; 34

recorded 2026-09-01 · last checked 2026-09-04

11 of Efalizumab's trials stopped: safety, accrual/recruitment, other?


safety (3), accrual/recruitment (2) and other (6): Efalizumab's stop wording, clustered. ClinicalTrials.gov · 2026-09-01

"efalizumab was withdrawn from market; full 2 years follow-up only 14 patients"; 11 of 34 registered studies

Show the evidence

Trial

  • NCT00184366
    terminated; "efalizumab was withdrawn from market; full 2 years follow-up only 14 patients"
  • NCT00308204
    withdrawn; "inadequate number of enrolled study subjects"
  • NCT00344448
    terminated; "Increased risk of PML associated with raptiva in other studies"
  • NCT00472082
    terminated; "This study was terminated at the request of the drug manufacturer."
  • NCT00489216
    terminated; "insufficient accrual/funding withdrawn"
  • NCT00672204
    terminated; "Raptiva was withdrawn from the market"
5 further recorded trials
  • NCT00676559
    withdrawn; "Unrelated serious adverse events involving one of the proposed medications"
  • NCT00737763
    withdrawn; "drug withdrawn from market"
  • NCT00746980
    withdrawn; "Newly identified safety concerns have changed the risk and benefit considerations"
  • NCT00777400
    withdrawn; "New safety information reported in the post-marketing setting with efalizumab for treatment of chronic plaque psoriasis and trial conduct feasibility issues."
  • NCT00972543
    terminated; "The study was terminated after the European Medicines Evaluation Agency recommended to suspend the marketing authorisation of Raptiva in the European Union"

recorded 2026-09-01 · last checked 2026-09-04

Which of experiencing adverse events, insulin independent full islet graft function and pasi psoriasis area and severity index did Efalizumab's trials measure?


experiencing adverse events, insulin independent full islet graft function and pasi psoriasis area and severity index lead 5 outcome terms across Efalizumab's trials. ClinicalTrials.gov · 2026-09-01

Interpretation renal allograft survival and psoriasis area and severity index follow.

Show the evidence
  • pasi psoriasis area and severity index
    1
  • experiencing adverse events
    1
  • insulin independent full islet graft function
    1
  • renal allograft survival
    1
  • psoriasis area and severity index
    1

recorded 2026-09-01 · last checked 2026-09-04

Which 14 trials of Efalizumab posted no result?


Posted no result
14 of 14 completed trials
Registrations
NCT00109317, NCT00109252, NCT00034203, NCT00051662, NCT00096980 and NCT00338143, and 8 more
Completion dates
oldest 2003-02; newest 2007-07
Show the evidence

Trial

  • NCT00109317
    2003-02
  • NCT00109252
    2003-04
  • NCT00034203
    2003-08
  • NCT00051662
    2004-02
  • NCT00096980
    2004-05
  • NCT00338143
    2004-05
8 further recorded trials
  • NCT00096603
    2004-07
  • NCT00256139
    2004-10
  • NCT00133107
    2005-11
  • NCT00442650
    2005-12
  • NCT00226057
    2006-05
  • NCT00134134
    2006-08
  • NCT00312026
    2006-11-28
  • NCT00302445
    2007-07

At the median, Efalizumab's trials enrolled 11 people — anything larger?


Median enrolment
11
Largest enrolment
1200
Registered trials counted
32

What do 558 spontaneous reports say about Efalizumab — 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.

Efalizumab appears in spontaneous reports to regulators. Across the 10 most-reported reaction terms, 558 reaction mentions were counted: psoriasis 264; white blood cell count increased 60; myalgia 47; psoriatic arthropathy 36. open-targets-adr · CHEMBL1201575 · 2026-06-24

Show the evidence
  • psoriasis
    264
  • white blood cell count increased
    60
  • myalgia
    47
  • psoriatic arthropathy
    36
  • influenza like illness
    34
  • arthritis
    33
4 more recorded rows
  • skin exfoliation
    28
  • lymphocyte count increased
    21
  • lymphocytosis
    18
  • dermatitis exfoliative
    17

recorded 2026-06-24 · last checked 2026-09-04

Where it is registered
Identifiers, relations and other names

The exact record

ChEMBL id
CHEMBL1201575
CAS number
214745-43-4
RxCUI
356988
Development code
ANTI-CD11A, HU-1124, HU1124
Trade name
Raptiva
Also called
EFALIZUMAB [EMA EPAR], EFALIZUMAB [HSDB], EFALIZUMAB [MART.], EFALIZUMAB [MI], EFALIZUMAB [USAN], EFALIZUMAB [VANDF], Efalizumab [WHO-DD], IMMUNOGLOBULIN G1, ANTI-(HUMAN CD11A (ANTIGEN)) (HUMAN-MOUSE MONOCLONAL HU1124 .GAMMA.1-CHAIN), DISULPHIDE WITH HUMAN-MOUSE MONOCLONAL HU1124 LIGHT CHAIN, DIMER
Sources (6)

Sources

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

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