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Disease indication · a behaviour of Crohn’s disease

Fibrostenotic Crohn's Disease

The endoscope that solved luminal Crohn's cannot see this. Every approved drug targets inflammation; the scarring it leaves behind still sends patients to surgery.

Therapeutic goal

Disease-modifying: Prevent, halt or reverse the collagen deposition that thickens and narrows the bowel wall. Nothing has ever been approved to do this in the intestine — not because nobody has tried a mechanism, but because there is no accepted way to demonstrate that a mechanism worked.

Approved antifibrotics for the gut
0

None anywhere in the world. The lung has two approved antifibrotics; the intestine has none, despite fibrosis being the dominant reason Crohn's patients reach an operating theatre.

high confidence
Accepted endpoint
None

There is no validated, regulator-accepted measure of intestinal fibrosis or its reversal. This — not the absence of candidate mechanisms — is what the field itself now identifies as the bottleneck.

high confidence
Patients reaching surgery
~50% by 10 years

Roughly half of Crohn's patients undergo surgery within ten years of diagnosis, and stricturing disease is the leading reason — a rate that modern biologics have reduced but not eliminated.

moderate confidence
Progress to B2 or B3
~50% by 20 years

Under the Montreal classification most patients begin as non-stricturing, non-penetrating disease; about half convert to stricturing or penetrating behaviour over two decades. The disease's own taxonomy treats this as a distinct behaviour, not a complication.

moderate confidence

Three behaviours, one disease

Crohn's is classified by behaviour, not just by site — the Montreal system types it as inflammatory, stricturing or penetrating — and about half of patients who start with inflammation alone convert to one of the other two within twenty years. The three respond very differently to the drugs on this site, and the numbers on any one of these pages describe only its own column.

Luminal inflammation (B1)

Largely solved

Mucosal and transmural inflammation of the bowel wall. Five approved mechanisms, and the behaviour every current drug was developed against.

Approved mechanisms
Five, independently effective
Accepted endpoint
Endoscopic healing — objective, and required since the 2020s
What treatment achieves
Inflammation can be driven into remission and the mucosa healed
Preclinical models
Moderately predictive; several carried mechanisms to approval
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Stricturing (B2)

Largely unaddressed

Fibrotic narrowing of the bowel. The leading reason Crohn's patients reach an operating theatre, and the behaviour no drug has ever been approved to treat.

Approved mechanisms
None — the lung has two approved antifibrotics, the gut has none
Accepted endpoint
None at all: imaging cannot separate scar from swelling
What treatment achieves
Obstruction is relieved mechanically; the fibrosis is untouched
Preclinical models
Two reproduce genuine fibrosis, and none has been carried to a readout

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Penetrating and perianal (B3)

Partly addressed

Fistulas tunnelling through the bowel wall to other organs or the skin. Anti-TNF genuinely helps, and a locally injected cell therapy was approved in Europe and then withdrawn when its confirmatory trial failed.

Approved mechanisms
Anti-TNF, plus a cell therapy approved in the EU and later withdrawn
Accepted endpoint
Fistula closure on compression, plus MRI-based indices
What treatment achieves
Real closure rates, well short of the luminal disease's
Preclinical models
Essentially none — no usable animal model of fistula formation

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The split is the field's own, not an editorial one. It matters here because every approved Crohn's mechanism was developed and measured against the first column: the endoscope that made luminal disease tractable reaches the mucosa, which is exactly where the other two behaviours are not.

What a trial has to prove

Nothing here is accepted, and the reason is mechanical rather than statistical. Parkinson's cannot separate a disease-modifying effect from symptom relief. This disease has a blunter problem: on every available instrument, inflamed bowel and scarred bowel look substantially alike — and the fibrosis is transmural, so the endoscope that solved the luminal disease cannot reach it.

No endpoint is accepted as establishing disease-modifying benefit in Fibrostenotic Crohn's Disease. Every measure below is either contested or exploratory. A trial can therefore succeed on its own terms and still not support the claim.

Endpoints used in Fibrostenotic Crohn's Disease trials for the Disease-modifying goal, how each is measured, how long it takes to read, and whether it is accepted as evidence for the claim. Select one for detail.
EndpointTypeRead atStanding
Fibrosis reversal or stabilisationComposite52+ weeksNone accepted
MR / CT enterography stricture morphologyImaging26–52 weeksExploratory
Intestinal ultrasoundImaging8–52 weeksExploratory
Surgery-free and obstruction-free survivalClinician-rated1–5+ yearsContested
Accepted
Regulators accept this as evidence for the claim.
Surrogate
A stand-in for clinical benefit, accepted or under negotiation for accelerated approval — the benefit itself must still be confirmed.
Contested
Used in trials, but not accepted as establishing the claim on its own.
Exploratory
Informative for development — target engagement, enrichment — but never the basis of an approval.
None accepted
No endpoint has ever been accepted as establishing this claim in this disease.

Preclinical model validity

Two models here reproduce genuine intestinal fibrosis, and neither has ever carried a drug to a readout — because there is nothing to carry one to. When no endpoint exists, model validity stops being the binding constraint, and this matrix should be read as untested rather than as failed.

Validity of Fibrostenotic Crohn's Disease preclinical models across face, construct, and predictive validity. Predictive validity is shown for the Reverse fibrosis goal.
ModelFaceDoes it look like the disease?ConstructDoes it arise the same way?PredictiveReverse fibrosis
ValidityNoneLowModerateHighNo evidencenever tested — not a low score

Translation ledger

The shortest ledger on this site, and the emptiness is the finding. No antifibrotic has ever been approved for the gut, and the entries below are mostly things that treat the inflammation alongside the fibrosis without touching the fibrosis itself.

Interventions that showed benefit in a Fibrostenotic Crohn's Disease preclinical model, and what happened when they reached the clinic.
InterventionRested onPrimary endpointReachedOutcome
Antifibrotic repurposing from other organs
Various · Agents targeting TGF-β signalling, ROCK, LOXL2 and related fibrogenic pathways
Chronic DSSHuman myofibroblasts
No agent has reached a controlled trial with a fibrosis endpoint in this disease
Preclinical / exploratory
2024
Ongoing
Early combined immunosuppression strategy
Investigator-led · Treating to a target of mucosal healing early, before fibrosis accumulates
Mucosal healing and clinical remission; stricture and surgery rates as longer-term secondary outcomes
Phase 3 strategy trials
REACT and related treat-to-target programmes · 2015
Benefit, no approval
TNF inhibitors
Multiple · Neutralisation of tumour necrosis factor
Chronic TNBSChronic DSS
No trial has used a fibrosis endpoint; evidence comes from stricture-related surgical rates as secondary or observational outcomes
Observational for this indication
2010
Benefit, no approval
Endpoint · Composite

Fibrosis reversal or stabilisation

Whether the collagen deposition narrowing the bowel has been halted or reduced

None acceptedRead at 52+ weeks
Scale
No validated scale exists. Candidate instruments disagree, and none has been qualified with a regulator.
How it is administered
There is no accepted instrument. Cross-sectional imaging, intestinal ultrasound, endoscopic passage and surgical outcome have all been proposed, and none has been validated against a reference standard that regulators accept.
What it contains
  • The measurement problem: on MR and CT enterography, inflamed bowel wall and fibrotic bowel wall both appear thickened, and separating the two reliably remains unsolved
  • The access problem: the fibrosis is transmural, so the endoscope that validated luminal healing physically cannot sample the tissue that matters
  • The reference-standard problem: the only definitive assessment is histology of a resected segment, available exclusively from patients who have already reached surgery — that is, from treatment failures
  • The timescale problem: strictures form over years, so any prevention trial must run far longer than a conventional programme
How a score is produced
None established. Without a reference standard obtainable from patients who have not had surgery, candidate measures cannot be calibrated against the thing they claim to measure.
What counts as success
No endpoint has ever been accepted for intestinal fibrosis by any regulator. Note how this differs from Parkinson's: there the problem is confounding — a symptomatic effect moves the progression score, so the measure cannot isolate the claim. Here the problem is more basic. The instrument cannot distinguish the target from its most common mimic, and cannot physically reach the tissue.
Why it matters here

The reason this page exists. Every mechanism below was tested against inflammation because inflammation is what the field could measure.

Consortium work on stricture outcome definitions; regulatory guidance gaps in intestinal fibrosishigh confidence

What the clinic costs

Every figure here is hypothetical, because you cannot size a pivotal trial against an endpoint that does not exist. What the model can show honestly is the shape of the problem: a long trial, an imaging endpoint nobody has validated, and a likelihood of approval with no precedent to anchor it.

Entirely hypothetical — no antifibrotic has ever run a controlled trial with a fibrosis endpoint in this disease. What the model can show is the shape such a trial would need: large, and long enough to outlast the years over which strictures actually form. That duration, not the headcount, is what makes it the second most expensive disease-modifying programme on this site.

7.2×
the exposure
4.1×
the cost

1,618 patient-years against 225 for a symptomatic programme. Cost grows more slowly than exposure because the fixed enrolment cost of each participant — screening, baseline imaging — is paid once however long they are then followed. The goal, not the molecule, still sets the budget.

Citedenrolment, duration and study counts, from named trials.Assumptionthe two cost parameters below.Computedeverything else.

Clinical programme by phase: enrolment, duration, exposure in patient-years, and modelled cost.
PhaseDesignN × studiesDurationPatient-yearsModelled cost
Phase 1
SAD / MAD, healthy volunteers
Reads: Safety, tolerability and pharmacokinetics
The one phase here that is not hypothetical in design, because it does not depend on being able to measure fibrosis.
Conventional first-in-human designmoderate confidence
50
9 mo
38
$1.4M–$2.2M
Phase 2
Dose-ranging against an imaging endpoint
Reads: Change in stricture morphology on cross-sectional imaging or ultrasound elastography
A full year, because fibrosis does not move on the timescale inflammation does — and read against an endpoint nobody has validated, which is the reason no sponsor has run this trial.
Hypothetical; no antifibrotic Phase 2 has been run in this diseaselow confidence
180
1 yr
180
$5.8M–$9.5M
Phase 3
Two pivotals, two years, imaging plus surgery-free survival
Reads: Stricture morphology on imaging, with obstruction-free and surgery-free survival
Two years is the floor for a structural claim in a disease whose strictures form over years, and it is what makes this programme cost more than any other on this site except Parkinson's disease modification. The endpoint it would read does not yet exist.
Hypothetical; sized by analogy with antifibrotic programmes in other organslow confidence
350 × 2
2 yr
1,400
$36M–$62M
Total3.8 yr of clinical development, treating phases as sequential1,618$44M–$73M
AssumptionThe two cost parameters
Fixed, per participant
$12,000–$18,000
Running, per year on study
$20,000–$35,000

Model assumptions, not citations — the only free parameters here. The fixed component covers screening, baseline imaging and randomisation, incurred once per participant; the running component covers visits, monitoring and site fees for each year on study. Splitting them matters: a single rate per patient-year would price short symptomatic trials far below what pivotal trials actually cost.

Calibration: priced this way a single pivotal trial in this programme comes to $4.0M–$6.4M, against published pivotal trial costs of $1233M (median $19M) across all indications. The symptomatic column here is procedural — dilation and surgical series rather than drug trials — so it lands far below the published range, which is set by pharmaceutical programmes. The disease-modifying column is hypothetical throughout: no trial of this shape has ever been run in this disease. Moore et al., JAMA Internal Medicine 2018.

ComputedRisk-adjusted cost
$870M$1.5B

Programme cost divided by a 5% likelihood of approval from Phase 1 in intestinal fibrosis (no observed programme history) — what one success costs once the failures are paid for. Not a published figure. No antifibrotic has ever run a registrational programme in the intestine, so there is no denominator from which a rate could be computed.

Read this one carefully: The most speculative number on this site, and stated as an assumption rather than a citation. It is set below every observed rate here on the reasoning that a field with no accepted endpoint cannot reliably demonstrate success even with a working drug — the constraint this whole page is about. Treat the resulting risk-adjusted figures as an illustration of what an absent endpoint costs, not as a forecast.

low confidence
For scale — industry-wide, per approved drug
$985M
Median R&D cost per approval
Wouters et al., JAMA 2020 (capitalised; estimates range $314M–$2.8B)
$2.6B
Capitalised cost per approval
DiMasi et al., J Health Econ 2016

These are portfolio figures spanning all of R&D and already carry the cost of failure, so they are not comparable with the per-programme clinical costs above — they are the order of magnitude those costs roll up into.

What is changing

The field has worked out that the endpoint is the bottleneck, and is building one — which is roughly where ulcerative colitis stood in the 1980s and where DM1 stands now.

Building the endpoint first

Dedicated consortium work is now attempting what this disease has never had: an agreed definition of a stricture, an agreed way to measure it, and an outcome regulators would accept. Getting that right is the precondition for everything else, and it puts this disease roughly where ulcerative colitis stood before the Mayo score and where DM1 stands with its splicing surrogate today.

Consortium initiatives on stricture definition and outcome measurement in Crohn's disease

Ultrasound elastography may separate scar from swelling

Shear-wave elastography measures tissue stiffness directly rather than inferring it from thickness, which is the one thing every current instrument fails at. If it can reliably distinguish a fibrotic stricture from an inflamed one at the bedside, the measurement problem that defines this page becomes tractable — with the same operator-variability hurdle that central blinded reading had to solve for the endoscope.

Shear-wave elastography validation studies in Crohn's strictures

The organ is the outlier, not the biology

Fibrosis is being treated successfully in the lung and pursued hard in the liver and kidney, drawing on largely shared pathways. The intestine is the conspicuous gap, and the reason is not that intestinal fibrosis is more mysterious — it is that the other organs have measurable endpoints. Lung fibrosis has spirometry; liver fibrosis has biopsy and increasingly elastography. The bowel has neither.

Cross-organ antifibrotic development literature

Educational. This is the most speculative page on this site, and deliberately so: no endpoint is accepted for intestinal fibrosis, so the trial designs and costs modelled here are hypothetical rather than drawn from registrational precedent, and are flagged low confidence throughout. The luminal inflammatory disease is covered separately. Not medical, regulatory, or investment advice.