Amyloidosis Drug Pipeline: 23 Assets by Phase

How many amyloidosis drugs are in each phase of development?

Douglas Insights counts 11 amyloidosis candidates in Phase III, 48% of the 23 assets in development; Phase II holds 5 and Phase I holds 7.

Phase I/II candidates sit in the Phase I row and Phase II/III candidates in the Phase II row, so each amyloidosis asset appears in exactly one row. The 95 trials still recruiting or running carry 53% of the 178 registered trials in scope.

Which approved amyloidosis medicines are still in trials?

2 medicines already on the market are still being tested in amyloidosis Phase IV trials; Acoramidis has the most, with 7 registered trials.

Post-approval amyloidosis trials test new doses, new patient groups and head-to-head comparisons. They set the standard of care that every candidate in development will be measured against, so the paid review profiles each one.

How many amyloidosis trials are recruiting patients now?

52 amyloidosis trials are recruiting patients and 15 more have not started recruiting yet, out of 178 interventional trials in scope; together they make up 38% of the trial base.

Terminated, suspended and withdrawn amyloidosis trials stay in the trial base so that a stalled programme is visible, but a candidate whose only trials stopped is no longer counted as in development.

Which companies lead the amyloidosis pipeline?

Alexion Pharmaceuticals, Inc. leads the amyloidosis pipeline with 3 candidates; the top 5 sponsors hold 43% of the 23 assets, and 18 sponsors run at least one.

Industry sponsors lead 75 of the 178 amyloidosis trials (42%); universities, hospitals and public funders lead the rest. A sponsor is credited with an asset when it is the lead sponsor on most of that asset's industry trials.

Which drug types and mechanisms dominate the amyloidosis pipeline?

Mechanism and drug-type classification for the 23 amyloidosis candidates is done by the research desk asset by asset, each with a cited source, and is published in the paid review together with the white-space map.

What amyloidosis trial readouts are coming next?

3 amyloidosis candidates in Phase II or later have a registered primary completion date in the next 12 months; the next three are below, each linked to its registry record.

Primary completion is the date a sponsor expects the last patient to finish the main measurement, so data usually follow within months. Dates marked estimated are the sponsor's own forecast on the registry and often move.

How many amyloidosis readouts fall in each quarter?

8 amyloidosis candidates in Phase II or later reach registered primary completion over the next eight quarters, with the most, 4, due in Q4 2027.

Each candidate is counted once, at its next readout. The paid catalyst calendar names every readout with its trial, sponsor and design, adds filings and regulatory decisions, and runs 24 months ahead.

Which countries host the most amyloidosis trials?

United States hosts the most amyloidosis trials, with sites in 95 of the 178 studies (53%); trials in scope run in 50 countries in all.

A multinational amyloidosis trial is counted once in every country where it has a site, so the shares add up to more than 100%. Country spread shows where sponsors are building the evidence that regulators in each market will ask for.

Where are amyloidosis drugs being tested?

95 of the 178 amyloidosis trials (53%) have a site in the United States, 62 (35%) in Europe and 44 (25%) in China; 17 of the 23 candidates in development are tested in the US.

Each region's trials are the evidence its regulator will review: the FDA in the United States, the EMA (with the MHRA and Swissmedic) in Europe, the NMPA in China and the PMDA in Japan. A multinational amyloidosis trial counts once in every region where it has a site, so the shares add up to more than 100%. A candidate tested only outside the US and Europe is often a licensing opportunity for sponsors in those markets.

What has changed in the amyloidosis pipeline since the last update?

This is the first edition of the amyloidosis pipeline count, dated 4 Oct 2026. The next refresh is due by 2 Jan 2027, and each refresh lists the assets that entered, advanced or dropped out.

How does Douglas Insights count the amyloidosis pipeline?

Douglas Insights read 178 interventional trials registered on ClinicalTrials.gov for the conditions "AL amyloidosis", "immunoglobulin light-chain amyloidosis", "transthyretin amyloidosis", "ATTR amyloidosis", "transthyretin amyloid cardiomyopathy", "familial amyloid polyneuropathy" (excluding "alzheimer disease", "dental plaque", "gingivitis") and updated since 4 Oct 2021, then grouped every drug or biologic named in them into 23 unique candidates.

1. Code names, generic names and brand names of the same molecule are merged into one candidate, and every merge is reviewed by the research desk.
2. A candidate counts when it has a trial that is recruiting, running, or completed in the last 24 months, and at least one industry-sponsored trial.
3. Its phase is the highest phase among those trials; placebo, standard-of-care and comparator arms are not candidates.
4. Each figure on this page is drawn from one frozen dataset that passes ten checks before release, and the owner approves every edition.

Read the full approach on the research methodology page, or browse other Pharmaceuticals research.

Frequently asked questions about the amyloidosis pipeline

Research & citation

This review was researched, written and reviewed by the Douglas Insights Research Desk under the Douglas Insights editorial standards. Material errors are logged in the corrections log. No section is sponsored.

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