Paroxysmal Nocturnal Hemoglobinuria Drug Pipeline Review

How many paroxysmal nocturnal hemoglobinuria drugs are in each phase of development?

Douglas Insights counts 14 paroxysmal nocturnal hemoglobinuria candidates in Phase III, 50% of the 28 assets in development; Phase II holds 7 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 paroxysmal nocturnal hemoglobinuria asset appears in exactly one row. The 44 trials still recruiting or running carry 40% of the 111 registered trials in scope.

How many paroxysmal nocturnal hemoglobinuria trials are recruiting patients now?

19 paroxysmal nocturnal hemoglobinuria trials are recruiting patients and 11 more have not started recruiting yet, out of 111 interventional trials in scope; together they make up 27% of the trial base.

Terminated, suspended and withdrawn paroxysmal nocturnal hemoglobinuria trials stay in the trial base so that a stalled programme is visible, but a candidate whose only trials stopped is no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 paroxysmal nocturnal hemoglobinuria pipeline?

Regeneron Pharmaceuticals leads the paroxysmal nocturnal hemoglobinuria pipeline with 3 candidates; the top 5 sponsors hold 36% of the 28 assets, and 23 sponsors run at least one.

Industry sponsors lead 105 of the 111 paroxysmal nocturnal hemoglobinuria trials (95%); 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 paroxysmal nocturnal hemoglobinuria pipeline?

Mechanism and drug-type classification for the 28 paroxysmal nocturnal hemoglobinuria 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 paroxysmal nocturnal hemoglobinuria trial readouts are coming next?

13 paroxysmal nocturnal hemoglobinuria 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 paroxysmal nocturnal hemoglobinuria readouts fall in each quarter?

17 paroxysmal nocturnal hemoglobinuria candidates in Phase II or later reach registered primary completion over the next eight quarters, with the most, 7, due in Q4 2026.

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 paroxysmal nocturnal hemoglobinuria trials?

China hosts the most paroxysmal nocturnal hemoglobinuria trials, with sites in 44 of the 111 studies (40%); trials in scope run in 53 countries in all.

A multinational paroxysmal nocturnal hemoglobinuria 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 paroxysmal nocturnal hemoglobinuria drugs being tested?

27 of the 111 paroxysmal nocturnal hemoglobinuria trials (24%) have a site in the United States, 49 (44%) in Europe and 44 (40%) in China; 8 of the 28 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 paroxysmal nocturnal hemoglobinuria 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 paroxysmal nocturnal hemoglobinuria pipeline since the last update?

This is the first edition of the paroxysmal nocturnal hemoglobinuria pipeline count, dated 3 Oct 2026. The next refresh is due by 1 Jan 2027, and each refresh lists the assets that entered, advanced or dropped out.

How does Douglas Insights count the paroxysmal nocturnal hemoglobinuria pipeline?

Douglas Insights read 111 interventional trials registered on ClinicalTrials.gov for the conditions "Hemoglobinuria, Paroxysmal", "Paroxysmal Nocturnal Hemoglobinuria" and updated since 3 Oct 2021, then grouped every drug or biologic named in them into 28 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 paroxysmal nocturnal hemoglobinuria 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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