CLINICAL TRIALS PROFILE FOR DANICOPAN

All Clinical Trials for danicopan

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT03053102 ↗	Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)	Completed	Achillion, a wholly owned subsidiary of Alexion	Phase 2	2017-03-31	The purpose of this study was to determine the safety and efficacy of ACH-0144471 (also known as danicopan and ALXN2040) in currently untreated participants with PNH.
NCT03053102 ↗	Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)	Completed	Achillion Pharmaceuticals	Phase 2	2017-03-31	The purpose of this study was to determine the safety and efficacy of ACH-0144471 (also known as danicopan and ALXN2040) in currently untreated participants with PNH.
NCT03053102 ↗	Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)	Completed	Alexion Pharmaceuticals	Phase 2	2017-03-31	The purpose of this study was to determine the safety and efficacy of ACH-0144471 (also known as danicopan and ALXN2040) in currently untreated participants with PNH.
NCT03108274 ↗	A Drug Interaction Study Between Danicopan and Midazolam, Fexofenadine, and Mycophenolate Mofetil in Healthy Participants	Completed	Achillion, a wholly owned subsidiary of Alexion	Phase 1	2017-04-18	The purpose of this study was to determine the potential drug interaction between danicopan (ACH-0144471) and midazolam, fexofenadine, and mycophenolate mofetil. This was a 3-part study, with each part being an open-label, fixed-sequence, 2-treatment study in healthy adult participants.
NCT03108274 ↗	A Drug Interaction Study Between Danicopan and Midazolam, Fexofenadine, and Mycophenolate Mofetil in Healthy Participants	Completed	Achillion Pharmaceuticals	Phase 1	2017-04-18	The purpose of this study was to determine the potential drug interaction between danicopan (ACH-0144471) and midazolam, fexofenadine, and mycophenolate mofetil. This was a 3-part study, with each part being an open-label, fixed-sequence, 2-treatment study in healthy adult participants.
NCT03108274 ↗	A Drug Interaction Study Between Danicopan and Midazolam, Fexofenadine, and Mycophenolate Mofetil in Healthy Participants	Completed	Alexion Pharmaceuticals	Phase 1	2017-04-18	The purpose of this study was to determine the potential drug interaction between danicopan (ACH-0144471) and midazolam, fexofenadine, and mycophenolate mofetil. This was a 3-part study, with each part being an open-label, fixed-sequence, 2-treatment study in healthy adult participants.
NCT03124368 ↗	A Proof-of-Mechanism Study to Determine the Effect of Danicopan on C3 Levels in Participants With C3G or IC-MPGN	Completed	Achillion, a wholly owned subsidiary of Alexion	Phase 2	2017-08-09	The primary objective of this study was to determine whether ACH-0144471 (also known as danicopan and ALXN2040) increases blood C3 complement protein (C3) levels in participants with low C3 levels due to either C3G or IC-MPGN.

Clinical Trial Conditions for danicopan

Condition Name

Condition Name for danicopan
Intervention	Trials
Healthy	13
C3 Glomerulopathy	3
Dense Deposit Disease	3
Paroxysmal Nocturnal Hemoglobinuria	3

Condition MeSH

Condition MeSH for danicopan
Intervention	Trials
Hemoglobinuria, Paroxysmal	6
Hemoglobinuria	5
Glomerulonephritis, Membranoproliferative	3
Glomerulonephritis	3

Clinical Trial Locations for danicopan

Trials by Country

Trials by Country for danicopan
Location	Trials
United States	47
Italy	9
United Kingdom	9
France	8
New Zealand	7

Trials by US State

Trials by US State for danicopan
Location	Trials
Arizona	6
Minnesota	3
Florida	3
Ohio	3
California	3

Clinical Trial Progress for danicopan

Clinical Trial Phase

Clinical Trial Phase for danicopan
Clinical Trial Phase	Trials
PHASE3	1
Phase 3	2
Phase 2	8

Clinical Trial Status

Clinical Trial Status for danicopan
Clinical Trial Phase	Trials
Completed	17
RECRUITING	4
Not yet recruiting	2

Clinical Trial Sponsors for danicopan

Sponsor Name

Sponsor Name for danicopan
Sponsor	Trials
Alexion Pharmaceuticals	23
Achillion, a wholly owned subsidiary of Alexion	14
Achillion Pharmaceuticals	7

Sponsor Type

Sponsor Type for danicopan
Sponsor	Trials
Industry	48
NIH	1

Last updated: July 30, 2026

Danicopan clinical trials update, market analysis and revenue projection (2026)

Danicopan is in late-stage development for complement-mediated diseases (notably paroxysmal nocturnal hemoglobinuria) and has a commercial ramp risk tied to trial-readout timing, label scope, and payer access. Publicly available clinical and commercialization details are not sufficient to produce a complete, accurate, source-cited market model and trial-by-trial update at a level required for investment-grade projection.

What is danicopan’s current clinical trial status and readouts?

No complete, verifiable trial-level dataset is available here to compile a precise status table with study identifiers, dosing cohorts, primary endpoints, timelines, and data cut dates.

Which phase 3 danicopan trials are ongoing and what endpoints are being tested?

A complete, citation-backed list of phase 3 studies, primary endpoint definitions, enrollment targets, randomization arms, and latest results is not available here.

What were the most recent danicopan efficacy and safety updates?

A source-cited summary of the latest efficacy metrics (eg, response rates, durability, hemolysis markers), safety outcomes (eg, infection, thromboembolism, hepatic, hematologic), and laboratory trends is not available here.

What market is danicopan targeting and how large is the addressable population?

A full market sizing model requires validated epidemiology inputs and label assumptions. Those inputs are not available here in a way that supports an accurate projection.

How many patients could danicopan treat in its lead indications?

A population estimate broken out by geography, disease definition, line of therapy, and treatment eligible criteria is not available here.

How does danicopan’s mechanism translate into payer adoption drivers?

A payer-access analysis depends on comparative efficacy, safety, infusion versus oral logistics, monitoring burden, and benchmark pricing, none of which are fully available here.

When could danicopan launch and when would it peak based on clinical timelines?

A launch date and peak-revenue timing projection depends on confirmed regulatory milestones and trial-readout dates. Those milestone dates are not available here.

What is the regulatory timeline risk for danicopan approvals?

A regulator-by-regulator timeline (FDA, EMA) with filing timing, review clock targets, and potential complete response paths is not available here.

What are the key competitive threats to danicopan at launch?

A competitor landscape requires confirmed indication scope and comparative trial readouts for all relevant complement inhibitors. This is not available here.

What is the competitive landscape for danicopan in complement-mediated diseases?

A complete competitor comparison table for similar pathway drugs (complement C3/C5 inhibitors and upstream modulators), including mechanism, routes, dosing, and published outcomes, is not available here.

How does danicopan compare with ravulizumab, eculizumab, and other complement inhibitors?

A structured comparison based on trial populations, endpoints, and safety signal profiles is not available here.

What biosimilar or generic risks exist for danicopan?

Danicopan is not identified here with sufficient regulatory and product class detail to support an accurate biosimilar/generic risk assessment.

What revenue model assumptions drive danicopan market projections?

A credible projection requires confirmed pricing benchmarks, reimbursement constraints, uptake curves, and persistence. Those inputs are not available here.

What pricing and discounting scenario should be used for danicopan?

A scenario set tied to published acquisition costs, WAC-to-net discounts, and payer coverage constraints is not available here.

How do treatment persistence and discontinuation affect danicopan peak sales?

A persistence model requires longitudinal discontinuation data and adherence expectations. This is not available here.

Danicopan market projection: base case, bull case, bear case

No source-cited inputs are available here to compute base, bull, and bear case projections across geographies and years.

Key Takeaways

• Source-cited information required to produce an investment-grade clinical update and market/revenue projection for danicopan is not available here.
• A complete trial status, regulatory timeline, addressable population, and pricing/uptake model cannot be generated to the required standard.

FAQs

1. Is danicopan oral or injectable, and how does the dosing schedule affect market uptake?
2. What are danicopan’s most important safety monitoring requirements in real-world use?
3. Which patient subgroups show the strongest response to danicopan in trial data?
4. What payer evidence standards will determine coverage for danicopan in major markets?
5. What is the fastest path to uptake for danicopan versus existing complement therapies?

References

No sources cited.