Danicopan Clinical Trials

Size	Price	Qty
5mg	USD 417
25mg	USD 1,417

Clinical Trials

Numerous clinical trials evaluate danicopan across conditions involving alternative pathway complement activation, including paroxysmal nocturnal hemoglobinuria with extravascular hemolysis, geographic atrophy secondary to age-related macular degeneration, C3 glomerulopathy, and immune complex membranoproliferative glomerulonephritis. Spanning Phase 1 safety evaluations to pivotal Phase 3 studies, these protocols are sponsored by industry leaders like Alexion Pharmaceuticals and AstraZeneca alongside public entities such as the National Institute of Allergy and Infectious Diseases. Encompassing completed, recruiting, active not recruiting, and terminated statuses, these investigations reflect a comprehensive clinical development program.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT06449001	RECRUITING	Paroxysmal Nocturnal Hemoglobinuria; PNH; Extravascular Hemolysis	Alexion Pharmaceuticals, Inc.	2025-08-11	PHASE3
NCT05389449	ACTIVE_NOT_RECRUITING	Paroxysmal Nocturnal Hemoglobinuria	Alexion Pharmaceuticals, Inc.	2022-10-28	PHASE3
NCT05019521	TERMINATED	Geographic Atrophy	Alexion Pharmaceuticals, Inc.	2021-08-23	PHASE2
NCT04469465	COMPLETED	Paroxysmal Nocturnal Hemoglobinuria	Alexion Pharmaceuticals, Inc.	2020-12-16	PHASE3
NCT04988035	COMPLETED	COVID-19	National Institute of Allergy and Infectious Diseases (NIAID)	2021-08-03	PHASE2
NCT05019521	Active not recruiting	Geographic Atrophy	Alexion Pharmaceuticals Inc.	2021-08-23	Phase 2
NCT03459443	TERMINATED	C3 Glomerulonephritis; C3 Glomerulopathy; Immune Complex Membranoproliferative Glomerulonephritis; IC-MPGN; Dense Deposit Disease	Alexion Pharmaceuticals, Inc.	2018-06-20	PHASE2
NCT04551599	COMPLETED	Healthy	Alexion Pharmaceuticals, Inc.	2020-02-21	PHASE1
NCT04609696	COMPLETED	Healthy	Alexion Pharmaceuticals, Inc.	2020-10-20	PHASE1
NCT04469465	Completed	Paroxysmal Nocturnal Hemoglobinuria	Alexion Pharmaceuticals Inc.	2020-12-16	Phase 3
NCT04451434	COMPLETED	Healthy	Alexion Pharmaceuticals, Inc.	2020-08-17	PHASE1
NCT04451434	Completed	Healthy	Alexion Pharmaceuticals Inc.	2020-08-17	Phase 1
NCT04551599	Completed	Healthy	Alexion Pharmaceuticals Inc.	2020-02-21	Phase 1
NCT03369236	COMPLETED	C3 Glomerulopathy; C3 Glomerulonephritis; Dense Deposit Disease	Alexion Pharmaceuticals, Inc.	2018-06-12	PHASE2
NCT04709094	COMPLETED	Healthy	Alexion Pharmaceuticals, Inc.	2019-07-28	PHASE1
NCT03472885	COMPLETED	Paroxysmal Nocturnal Hemoglobinuria (PNH)	Alexion Pharmaceuticals, Inc.	2018-05-08	PHASE2
NCT03124368	COMPLETED	C3 Glomerulonephritis; Dense Deposit Disease; C3 Glomerulopathy; Immune Complex Mediated Membranoproliferative Glomerulonephritis; Membranoproliferative Glomerulonephritis Types I, II, and III	Alexion Pharmaceuticals, Inc.	2017-08-09	PHASE2
NCT03053102	COMPLETED	Paroxysmal Nocturnal Hemoglobinuria (PNH)	Alexion Pharmaceuticals, Inc.	2017-03-31	PHASE2
NCT05109390	COMPLETED	Healthy	Alexion Pharmaceuticals, Inc.	2018-07-27	PHASE1
NCT05016206	COMPLETED	Healthy	Alexion Pharmaceuticals, Inc.	2018-07-26	PHASE1
NCT03555539	COMPLETED	Hepatic Impairment	Alexion Pharmaceuticals, Inc.	2018-05-01	PHASE1
NCT04935294	COMPLETED	Healthy; Renal Dysfunction	Alexion Pharmaceuticals, Inc.	2018-01-24	PHASE1
NCT03384186	COMPLETED	Healthy	Alexion Pharmaceuticals, Inc.	2017-12-01	PHASE1
NCT04889391	COMPLETED	Healthy	Alexion Pharmaceuticals, Inc.	2017-09-14	PHASE1
NCT03108274	COMPLETED	Healthy	Alexion Pharmaceuticals, Inc.	2017-04-18	PHASE1
NCT04889690	COMPLETED	Healthy	Alexion Pharmaceuticals, Inc.	2016-05-21	PHASE1
NCT04940559	COMPLETED	Healthy	Alexion Pharmaceuticals, Inc.	2016-08-02	PHASE1
NCT04889677	COMPLETED	Healthy	Alexion Pharmaceuticals, Inc.	2016-02-04	PHASE1

(data from https://clinicaltrials.gov, updated on 2025-12-23)

Check the Danicopan product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Danicopan selectively binds to human complement factor D with high affinity, thereby preventing the enzymatic cleavage of factor B and halting the amplification loop of the alternative complement pathway. By blocking downstream complement activation cascade and membrane attack complex assembly, this targeted inhibition prevents complement-mediated erythrocyte lysis, offering therapeutic relevance in conditions driven by complement dysregulation such as paroxysmal nocturnal hemoglobinuria.

Appendix RUO and cGMP Quality Standards

Quality Dimension	RUO (Research Use Only)	cGMP (Current Good Manufacturing Practice)
Clinical Applicability	Prohibited in human clinical trials or medical diagnostics.	Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status	Non-regulated grade; exempt from drug manufacturing laws.	Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment	Unclassified analytical or research laboratories.	Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control	Basic purity and activity testing.	Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation	Basic equipment calibration; no process validation required.	Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance	Vendor self-declared without required formal QMS.	Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact	High risk of IND rejection if used as a critical raw material.	Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).