Danicopan

Danicopan, sold under the brand name Voydeya, is a medication used for the treatment of paroxysmal nocturnal hemoglobinuria.^[6][7] It is a complement inhibitor which reversibly binds to factor D to prevent alternative pathway-mediated hemolysis and deposition of complement C3 proteins on red blood cells.^[7]

Danicopan

Clinical data
Trade names	Voydeya
Other names	ACH-4471
AHFS/Drugs.com	Monograph
License data	US DailyMed: Danicopan
Pregnancy category	AU: B3[1]
Routes of administration	By mouth
Drug class	Complement factor D inhibitor
ATC code	L04AJ09 (WHO)
Legal status
Legal status	AU: S4 (Prescription only)[2] CA: ℞-only[3][4][5] US: ℞-only[6] EU: Rx-only[7][8] In general: ℞ (Prescription only)
Identifiers
IUPAC name (2S,4R)-1-{2-[3-acetyl-5-(2-methylpyrimidin-5-yl)-1H-indazol-1-yl]acetyl}-N-(6-bromopyridin-2-yl)-4-fluoropyrrolidine-2-carboxamide
CAS Number	1903768-17-1
DrugBank	DB15401
ChemSpider	75531295
UNII	JM8C1SFX0U
KEGG	D11641
ChEMBL	ChEMBL4250860
ECHA InfoCard	100.398.865
Chemical and physical data
Formula	C6H3BrFN7O3
Molar mass	320.038 g·mol−1
3D model (JSmol)	Interactive image
SMILES CC(=O)C1=NN(CC(=O)N2C[C@H](F)C[C@H]2C(=O)NC2=CC=CC(Br)=N2)C2=C1C=C(C=C2)C1=CN=C(C)N=C1
InChI InChI=1S/C26H23BrFN7O3/c1-14(36)25-19-8-16(17-10-29-15(2)30-11-17)6-7-20(19)35(33-25)13-24(37)34-12-18(28)9-21(34)26(38)32-23-5-3-4-22(27)31-23/h3-8,10-11,18,21H,9,12-13H2,1-2H3,(H,31,32,38)/t18-,21+/m1/s1 Key:PIBARDGJJAGJAJ-NQIIRXRSSA-N

The most common side effects include fever, headache, increased levels of liver enzymes (a sign of possible liver problems) and pain in the extremities (arms and legs).^[7]

Danicopan was approved for medical use in Japan in January 2024,^[9] in the United States in March 2024,^[6][10] and in the European Union in April 2024.^[7] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.^[11]

Medical uses

Danicopan is indicated as add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria.^[6][7]

Society and culture

Legal status

Danicopan was approved for medical use in Japan,^[12] the United States,^[6] and the European Union in 2024.^[7] The FDA granted the application breakthrough therapy designation.^[13]

In February 2024, the Committee for Medicinal Products for Human Use of the EMA adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Voydeya, intended as add-on therapy to ravulizumab or eculizumab for the treatment of residual hemolytic anemia in adults with paroxysmal nocturnal hemoglobinuria (PNH).^[7][14] The applicant for this medicinal product is Alexion Europe.^[7] Danicopan was approved for medical use in the European Union in April 2024.^[7][8]

In October 2024, the National Institute for Health and Care Excellence (NICE) adopted a recommendation for danicopan as an add-on therapy for adults with the paroxysmal nocturnal haemoglobinuria (PNH) based results from the phase 3 ALPHA trial, which evaluated the efficacy and safety of the drug as an add-on to ravulizumab or eculizumab in patients with PNH who experienced clinically significant extravascular haemolysis.^[15]

Names

Danicopan is the international nonproprietary name.^[16]