Danicopan

Danicopan
Clinical data
Trade names	Voydeya
Other names	ACH-4471
AHFS/Drugs	Monograph
License data	USDailyMed:Danicopan;
Routes of; administration	By mouth
Drug class	Complement factor Dinhibitor
ATC code	L04AJ09(WHO);
Legal status
Legal status	US:℞-only; EU:Rx-only; 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	320.038g·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;

Danicopan,sold under the brand nameVoydeya,is a medication used for the treatment ofparoxysmal nocturnal hemoglobinuria.^[1]^[2]It is acomplementinhibitor which reversibly binds to factor D to prevent alternative pathway-mediatedhemolysisand deposition ofcomplement C3proteins on red blood cells.^[2]

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).^[2]

Danicopan was approved for medical use in Japan in January 2024,^[4]in the United States in March 2024,^[1]^[5]and in the European Union in April 2024.^[2]

Medical uses

Danicopan isindicatedas add-on therapy toravulizumaboreculizumabfor the treatment of extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria.^[1]^[2]

Society and culture

Legal status

Danicopan was approved for medical use in Japan,^[6]the United States,^[1]and the European Union in 2024.^[2]

In February 2024, theCommittee for Medicinal Products for Human Useof 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).^[2]^[7]The applicant for this medicinal product is Alexion Europe.^[2]Danicopan was approved for medical use in the European Union in April 2024.^[2]^[3]

Names

Danicopan is theinternational nonproprietary name.^[8]

References

^^a ^b ^c ^d ^e"Voydeya- danicopan tablet, film coated; Voydeya- danicopan kit".DailyMed.3 April 2024.Archivedfrom the original on 25 April 2024.Retrieved25 April2024.
^^a ^b ^c ^d ^e ^f ^g ^h ⁱ ^j"Voydeya EPAR".European Medicines Agency.22 February 2024.Archivedfrom the original on 23 February 2024.Retrieved24 February2024.Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
^^a ^b"Voydeya PI".Union Register of medicinal products.25 April 2024.Retrieved7 September2024.
^"Voydeya (danicopan) granted first-ever regulatory approval in Japan for adults with PNH to be used in combination with C5 inhibitor therapy".AstraZeneca(Press release). 19 January 2024.Archivedfrom the original on 24 February 2024.Retrieved24 February2024.
^"Novel Drug Approvals for 2024".U.S.Food and Drug Administration(FDA).29 April 2024.Archivedfrom the original on 30 April 2024.Retrieved30 April2024.
^Kang C (May 2024). "Danicopan: First Approval".Drugs.84(5): 613–618.doi:10.1007/s40265-024-02023-6.PMID 38528310.
^"First oral treatment against residual hemolytic anemia in patients with paroxysmal nocturnal hemoglobinuria".European Medicines Agency(EMA)(Press release). 23 February 2024.Archivedfrom the original on 24 February 2024.Retrieved24 February2024.
^World Health Organization(2019). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 81".WHO Drug Information.33(1).hdl:10665/330896.

External links

"Danicopan (Code C148181)".NCI Thesaurus.

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[Voydeya_FDA_label-1] "Voydeya- danicopan tablet, film coated; Voydeya- danicopan kit".DailyMed.3 April 2024.Archivedfrom the original on 25 April 2024.Retrieved25 April2024.

[Voydeya_EPAR-2] ^^a ^b ^c ^d ^e ^f ^g ^h ⁱ ^j"Voydeya EPAR".European Medicines Agency.22 February 2024.Archivedfrom the original on 23 February 2024.Retrieved24 February2024.Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.

[Voydeya_PI-3] "Voydeya PI".Union Register of medicinal products.25 April 2024.Retrieved7 September2024.

[4] "Voydeya (danicopan) granted first-ever regulatory approval in Japan for adults with PNH to be used in combination with C5 inhibitor therapy".AstraZeneca(Press release). 19 January 2024.Archivedfrom the original on 24 February 2024.Retrieved24 February2024.

[5] "Novel Drug Approvals for 2024".U.S.Food and Drug Administration(FDA).29 April 2024.Archivedfrom the original on 30 April 2024.Retrieved30 April2024.

[6] Kang C (May 2024). "Danicopan: First Approval".Drugs.84(5): 613–618.doi:10.1007/s40265-024-02023-6.PMID 38528310.

[7] "First oral treatment against residual hemolytic anemia in patients with paroxysmal nocturnal hemoglobinuria".European Medicines Agency(EMA)(Press release). 23 February 2024.Archivedfrom the original on 24 February 2024.Retrieved24 February2024.

[8] World Health Organization(2019). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 81".WHO Drug Information.33(1).hdl:10665/330896.

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