Danicopan

Last updated

Danicopan
Danicopan structure.svg
Clinical data
Trade names Voydeya
Other namesACH-4471
AHFS/Drugs.com Monograph
License data
Pregnancy
category
Routes of
administration 		By mouth
Drug class Complement factor D inhibitor
ATC code
Legal status
Legal status
Identifiers
  • (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
DrugBank
ChemSpider
UNII
KEGG
ChEMBL
ECHA InfoCard 100.398.865 OOjs UI icon edit-ltr-progressive.svg
Chemical and physical data
Formula C6H3BrFN7O3
Molar mass 320.038 g·mol−1
3D model (JSmol)
  • 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=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 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]

Contents

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

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]

References

  1. ↑ "Product and Consumer Medicine Information Licence". TGA eBS. 19 December 2024. Retrieved 19 December 2024.
  2. ↑ "Voydeya danicopan 100 mg film-coated tablets, bottle (416090)". Therapeutic Goods Administration (TGA). 26 November 2024. Retrieved 19 December 2024.
  3. ↑ "Notice: Multiple additions to the Prescription Drug List (PDL) [2024-10-18]". Health Canada . 18 October 2024. Retrieved 25 October 2024.
  4. ↑ "Summary Basis of Decision for Voydeya". Health Canada . 1 September 2012. Retrieved 17 December 2024.
  5. ↑ "Voydeya product information". Health Canada . 7 November 2024. Retrieved 27 December 2024.
  6. 1 2 3 4 5 "Voydeya- danicopan tablet, film coated; Voydeya- danicopan kit". DailyMed. 3 April 2024. Archived from the original on 25 April 2024. Retrieved 25 April 2024.
  7. 1 2 3 4 5 6 7 8 9 10 "Voydeya EPAR". European Medicines Agency. 22 February 2024. Archived from the original on 23 February 2024. Retrieved 24 February 2024. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  8. 1 2 "Voydeya PI". Union Register of medicinal products. 25 April 2024. Retrieved 7 September 2024.
  9. ↑ "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. Archived from the original on 24 February 2024. Retrieved 24 February 2024.
  10. ↑ "Novel Drug Approvals for 2024". U.S. Food and Drug Administration (FDA). 29 April 2024. Archived from the original on 30 April 2024. Retrieved 30 April 2024.
  11. ↑ New Drug Therapy Approvals 2024 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2025. Archived from the original on 21 January 2025. Retrieved 21 January 2025.
  12. ↑ Kang C (May 2024). "Danicopan: First Approval". Drugs. 84 (5): 613–618. doi:10.1007/s40265-024-02023-6. PMID   38528310.
  13. ↑ "CY 2024 CDER Breakthrough Therapy Calendar Year Approvals" (PDF). U.S. Food and Drug Administration (FDA). 30 September 2024.
  14. ↑ "First oral treatment against residual hemolytic anemia in patients with paroxysmal nocturnal hemoglobinuria". European Medicines Agency (EMA) (Press release). 23 February 2024. Archived from the original on 24 February 2024. Retrieved 24 February 2024.
  15. ↑ "AstraZeneca's Voydeya recommended by NICE to treat rare blood disorder PNH - PMLiVE". pmlive.com. 25 October 2024. Retrieved 28 October 2024.
  16. ↑ 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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