Medicine:Danicopan
Danicopan, sold under the brand name Voydeya, is a medication used for the treatment of paroxysmal nocturnal hemoglobinuria.[1][2] 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.[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,[3] in the United States in March 2024,[1][4] and in the European Union in April 2024.[2] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.[5]
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.[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] The FDA granted the application breakthrough therapy designation.[7]
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).[2][8] The applicant for this medicinal product is Alexion Europe.[2] Danicopan was approved for medical use in the European Union in April 2024.[2][9]
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.[10]
Names
Danicopan is the international nonproprietary name.[11]
References
- ↑ 1.0 1.1 1.2 1.3 "Voydeya- danicopan tablet, film coated; Voydeya- danicopan kit". 3 April 2024. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=ab8d1cbd-a28b-4d49-82ab-5c7460719ac7.
- ↑ 2.0 2.1 2.2 2.3 2.4 2.5 2.6 2.7 2.8 "Voydeya EPAR". 22 February 2024. https://www.ema.europa.eu/en/medicines/human/EPAR/voydeya. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ↑ "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.
- ↑ "Novel Drug Approvals for 2024". 29 April 2024. https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2024.
- ↑ (PDF) New Drug Therapy Approvals 2024 (Report). January 2025. https://www.fda.gov/media/184967/download. Retrieved 21 January 2025.
- ↑ "Danicopan: First Approval". Drugs 84 (5): 613–618. May 2024. doi:10.1007/s40265-024-02023-6. PMID 38528310. https://figshare.com/articles/online_resource/Danicopan_First_Approval/25366471.
- ↑ "CY 2024 CDER Breakthrough Therapy Calendar Year Approvals" (PDF). U.S. Food and Drug Administration (FDA). 30 September 2024. https://www.fda.gov/media/97001/download.
- ↑ "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.
- ↑ "Voydeya PI". 25 April 2024. https://ec.europa.eu/health/documents/community-register/html/h1792.htm.
- ↑ "AstraZeneca's Voydeya recommended by NICE to treat rare blood disorder PNH - PMLiVE" (in en-GB). 25 October 2024. https://pmlive.com/pharma_news/astrazenecas-voydeya-recommended-by-nice-to-treat-rare-blood-disorder-pnh/.
- ↑ "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 81". WHO Drug Information 33 (1). 2019.
Further reading
- "Addition of danicopan to ravulizumab or eculizumab in patients with paroxysmal nocturnal haemoglobinuria and clinically significant extravascular haemolysis (ALPHA): a double-blind, randomised, phase 3 trial". The Lancet. Haematology 10 (12): e955–e965. December 2023. doi:10.1016/S2352-3026(23)00315-0. PMID 38030318.
External links
- "Danicopan (Code C148181)". https://ncit.nci.nih.gov/ncitbrowser/ConceptReport.jsp?dictionary=NCI%20Thesaurus&code=C148181.
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