Danicopan
Danicopan (also known as ACH-4471) is an experimental drug developed by Achillion Pharmaceuticals for the treatment of Paroxysmal nocturnal hemoglobinuria (PNH). It is a small molecule complement inhibitor that specifically targets factor D of the alternative pathway of the complement system.
Mechanism of Action[edit | edit source]
Danicopan works by inhibiting factor D, a serine protease that is essential for the amplification of the alternative pathway of the complement system. By blocking factor D, Danicopan prevents the formation of the C3 convertase, C3bBb, thereby inhibiting the downstream activation of the complement system and the subsequent lysis of red blood cells.
Clinical Trials[edit | edit source]
Danicopan has undergone Phase 2 clinical trials for the treatment of PNH. The results of these trials have shown that Danicopan, when used in combination with a C5 inhibitor, can effectively control hemolysis, improve anemia, and reduce transfusion dependence in PNH patients.
Potential Applications[edit | edit source]
In addition to PNH, Danicopan may also have potential applications in other diseases that are driven by the alternative pathway of the complement system, such as atypical hemolytic uremic syndrome (aHUS) and C3 glomerulopathy (C3G).
See Also[edit | edit source]
- Paroxysmal nocturnal hemoglobinuria
- Complement system
- Complement inhibitor
- Achillion Pharmaceuticals
References[edit | edit source]
 | This pharmacology-related article is a stub. You can help WikiMD by expanding it. |
Search WikiMD
Ad.Tired of being Overweight? Try W8MD's physician weight loss program.
Semaglutide (Ozempic / Wegovy and Tirzepatide (Mounjaro / Zepbound) available.
Advertise on WikiMD
|
WikiMD's Wellness Encyclopedia |
| Let Food Be Thy Medicine Medicine Thy Food - Hippocrates |
Translate this page: - East Asian
中文,
日本,
한국어,
South Asian
हिन्दी,
தமிழ்,
తెలుగు,
Urdu,
ಕನ್ನಡ,
Southeast Asian
Indonesian,
Vietnamese,
Thai,
မြန်မာဘာသာ,
বাংলা
European
español,
Deutsch,
français,
Greek,
português do Brasil,
polski,
română,
русский,
Nederlands,
norsk,
svenska,
suomi,
Italian
Middle Eastern & African
عربى,
Turkish,
Persian,
Hebrew,
Afrikaans,
isiZulu,
Kiswahili,
Other
Bulgarian,
Hungarian,
Czech,
Swedish,
മലയാളം,
मराठी,
ਪੰਜਾਬੀ,
ગુજરાતી,
Portuguese,
Ukrainian
Medical Disclaimer: WikiMD is not a substitute for professional medical advice. The information on WikiMD is provided as an information resource only, may be incorrect, outdated or misleading, and is not to be used or relied on for any diagnostic or treatment purposes. Please consult your health care provider before making any healthcare decisions or for guidance about a specific medical condition. WikiMD expressly disclaims responsibility, and shall have no liability, for any damages, loss, injury, or liability whatsoever suffered as a result of your reliance on the information contained in this site. By visiting this site you agree to the foregoing terms and conditions, which may from time to time be changed or supplemented by WikiMD. If you do not agree to the foregoing terms and conditions, you should not enter or use this site. See full disclaimer.
Credits:Most images are courtesy of Wikimedia commons, and templates Wikipedia, licensed under CC BY SA or similar.
Contributors: Prab R. Tumpati, MD
