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View Full Version : ACH-4471 in trials for PNH treatment


Marrowforums
Thu Mar 29, 2018, 02:23 PM
Achillion Pharmaceuticals (http://www.achillion.com) is sponsoring a tiny Phase II trial (https://clinicaltrials.gov/ct2/show/NCT03053102) of ACH-4471 (http://www.achillion.com/pipeline/factor-d-inhibitors/complement-mediated-diseases-i-ach-4471/ach-4471/), a new drug for the treatment of PNH.

ACH-4471 inhibits a specific molecule (http://www.bloodjournal.org/content/126/23/275) that is part of the complement process, and may reduce hemolysis.

ACH-4471 was recently approved in Europe (http://ir.achillion.com/releasedetail.cfm?releaseid=1058593) for treatment of C3 glomerulopathy.