Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), better known as a leading RNAi therapeutics company, announced that it had started the dosing procedure in hemophilia patients as part of Phase 1 clinical study. As per the reports, the study focuses on evaluating fitusiran’s monthly subcutaneous dose regimen.
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Alnylam Pharmaceuticals has dosed fitusiran to all the patients of hemophilia B with inhibitors and hemophilia A with inhibitors. Fitusiran is one of the leading RNAi therapeutics with primary focus on antithrombin, and can easily be used in the case of rare bleeding disorders as well as hemophilia A and B. It’s designed in such a way that patients experiencing excessive bleeding can benefit instantly as a result of the reduced level of antithrombin and increased fibrin clot formation, which can restore hemostasis, a process that prevents bleeding after any physical injury.
Going by what it claimed in the previous guideline, Alnylam looks to report more data from this study in a couple of months time. It also expects to conduct pivotal studies of fitusiran in the patients (with or without inhibitors) suffering from hemophilia A and B in mid-2016.
The senior management team of Alnylam is delighted to initiate the dosing procedure and hopes that things will continue to turn Alnylam’s way in the future as well. According to Akin Akinc, Ph.D., GM – Fitusiran, Alnylam, about 33% hemophilia patients develop inhibitory antibodies, which further make them refractory to replacement-factor therapy. As a result of this, their clinical course becomes very difficult to manage efficiently. The clinical outcomes of such patients are poorer than others and have long term impacts.
The initial studies suggest that fitusiran can bring positive results. Alnylam will continue to introduce new changes in the trials to ensure the final outcomes can benefit hemophilia patients in the best way possible.