Cheshire, CT, United States

Alexion Pharmaceuticals

www.alexionpharma.com
Cheshire, CT, United States

Alexion Pharmaceuticals is the original developer and distributor of Soliris, a drug used to treat the rare disorders atypical hemolytic uremic syndrome and Paroxysmal nocturnal hemoglobinuria .The company is also involved in research with regards to the immune system and how it can be stopped from attacking healthy tissue .Soliris, the company's sole marketed drug, has been approved for use in the EU and Japan however availability in other places such as the USA and Canada is limited due to marketing restrictions and lack of ensured access .In 2013 36% of sales originated in the US down from 37%, with 33% coming from Europe down from 35%; Japan accounts for just over 10%. In 2013 revenue was impacted by higher unit volumes for Soliris , and a negative price impact related to rebates in Europe; acquisition related costs fell significantly from $22 million to just $5 million. In 2011 50% of the increase in assets came from cash and cash equivalents, the value of which has since gone down since part of it was spent on acquiring Enobia. In the last quarter, R&D spending was higher due to expansion of development programs while net sales of Soliris increased 45% to $227.6 million on account of new international patients with paroxysmal nocturnal hemoglobinuria. By September 2013 quarterly sales of Soliris topped $400 million. Wikipedia.

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Eculizumab, a humanized monoclonal antibody against C5 that inhibits terminal complement activation, showed activity in a preliminary 12-week open-label trial in a small cohort of patients with paroxysmal nocturnal hemoglobinuria (PNH). The present study examined whether chronic eculizumab therapy could reduce intravascular hemolysis, stabilize hemoglobin levels, reduce transfusion requirements, and improve quality of life in a double-blind, randomized, placebo-controlled, multi-center global Phase III trial. It has been found that eculizumab stabilized hemoglobin levels, decreased the need for transfusions, and improved quality of life in PNH patients via reduced intravascular hemolysis. Chronic eculizumab treatment appears to be a safe and effective therapy for PNH.


Patent
Alexion Pharmaceuticals | Date: 2017-08-02

The use of a therapeutic capable of inhibiting complement, e.g., an anti-C5 antibody, to treat antibody mediated neuropathies is disclosed.


Patent
Alexion Pharmaceuticals | Date: 2017-02-10

The present invention provides compositions comprising an isolated mixture of recombinant human NaGlu proteins in which a substantial amount of the NaGlu proteins in the mixture has increased levels of phosphorylated mannose that confer the proteins to be efficiently internalized into human cells. The present invention also provides methods of producing such mixture of NaGlu proteins, vectors used in transgenesis and expression, host cells harboring such vectors, and methods of isolating and purifying the mixture of NaGlu proteins. The invention further provides methods of treating NaGlu associated diseases.


Patent
Alexion Pharmaceuticals | Date: 2017-03-01

This disclosure relates to inexpensive and efficient methods of preparing egg white (e.g., obtained from eggs laid by transgenic chickens) for bulk chromatographic isolation of proteins (e.g., recombinant proteins) from the egg white, as well as methods of filtering acidified egg white and methods of isolating proteins from the egg white.


Eculizumab, a humanized monoclonal antibody against C5 that inhibits terminal complement activation, showed activity in a preliminary 12-week open-label trial in a small cohort of patients with paroxysmal nocturnal hemoglobinuria (PNH). The present study examined whether chronic eculizumab therapy could reduce intravascular hemolysis, stabilize hemoglobin levels, reduce transfusion requirements, and improve quality of life in a double-blind, randomized, placebo-controlled, multi-center global Phase III trial. It has been found that eculizumab stabilized hemoglobin levels, decreased the need for transfusions, and improved quality of life in PNH patients via reduced intravascular hemolysis. Chronic eculizumab treatment appears to be a safe and effective therapy for PNH.


Patent
Alexion Pharmaceuticals | Date: 2017-01-25

The present disclosure relates to, inter alia, compositions containing an inhibitor of human complement and use of the compositions in methods for treating or preventing complement-associated disorders. In some embodiments, the inhibitor is chronically administered to patients. In some embodiments, the inhibitor is administered to a patient in an amount and with a frequency to maintain systemic complement inhibition and prevent breakthrough. In some embodiments, the composition contain an antibody, or antigen-binding thereof, that binds to a human complement component C5 protein or a fragment of the protein such as C5a or C5b.


Patent
Alexion Pharmaceuticals | Date: 2017-08-09

Methods for treating the central nervous system (CNS) in a human patient with mucopolysaccharidosis (MPS) IIIB who has an intact blood brain barrier, comprising intravenous administration of recombinant NaGlu.


Patent
Alexion Pharmaceuticals | Date: 2017-01-27

The present invention provides a novel fusion polypeptide containing a catalytic domain of NPP1 fused to a targeting moiety, nucleic acids encoding the fusion polypeptide, a vector containing the nucleic acid integrated thereinto, a host cell transformed with the vector and pharmaceutical compositions comprising the fusion polypeptide.


Patent
Alexion Pharmaceuticals | Date: 2017-08-16

The present invention provides compositions of recombinant human lysosomal acid lipase having particular glycosylation patterns for internalization into target cells, a vector containing the nucleic acid encoding human lysosomal acid lipase, a host cell transformed with the vector, pharmaceutical compositions comprising the recombinant human lysosomal acid lipase and method of treating conditions associated with lysosomal acid lipase deficiency.


Patent
Alexion Pharmaceuticals | Date: 2016-09-09

The present disclosure relates to, inter alia, a recombinant eculizumab protein or a recombinant eculizumab variant protein having specific glycosylation patterns. The present disclosure relates to, inter alia, a recombinant eculizumab protein or a recombinant eculizumab variant protein made from CHO cells. The present disclosure also relates to methods for the use of these proteins.

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