Alexion Submits Applications for Soliris® (Eculizumab) as a Treatment for Patients with Atypical Hemolytic Uremic Syndrome (aHUS) in the United States

Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) announced today that the company has submitted marketing applications to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for Soliris^® (eculizumab) as a treatment for patients with atypical Hemolytic Uremic Syndrome (aHUS).

Both the US and EU filings include the positive data from the two 26-week Phase 2 studies of Soliris as a treatment for adult and adolescent patients with aHUS. Preliminary data from these two studies were presented at the American Society of Nephrology (ASN) annual meeting in November 2010. ^1-3 Primary endpoints in both studies were achieved with statistical significance. aHUS is an ultra-rare, chronic and life-threatening disease in which uncontrolled complement activation causes blood clots in small blood vessels throughout the body (thrombotic microangiopathy, or TMA) leading to kidney failure, stroke, heart attack and death.

“The US and EU regulatory submissions put us one step closer toward accomplishing our goal to transform the lives of patients suffering with aHUS,” said Leonard Bell, M.D., Chief Executive Officer of Alexion. “We recognize that patients with aHUS, a life-threatening and ultra-rare disorder, lack adequate treatment options, and we look forward to working with regulatory authorities as they review our applications.”

Alexion Pharmaceutical's Soliris meets key goals in midstage studies on rare kidney condition

Alexion Pharmaceuticals Inc. said Thursday that Soliris met key goals in two midstage studies focusing on a rare kidney disorder in children.

The company is studying Soliris as a treatment for atypical hemolytic uremic syndrome, a rare disease among children that can cause kidney failure and high blood pressure. The drug is already approved to treat a rare chronic disease called paroxysmal nocturnal hemoglobinuria, which causes a breakdown of red blood cells and leads to anemia.

Alexion said Soliris met its key goals of increasing platelet count and cutting back dialysis treatments. About 60 percent of patients with the condition require dialysis and a kidney transplant, or they could die with a year of diagnosis.

The two midstage studies are ongoing and results will be presented at the American Society of Nephrology's annual meeting held between Nov. 18 and Nov. 21 in Denver.

Shares of Alexion rose $1.29, or 1.9 percent, to $69.36 in early afternoon trading. The stock earlier reached a 52-week high of $71.99 after the company reported upbeat third-quarter financial results and boosted its financial guidance for 2010.

Small Cap Network Issues Analysis of Pharmaceutical Stocks NUVA, ALXN, GTXI, ACOR

Drug makers GTX Inc., Acorda Therapeutics Inc., NuVasive Inc., and Alexion Pharmaceuticals Inc. 

The Small Cap Network, a leading online investment community focused on small cap stocks, has issued an analysis of pharmaceutical stocks GTX Inc.(GTXI), Acorda Therapeutics Inc. (ACOR), NuVasive Inc.(NUVA), and Alexion Pharmaceuticals Inc. (ALXN) 

These drug makers all offer investors an opportunity that doesn't show up in the current income statements alone. Potential shareholders should look at each company's potential and recent product launches that are likely to dramatically change revenues and earnings for these organizations. The article's author Karen Riccio explores those details. 

Investors and traders are invited to subscribe to the free Small Cap Network Newsletter, which offers other trading ideas and insights atDrug makers GTX Inc., Acorda Therapeutics Inc., NuVasive Inc., and Alexion Pharmaceuticals Inc. are Reviewed by SmallCapNetwork.com

The Small Cap Network, a leading online investment community focused on small cap stocks, has issued an analysis of pharmaceutical stocks GTX Inc.(GTXI), Acorda Therapeutics Inc. (ACOR), NuVasive Inc.(NUVA), and Alexion Pharmaceuticals Inc. (ALXN) 

These drug makers all offer investors an opportunity that doesn't show up in the current income statements alone. Potential shareholders should look at each company's potential and recent product launches that are likely to dramatically change revenues and earnings for these organizations. The article's author Karen Riccio explores those details.