Alexion Pharmaceuticals, Inc. (ALXN - Free Report) revealed new long-term data from an ongoing, open-label extension of the pivotal phase III ARISE study on Kanuma (sebelipase alfa) in children and adults with lysosomal acid lipase deficiency (LAL-D). Data was presented at the annual meeting of the American Association for the Study of Liver Diseases.
Data demonstrated that two-third of the patients treated with Kanuma for 52 weeks experienced a reduction in the liver fibrosis stage from baseline while half of them achieved at least a two-stage reduction, including patients who had fibrosis and cirrhosis at baseline. Moreover, the reduction of the liver fibrosis stage was accompanied by sustained improvements in alanine aminotransferase (ALT), LDL cholesterol and liver fat content in the Kanuma arm.
Long-term data from the ARISE study also demonstrated rapid and consistent improvement in important markers of liver injury and lipid abnormalities in children and adults with LAL-D, when treated with Kanuma. In 76 weeks of treatment with Kanuma, nearly all patients (98%) witnessed a sustained reduction in ALT levels, with a mean reduction from baseline of 56%.
The new data is quite encouraging as it underscores the benefits of treatment with Kanuma. According to the company, LAL-D is a genetic, chronic and progressive ultra-rare metabolic disease that can lead to life-threatening consequences, including cirrhosis and severe dyslipidemia.
We note that Kanuma is the first and only approved treatment for patients with LAL-D. The company has received approval for the drug in the U.S., the EU and Japan. It was launched in the U.S. in early first-quarter 2016.
We remind investors that Kanuma became part of Alexion’s portfolio following the Jun 2015 acquisition of Synageva BioPharma. We expect investor focus to remain on the commercialization and sales ramp-up efforts for the drug.
Alexion currently has a Zacks Rank #5 (Strong Sell)
ALEXION PHARMA Price
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