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Alexion Pharmaceuticals, Inc. (ALXN - Free Report) announced that new long-term data from an ongoing, open-label extension of the pivotal phase III ARISE study on Kanuma, in children and adults suffering from lysosomal acid lipase deficiency (LAL-D), were presented at the World Congress of Pediatric Gastroenterology, Hepatology and Nutrition.

Data demonstrated that marked and sustained improvements in disease-related lipid and liver abnormalities were observed in patients with LAL-D when treated with Kanuma. In addition, continued treatment with the drug led to a rapid and sustained reduction in alanine aminotransferase (ALT, a marker of liver injury) in 97% of patients, including higher proportion of the same achieving ALT normalization at 52 weeks compared with the initial 20-week period. Moreover, safety profile of Kanuma during the extended open-label period was found to be consistent with that observed in the double-blind period.

Updated data from the ongoing, open-label, phase II/III VITAL study on Kanuma in infants were also presented. Results showed that five out of nine infants, when treated with the drug,  survived beyond three years of age, with the oldest patient now more than five years old. All five patients continue to receive treatment.

Alexion also intends to present data from the first global LAL-D registry. We note that the registry collects long-term, real-world data on the epidemiology of LAL-D, as well as patient outcomes data.

The reported long-term data are encouraging and continue to highlight the benefits of ongoing treatment with Kanuma. Per the company’s press release, 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. It is approved in the U.S., EU and Japan.

Kanuma was launched in the U.S. early in the first quarter of 2016. Meanwhile, the company expects launches outside Germany in the second half of the year with additional launches to follow in 2017. Geographical expansion would boost the drug’s commercial potential.

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 of Kanuma.

Other companies that focus on the development of treatments for rare and ultra-rare diseases include Shire plc (SHPG - Free Report) , BioMarin Pharmaceutical Inc. (BMRN - Free Report) and Ultragenyx Pharmaceutical Inc. (RARE - Free Report) .

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