Alexion's Soliris Receives Orphan Drug Status in Japan - Analyst Blog

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Alexion Pharmaceuticals, Inc. ( ALXN ) announced that Japan's Ministry of Health, Labour and Welfare (MHLW) has granted Soliris orphan drug designation for the treatment of patients suffering from myasthenia gravis (MG).

We note that the designation is granted by Japan's MHLW to candidates being developed to treat diseases with a high medical need and affecting less than 50,000 patients in the country. This designation makes Alexion eligible for certain benefits and incentives including priority review for the marketing application of Soliris and marketing exclusivity of 10 years following approval for the MG indication. The opinion of the Pharmaceutical Affairs and Food Sanitation Council forms the basis on which the MHLW awards this special status.

We note that Soliris is currently approved in the U.S., EU, Japan and other countries for the treatment of patients suffering from paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome (aHUS). Last month, the National Institute for Health and Care Excellence recommended Soliris for use within the National Health Service in England as the first and only treatment for patients with aHUS.

We are pleased with the regulatory body granting the orphan drug designation. Soliris enjoys orphan drug designation in both the U.S. and EU for the treatment of MG. Moreover, Soliris was granted orphan drug designation in Japan for the treatment of patients with neuromyelitis optica last month.

Currently, Alexion is enrolling patients in a multinational, placebo-controlled registration REGAIN ( RE fractory G ener A l I zed Myasthe N ia Gravis) study on Soliris in patients with a refractory generalized MG.

Alexion is also developing Soliris for other indications including neuromyelitis optica (phase III) and delayed kidney transplant graft function (phase III) among others. Approval for additional indications would boost Soliris' sales potential.

Alexion carries a Zacks Rank #2 (Buy). Some better-ranked stocks in the health care sector include Biogen Idec Inc. ( BIIB ), ANI Pharmaceuticals, Inc. ( ANIP ) and Amgen Inc. ( AMGN ). All three stocks carry a Zacks Rank #1 (Strong Buy).

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The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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