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Orphan Status for Alexion Drug

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The US Food and Drug Administration (FDA) recently granted Alexion Pharmaceuticals, Inc.’s (ALXN) Soliris an orphan drug designation in the neuromyelitis optica (NMO) indication.

We note that the orphan drug designation is granted in the US only to those drugs that treat a disease affecting less than 200,000 people in the country. Moreover, the orphan drug status for Soliris provides Alexion with seven years of marketing exclusivity (from the day Soliris is cleared by the FDA) for the NMO indication.

Soliris has performed well so far in studies for treating patients suffering from NMO, a neurological disorder. Late last year, Alexion presented encouraging data from a phase II study which evaluated Soliris for treating patients suffering from severe, relapsing NMO. Data from the study revealed a significant decline in the frequency of relapses following treatment with Soliris.

Alexion stated in its press release that half the patients who suffer from relapses in NMO sustain permanent disabilities such as paralysis and blindness, within five years of the onset of the rare disease. Alexion further stated in the release that mostly women are affected by the disease with a 9:1 female to male ratio.

We note that Soliris is available in the US, EU, Japan and many other countries for the treatment of paroxysmal nocturnal hemoglobinuria, a rare genetic blood disorder. The drug is also approved for treating patients suffering from atypical hemolytic uremic syndrome, an ultra-rare genetic disorder.

Alexion is studying Soliris for additional indications, including NMO. Approval of Soliris, Alexion’s sole marketed product, for additional indications would boost the drug’s sales potential.

Alexion currently carries a Zacks Rank #3 (Hold). Companies such as Salix Pharmaceuticals (SLXP), Santarus Inc. (SNTS) and Jazz Pharmaceuticals Public Limited Company (JAZZ) look more attractive with a Zacks Rank #1 (Strong Buy).

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