The US Food and Drug Administration (FDA) recently granted
Alexion Pharmaceuticals, Inc.
) Soliris an orphan drug designation in the neuromyelitis optica
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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
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
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
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
Jazz Pharmaceuticals Public Limited Company
) look more attractive with a Zacks Rank #1 (Strong Buy).