By Dow Jones Business News,
June 17, 2014, 07:28:00 AM EDT
By Michael Calia
The U.S. Food and Drug Administration has granted orphan-drug status to Alexion Pharmaceuticals Inc.'s ( ALXN ) Soliris
for the treatment of a rare neuromuscular disorder.
Alexion said it is enrolling participants for an international, placebo-controlled trial for patients with the
refractory generalized form of the disorder, myasthenia gravis.
The drug isn't yet approved anywhere for treatment of myasthenia gravis, although it has received approval for the
treatment of other rare disorders.
Myasthenia gravis is a debilitating neuromuscular disorder that leads to weakness in muscles, starting with the eye
and progressing into other, larger muscle groups. It leads to weakness, slurred speech, and difficultly chewing,
swallowing and breathing.
"By specifically inhibiting the terminal complement pathway, which is believed to play a pivotal role in the
pathophysiology of MG, we believe that eculizumab has the potential to help patients living with this devastating rare
disorder," Alexion executive Martin Mackay said in a press release.
The FDA grants orphan status to drugs and other products intended to treat, diagnose or prevent rare diseases and
disorders affecting fewer than 200,000 people in the U.S.
Soliris, also called eculizumab, had already received an FDA orphan-drug designation earlier this year, for the
prevention of delayed graft function in kidney-transplant patients.
Soliris is Alexion's only product on the market. In April, the company said Soliris sales in the first quarter jumped
67% over the prior-year period.
Write to Michael Calia at firstname.lastname@example.org
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