Alexion’s Soliris Approved for Orphan Drug Status

Two international bodies approved the drug Soliris (eculizumab) for orphan drug status, giving manufacturer Alexion Pharmaceuticals, Inc., of Cheshire, Conn., special consideration on its way through the drug approval process. Orphan drugs are those that most likely wouldn’t be developed because of the rarity of the disease they treat, in this case atypical hemolytic uremic syndrome (aHUS).

The prognosis for aHUS patients is grim. About 70 percent of patients with the most common mutation for aHUS experience chronic renal insufficiency, chronic dialysis, or death within one year of the first clinical episode.

Both the U.S. Food and Drug Administration and the European Commission have approved the orphan status. The intervention by government on behalf of orphan drug development can take a variety of forms, including tax incentives, better patent protection, and financially subsidized clinical research.

Alexion is currently enrolling patients in four clinical studies of Soliris as an investigational treatment for adolescent and adult patients with aHUS. Clinical studies are also currently being planned on the use of Soliris as a treatment for children with aHUS.

If the drug is approved for treatment, the drug’s orphan status would let Alexion market Soliris for 10 years exclusively in Europe and for seven years exclusively in the United States.