Pricey Soliris performs in another rare disease

Alexion's ($ALXN) Soliris is arguably the most expensive drug in the world. The rare-disease therapy fights two life-threatening diseases--and it costs up to $400,000 per year, depending upon the patient. And if the company has its way, the drug may be targeted at another group of patients.

In a Phase II study, Soliris reduced the frequency of attacks in patients with relapsing neuromyelitis optica, a serious neurological disorder that worsens with each relapse. The 14-patient trial met that primary goal "with high degrees of clinical and statistical significance," Alexion said in a statement.

Patients with NMO are progresively disabled by each attack. Inflammation of the spinal cord and eye nerves can lead to paralysis, blindness and death, study investigator Sean J. Pittock of the Mayo Clinic said. One report found that 30% of NMO patients died wihtin 5 years of developing the disease.

"If we can completely stop such attacks in NMO, we can prevent disability and provide patients the opportunity for significantly improved outcomes," said Dr. Pittock, a neurology professor and co-director of Mayo's Neuroimmunology Laboratory. "In this study, [Soliris] was associated with a substantial reduction in the number of attacks," including in patients who were resistant to several other drugs.

Soliris is now approved to treat a rare kidney disorder, atypical hemolytic uremic syndrome, and the blood disorder paroxysmal nocturnal hemoglobinuria.

- read the release from Alexion
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