Advertise With UsAdvertise on our extensive network of industry websites and newsletters.
Get the PBR newsletterSign up to our free email to get all the latest PBR
news.A clinical study by the Connecticut-based drug development company Alexion has shown that its investigational drug eculizumab can reduce the damage to red blood cells produced by a rare disease of the blood.
Subscribe to our email newsletter
The study shows that patients with a rare and potentially deadly blood disease, paroxysmal nocturnal hemoglobinuria (PNH), who received eculizumab maintained significant reductions in red blood cell destruction and blood transfusions.
Eculizumab has been granted orphan drug status in both the US and Europe for the treatment of PNH. If approved, eculizumab would represent the first approved drug from a new class of anti-inflammatory therapeutics-terminal complement inhibitors, as well as being the first drug available specifically for patients suffering from this disease.
Following the initial 12-week pilot trial, all 11 patients chose to participate in a one-year extension study for a total of 64 weeks of treatment. A 900mg dose of eculizumab was administered approximately every two weeks, and was shown to be sufficient to consistently block terminal complement activity in all patients.
A dramatic reduction in hemolysis was maintained throughout the study and a decrease in levels of lactate dehydrogenase, a biochemical marker of red blood cell lysis, was also observed. The proportion of PNH type III red blood cells increased from 36.7% at baseline to 58.4%. Patients reported improvements in their quality of life and generally found the drug to be well tolerated.
Alexion is currently undertaking phase III trials of the drug, which, if successful, will form the basis of a submission for further regulatory approval.