FDA grants priority review to drug for PNH

• The proportion of patients with breakthrough hemolysis
• The change from baseline in quality of life as assessed via the FACIT-Fatigue Scale
• The proportion of patients avoiding transfusion
• The proportion of patients with stabilized hemoglobin levels.

ALXN1210 had a safety profile that is consistent with that seen for eculizumab, according to Alexion. The most frequently observed AEs were headache and upper respiratory infection. The most frequently observed serious AEs were pyrexia and hemolysis.

There were no treatment-emergent ADAs in the ALXN1210 arm, but one patient in the eculizumab arm did have ADAs. There were no neutralizing antibodies and no cases of meningococcal infection in either arm.