The Biologics License Application (BLA) for ALXN1210, an investigational treatment for paroxysmal nocturnal hemoglobinuria (PNH), has been accepted for priority review by the Food and Drug Administration (FDA). 

ALXN1210 works by inhibiting the C5 complement protein in the terminal complement cascade, which when overactivated, can lead to the development of disorders such as PNH. The Phase 3 clinical development program of ALXN1210 included both complement inhibitor treatment-naïve patients (N=246) as well as those who had been stable on Soliris (eculizumab), another complement inhibitor, and then switched to ALXN1210 (N=195); in both studies, treatment with ALXN1210 every 8 weeks was found to be non-inferior to Soliris every 2 weeks. 

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“We are working with the FDA to facilitate a smooth review,” said John Orloff, MD, EVP and Head of R&D at Alexion. “We are on track with our efforts to establish ALXN1210 as the new standard of care for patients with PNH.”

ALXN1210 is also currently being evaluated in a Phase 3 study in complement inhibitor-naïve patients with atypical hemolytic uremic syndrome. 

A Prescription Drug User Fee Act (PDUFA) target date of February 18, 2019 has been set by the FDA for the PNH indication.

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