FDA Grants Orphan Drug Status to Coversin for Rare Blood Disorder

Paroxysmal Nocturnal Hemoglobinuria is an ultra-rare, life-threatening disorder
Paroxysmal Nocturnal Hemoglobinuria is an ultra-rare, life-threatening disorder

The Food and Drug Administration (FDA) has granted Orphan Drug designation to Coversin (Akari Therapeutics) for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH), an ultra-rare, life-threatening and debilitating hematological disorder.

PNH is due to an acquired genetic deficiency which causes the uncontrolled activation of the complement system, thereby attacking and destroying the patient's own blood cells. 

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The Company has investigated Coversin in a Phase 1b trial of healthy volunteers where it was found to inhibit complement C5 activity by 100% within 12 hours. Akari has also demonstrated Coversin's ability to inhibit PNH red blood cell lysis and to achieve full complement inhibition in the blood of eculizumab-resistant patients in vitro. Coversin is currently in a Phase 2 study in patients with PNH.

Coversin is a subcutaneous, second-generation, recombinant small protein inhibitor of complement component-C5. Inhibition of C5 prevents the release C5a and formation of the membrane attack complex (MAC) or C5b-9.

For more information visit Akaritx.com.

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