FDA approves new drug for rare, life-threatening blood disease

The US Food and Drug Administration today approved Ultomiris (ravulizumab) injection for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disease.

The FDA granted this application from Alexion Pharmaceuticals (Nasdaq: ALXN) Priority Review designation, and the decision came nearly two months earlier than expected. Ultomiris also received Orphan drug status, which provides incentives to assist and encourage the development of drugs for rare diseases.

The drug’s sales are expected to reach $1.66 billion by 2022, according to IBES data from Refinitiv. Surprisingly, Alexion’s shares were down 1.87% at $99.33 by late morning. Ultomiris is expected to become the successor in PNH to Alexion's own Soliris (eculizumab).