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Alexion's Ultomiris Receives EU Approval for PNH in Adults

Zacks Equity Research

Alexion Pharmaceuticals, Inc. ALXN announced that the European Commission has approved its long-acting C5 complement inhibitor, Ultomiris.

The drug has been approved for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) with hemolysis, with clinical symptoms indicative of high disease activity. The drug is also approved for adult patients who are clinically stable after treatment with Alexion’s lead drug, Soliris, for at least the past six months.

The approval in the European Union was based on comprehensive results from two phase III studies, which included more than 440 patients who had either never been treated with a complement inhibitor or had been stable on Soliris.

Data from the studies show that the efficacy of Ultomiris administered every eight weeks was non-inferior to the efficacy of Soliris administered every two weeks at all 11 primary and secondary endpoints.

Additional data showed that Ultomiris provided immediate and complete C5 inhibition that was sustained for eight weeks between doses. The drug eliminated breakthrough hemolysis associated with incomplete C5 inhibition.

Moreover, patients could easily transition to Ultomiris from Soliris.

The approval will strengthen Alexion’s PNH franchise, as Soliris is already approved for the same. The drug is already approved in the United States for this indication. Initial conversion rates of Soliris patients have been encouraging. Most recently, the drug has been also approved in Japan.

Alexion’s share price has rallied 36.9% year to date compared with the industry’s growth of 7.1%.


The approval of long-acting C5 complement inhibitor, Ultomiris, has boosted growth prospects significantly for the company. Moreover, the company is working to expand the drug’s label. The FDA recently accepted Alexion’s supplemental biologics license application (sBLA) for Ultomiris under priority review for the treatment of patients with typical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA). The regulatory body has set an action date of Oct 19, 2019.

Meanwhile, companies like Achillion Pharmaceuticals Inc.ACHN and Akari Therapeutics, Plc AKTX are also developing drugs to address PNH.

Zacks Rank & Key Pick

Alexion currently carries a Zacks Rank #3 (Hold).

A better-ranked stock in the same space is AMAG Pharmaceuticals, Inc. AMAG, which carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

AMAG’s loss estimates have narrowed to $2.15 from $2.27 for 2019 over the past 60 days.

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