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Alexion Gets FDA Approval for Label Expansion of Ultomiris

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Alexion Pharmaceuticals, Inc. announced that the FDA approved a label expansion of its long-acting C5 complement inhibitor, Ultomiris (ravulizumab-cwvz).

The drug has been approved for the treatment of atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA) for adult and pediatric (aged one month or older) patients.

The FDA approval was based on data from two global, single-arm, open-label studies of Ultomiris — one in adults and the other in children with aHUS. Data showed that at 26 weeks, 54% of adults and 71% of children treated with Ultomiris demonstrated complete TMA response.

The regulatory application for Ultomiris for the indication of aHUS, an ultra-rare disease that can cause progressive injury to vital organs, primarily the kidneys, is under review in the European Union and Japan.

We note that Ultomiris is already approved for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) in the United States. The initial uptake of the drug for this indication has been encouraging.

The label expansion of the drug for additional indications will further boost sales. The drug is also being evaluated for the treatment of generalized myasthenia gravis.

Moreover, Alexion’s blockbuster drug, Soliris, is already approved for the treatment of PNH and aHUS. The company now has a strong portfolio for both indications.

Shares of this company have inched up 1.1% in the year so far against the industry’s decline of 5.8%.

 

Alexion is currently looking to strengthen pipeline with acquisitions and collaborations.  Last week, the company entered a definitive agreement to acquire a clinical-stage biopharmaceutical company, Achillion Pharmaceuticals, Inc. .  The acquisition will add two clinical-stage candidates to Alexion’s pipeline — lead candidate, danicopan (ACH-4471), in phase II for PNH and ACH-5228 in phase I.

The development of other drugs will also reduce the company’s dependence on Soliris. Notably, Amgen (AMGN - Free Report) is developing a biosimilar of Soliris.

Zacks Rank & A Stock to Consider

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

A better-ranked stock in the biotech sector is Vertex Pharmaceuticals Incorporated (VRTX - Free Report) , with a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Vertex’s earnings estimates have increased 38 cents in the past 90 days.

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