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AstraZeneca's (AZN) Ultomiris Gets EU Nod for Rare Disease in Kids

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AstraZeneca (AZN - Free Report) announced that its rare drug, Ultomiris (ravulizumab), has received approval for expanded use in the European Union (“EU”) for the treatment of paroxysmal nocturnal haemoglobinuria (“PNH”) in children and adolescents. The drug is already approved in the EU for the treatment of PNH in adults.

Ultomiris was added to AstraZeneca’s portfolio with the recent acquisition of Alexion, which closed in July.

The approval for children is based on interim data from phase III study, which evaluated the drug in children and adolescents with PNH. Data from the study demonstrated that Ultomiris achieved complete C5 complement inhibition through the 26-week treatment period. Treatment with Ultomiris also demonstrated consistency, in line with efficacy and the safety profile of the drug for the treatment of adults with PNH.

The stock has rallied 17.7% so far this year in comparison with the industry’s 15.6% rally.

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During the study, Ultomiris was administered intravenously with reduced dosing frequency than Soliris, another C5 inhibitor that was also added to its portfolio on account of Alexion’s buyout. Soliris is also the current standard of care treatment in the EU to treat children and adolescents with PNH.

Please note that PNH is an ultra-rare severe blood disorder that is characterized by the destruction of red blood cells, followed by organ damage and even premature death.

We remind investors that Ultomiris has also been approved in the United States to treat PNH in children aged at least one month as well as adults. The drug has also been approved in both the EU and United States to treat adults and children with atypical haemolyticuraemic syndrome.

In a separate press release, AstraZeneca announced that it has reached a settlement with the European Union under a litigation related to the supply agreement for its COVID-19 vaccine, Vaxzevria. Per the terms of this agreement, the company will supply 60 million doses of the vaccine by the end of third-quarter 2021, 75 million by the end of fourth-quarter 2021 and 65 million by the end of first-quarter 2022.

AstraZeneca had earlier received a court ruling in its favor over the EU in June, as the Court of First Instance in Brussels had ordered the company to deliver 80.2 million doses by Sep 27, 2021, including the 30.2 million doses that were delivered to Europe in first-quarter 2021. The relief was granted to AstraZeneca since it faced difficulties to fulfil its earlier supply delivery to the EU. The earlier supply agreement with the EU required the company to deliver a total 120 million doses by June-end and 300 million vaccine doses cumulatively by September-end.

Zacks Rank & Stocks to Consider

AstraZeneca currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the biotech sector include Moderna (MRNA - Free Report) , Regeneron Pharmaceuticals (REGN - Free Report) and Vertex Pharmaceuticals (VRTX - Free Report) . While both Regeneron and Vertex sport a Zacks Rank #1 (Strong Buy), Moderna currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Moderna’s earnings per share estimates for 2021 have increased from $25.60 to $29.04 in the past 30 days. The same for 2022 has risen from $17.70 to $22.26 over the same period. The stock has rallied 298.9% in the year so far.

Regeneron’s earnings per share estimates for 2021 have increased from $49.96 to $54.15 in the past 30 days. The same for 2022 has risen from $40.91 to $44.11 over the same period. The stock has rallied 40.7% in the year so far.

Vertex’s earnings per share estimates for 2021 have increased from $12.28 to $12.37 in the past 30 days. The same for 2022 has risen from $13.02 to $13.13 over the same period.

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