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Alexion's Ultomiris Gets Approval in Japan for Rare Disease

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Alexion Pharmaceuticals, Inc. (ALXN - Free Report) announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) approved Ultomiris (ravulizumab) for adults and children living with atypical hemolytic uremic syndrome (aHUS). Ultomiris is the first and only long-acting C5 inhibitor for aHUS, reducing the treatment burden for adults and children with administration every other month. Ultomiris is the first and only long-acting C5 inhibitor for aHUS and is administered every other month for adults and children (20 kg or more) and monthly for children (<20 kg).

Ultomiris is approved for the treatment of two ultra-rare diseases—paroxysmal nocturnal hemoglobinuria (PNH) and aHUS.

PNH is a serious ultra-rare blood disorder and aHUS is an ultra-rare disease that can cause progressive injury to vital organs, primarily the kidneys.

The approval inJapan was based on data from two ongoing, global, single-arm, open-label studies of Ultomiris— one in adults and another in children.In the initial 26-week treatment period, 54% of adults and 77.8% (interim data) of children demonstrated Complete TMA Response. Treatment with Ultomiris resulted in normalization of platelet count in 84 percent of adults and 94% of children, normalization of LDH (marker of hemolysis) in 77% of adults and 90% of children and improved kidney function in 59% of adults and 83% (interim data) of children. In the 52-week follow-up period, four additional adult patients and three pediatric patients had a Complete TMA Response that was confirmed after the 26-week Initial Evaluation Period, resulting in an overall Complete TMA Response of 61% in adults and 94% in children (interim data).

Shares of the company have gained 5.8% compared with the industry’s growth of 1.5%.


Last week, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending marketing authorization for a new 100 mg/mL intravenous (IV) advanced formulation of Ultomiris in the European Union. A decision on the same is expected in November 2020.

Alexion plans to submit regulatory filings in the United States and the EU in the third quarter of 2021 for an Ultomiris subcutaneous formulation and device combinations for PNH and aHUS that can be self-administered at home, after completion of the ongoing phase III study and collection of 12-month safety data.

Zacks Rank &Stocks to Consider

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

Some better-ranked stocks in the healthcare sector include Emergent Biosolutions Inc. (EBS - Free Report) , Bio Techne Corp (TECH - Free Report) and Alimera Sciences Inc. (ALIM - Free Report) . While Emergent and Bio Techne sport a Zacks Rank #1 (Strong Buy), Alimera carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Emergent’s earnings estimates have increased from $4.23to $6.58 for 2020 and from $5.55 to $7.62 for 2021 over the past 60 days. Shares of the company have increased 88.3% year to date.

Bio Techne’s earnings estimates have increased from $5.33 to $5.48 for 2020 and from $6.41 to $6.50 for 2021 over the past 60 days. Shares of the company have increased 10.1% year to date.

Alimera’s loss per share estimates have narrowed from $2.58 to $1.31 for 2020 and from $1.25 to 52 cents for 2021 over the past 60 days. Shares of the company have decreased 33.3% year to date.



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