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Alexion's Ultomiris Gets FDA Approval for New Formulation

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Alexion Pharmaceuticals Inc. announced that the FDA has approved a new advanced formulation of Ultomiris (ravulizumab-cwvz). The FDA approved Ultomiris100 mg/mL formulation for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in adults and atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy for adult and pediatric (one month of age and older) patients.

Shares of the company have gained 15% year to date compared with the industry’s growth of 2.6%.

Ultomiris is the first and only long-acting C5 inhibitor administered every eight weeks in adults. It is approved for the treatment of adult patients with PNH and the treatment of adults and pediatric patients one month of age and older with aHUS.

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

The approval of Ultomiris 100 mg/mL represents reduction of infusion times for patients and a decreased number of vials that need to be stored and prepared for the majority of patient infusions, allowing healthcare providers more time to focus on the patient. The advanced formulation demonstrated comparable safety and efficacy to the original formulation, with the additional benefit of significantly shorter infusion times. With Ultomiris 100 mg/mL, most patients will spend six hours or less each year for receiving the treatment.

Last month, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending marketing authorization for the 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.

Further, Ultomiris is in clinical studies to expand the treated patient population across hematology, nephrology, neurology and for the treatment of severe COVID-19, with seven ongoing phase III programs. The company has planned further clinical trial initiations for 2020.

 

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) , Castle Biosciences, Inc. (CSTL - Free Report) and AVEO Pharmaceuticals Inc. . While Emergent and Castle Biosciences sport a Zacks Rank #1 (Strong Buy), AVEO 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 $4.03 to $6.61 for 2020 and from $5.07 to $8.42 for 2021 over the past 90 days. Shares of the company have increased 106.6% year to date.

Castle Biosciences’ loss per share estimates have narrowed from 51 cents to 11 cents for 2020 over the past 90 days. Shares of the company have increased 183.4% year to date.

AVEO’s loss per share estimates have narrowed from $1.68 to $1.25 for 2020 and from $1.71 to $1.22 for 2021 over the past 60 days. Shares of the company have increased 5.8% year to date.

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