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Alexion's sBLA for Ultomiris Gets Priority Review From FDA

Zacks Equity Research

Alexion Pharmaceuticals, Inc. ALXN announced that the FDA has accepted its supplemental biologics license application (sBLA) for its long-acting C5 complement inhibitor, Ultomiris (ravulizumab-cwvz), under a priority review for the treatment of patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA). The regulatory body has set an action date of Oct 19, 2019.

Shares of Alexion were up almost 4.1% following this news on Thursday. In fact, so far this year, the stock has surged 32.3%, outperforming the industry’s rise of 5.8%.

This sBLA was based on data from the phase III study on Ultomiris, conducted in complement inhibitor-naive patients with aHUS, which met its primary objective of complete TMA response. The results were announced this January. The primary endpoint of a complete TMA response was defined by hematologic normalization and improved kidney function. Further, a phase III program of Ultomiris on adolescents and children with aHUS is underway.

Notably, aHUS is a chronic, ultra-rare disease that affects both children and adults, inducing a potentially irreversible damage to kidneys and other vital organs, sudden or progressive kidney failure (requiring dialysis or transplant) and premature death.

Last December, Ultomiris received the FDA approval for treating adult patients with paroxysmal nocturnal hemoglobinuria (PNH), to be administered every eight weeks. This nod came well ahead of its action date set for Feb 18, 2019. Following the same, Ultomiris became the first and the only long-acting C5 complement inhibitor to get an approval for PNH. A phase III study on Ultomiris evaluating children and adolescents with PNH is currently underway.

Alexion has launched Ultomiris in the United States, ahead of schedule. Initial conversion rates of Soliris — Alexion’s key PNH drug — patients have been encouraging. Applications for approval in the EU are currently under review. Earlier this week, Japan’s Ministry of Health, Labour and Welfare (MHLW) approved Ultomiris for the treatment of adult patients with PNH. The approval will strengthen Alexion’s PNH franchise.

Notably, companies like Achillion Pharmaceuticals Inc. ACHN and Akari Therapeutics, Plc AKTX are also developing drugs for addressing PNH.

Meanwhile, Alexion is working to expand Ultomiris’ label. It has initiated a single, PK-based phase III probe on Ultomiris, administered subcutaneously once a week to support registration in PNH and aHUS.

The company also initiated a phase III investigation on the drug for generalized myasthenia gravis (gMG) in the first quarter of 2019 and plans to initiate another phase III study on the same for the neuromyelitis optica spectrum disorder (NMOSD) indication by the end of 2019. Additionally, the company plans to conduct a proof-of-concept analysis on Ultomiris for amyotrophic lateral sclerosis (ALS) and an exploratory clinical study for primary progressive multiple sclerosis (PPMS).

Zacks Rank & Key Pick

Alexion currently carries a Zacks Rank #3 (Hold). A better-ranked stock in the healthcare sector is Acorda Therapeutics, Inc. ACOR, which sports a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Acorda’s loss per share estimates have been narrowed 6.5% for 2019 and 6.9% for 2020 over the past 60 days.

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