Alexion Pharmaceuticals, Inc. announced that the FDA has approved Ultomiris (ravulizumab-cwvz/ALXN1210) for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH), well ahead of its action date set for Feb 18, 2019. PNH, a rarely acquired disorder, causes rupture or destruction of red blood cells called hemolysis and can induce a wide range of debilitating symptoms and complications including blood clotting.
Ultomiris is the first and the only long-acting C5 complement inhibitor to get an approval for the above indication. It is to be administered every eight weeks. The nod for Ultomiris is quite compelling for patients with PNH as they no longer have to go for twice-a-week infusions but can now settle for just six to seven infusions a year.
Share price of Alexion has decreased 20.9% year to date, narrower than the industry’s decline of 28.6%.
The FDA approval was supported by results from two phase III studies, which included 441 patients, who had either never been treated with a complement inhibitor before or who had been stable on Soliris (eculizumab). The studies showed that the efficacy of Ultomiris, administered every eight weeks, was non-inferior to the efficacy of Alexion’s key PNH drug Soliris (administered every two weeks on all 11 endpoints). The safety profile of Ultomiris was similar to that of Soliris.
The application for Ultomiris in adults with PNH is currently under review in Europe and Japan. In September 2018, Ultomiris was granted an Orphan Drug Designation in Japan. Further, a phase III study of Ultomiris on children and adolescents suffering PNH is currently ongoing.
The company is also conducting studies on Ultomiris for other indications. It has completed enrolment for a phase III study of ALXN1210 administered intravenously every eight weeks in complement inhibitor treatment-naïve adolescent and adult patients with atypical hemolytic uremic syndrome (aHUS). The company expects to report data from this study in early 2019. It is also assumed to file for a regulatory approval of ALXN1210 regarding aHUS now that it has received a nod for Ultomiris with respect to PNH. A phase III study of ALXN1210 on children afflicted with aHUS is currently underway.
Alexion is also conducting a phase I study of subcutaneous ALXN1210, co-administered with Halozyme Therapeutics, Inc.’s (HALO - Free Report) ENHANZE drug-delivery technology, PH20. Pending co-formulation data, this next-generation subcutaneous formulation will be called ALXN1810 and has potential to further extend the dosing interval to once every two weeks or once a month.
Zacks Rank and Other Stocks to Consider
Alexion sports a Zacks Rank #1 (Strong Buy). Some other top-ranked stocks in the same space are Anika Therapeutics Ltd. (ANIK - Free Report) and Gilead Sciences Inc. (GILD - Free Report) , both sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Anika’s earnings per share estimates have increased from $1.01 to $1.11 for 2018 and $1.50 to $1.60 for 2019 over the past 60 days. The company delivered a positive surprise in two of the trailing four reported quarters, the average beat being 31.76%.
Gilead’s earnings per share estimates have been raised from $6.71 to $6.93 for 2018 and $6.64 to $6.84 for 2019 over the past 60 days. The company pulled off an earnings surprise in three of the last four reported quarters, the average beat being 6.99%.
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