Alexion Pharmaceuticals, Inc. ALXN announced that the European Commission (EC) has approved the new 100 mg/mL intravenous (IV) formulation of Ultomiris (ravulizumab) for the treatment of two ultra-rare diseases — paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).
Per the company, Ultomiris is the first and only long-acting C5 inhibitor administered to patients every eight weeks or every four weeks for pediatric patients less than 20 kg.
This formulation reduces average annual infusion times by approximately 60% compared to Ultomiris 10 mg/mL, while delivering comparable safety and efficacy. Most patients will spend six hours or less a year receiving treatment with Ultomiris 100 mg/ml.
The European Commission’s approval was based on a comprehensive chemistry, manufacturing and control submission and a supplementary clinical data set showing that the safety, pharmacokinetics and immunogenicity following administration of Ultomiris 10 mg/mL and Ultomiris 100 mg/mL were comparable. The data set showed no relevant changes in the efficacy measure of mean lactate dehydrogenase (LDH) levels across the two formulations.
Ultomiris 100 mg/mL was also approved by the FDA in October 2020 and a regulatory filing is under review in Japan. Label expansion of the drug should fuel sales further.
Alexion plans to submit regulatory filings in the United States and the EU in the third quarter of 2021 for the subcutaneous formulation of Ultomiris and the device combination for PNH and aHUS that can be self-administered at home, pending completion of the ongoing phase III study and collection of 12-month safety data.
Alexion’s shares have surged 14.5% in the year so far against the industry’s decline of 0.9%.
We note that Alexion is looking to develop other drugs and reduce dependence on its blockbuster drug, Soliris.
Soliris maintains momentum and the underlying growth of the drug has been robust for all approved indications — PNH, aHUS and refractory generalized myasthenia gravis (gMG).
It has also inked collaborations to diversify its pipeline. Earlier, Alexion obtained an exclusive license from BridgeBio Pharma, Inc.’s subsidiary, Eidos Therapeutics, Inc. EIDX, to develop and commercialize AG10 in Japan. Eidos is currently evaluating AG10 in a phase III study in the United States and Europe for ATTR cardiomyopathy (ATTR-CM) and plans to begin a phase III study in ATTR polyneuropathy (ATTR-PN) shortly.
Alexion currently carries a Zacks Rank #3 (Hold). Two better-ranked stocks in the healthcare space are Vanda Pharmaceuticals Inc. VNDA and Halozyme Therapeutics, Inc. HALO, both carrying a Zacks Rank #2 (Buy), presently. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Vanda’s earnings estimates have been raised 12 cents for 2020 in the past 30 days.
Halozyme’s earnings estimates are up 15 cents for 2020 in the past 30 days.
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