Novartis sets eyes on newly-acquired Alexion with breakthrough tag for rare disease hopeful iptacopan
For years, Alexion’s rare-disease portfolio that centered on C5 inhibitor Soliris has held its grip on a group of profitable ultra-rare indications. But challengers are looking to take the throne, including Novartis and its mid-stage candidate that has garnered interest from the FDA.
The FDA on Wednesday slapped Novartis’ iptacopan (LNP023) with its breakthrough tag to treat rare blood disorder paroxysmal nocturnal hemoglobinuria (PNH), setting the drug up for a quick review and potential challenge to Alexion’s Soliris/Ultomiris blockbuster franchise.
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