One win, one delay for Alexion's lifecycle management

Alexion’s creeping domination of complement inhibition took another step forward today with EU approval for Ultomiris in haemolytic uremic syndrome. This is the second EU use for the group’s Soliris follow-on, which already has a label for paroxysmal nocturnal haemoglobinuria (PNH). Still, investors are naturally more focused on the US, where a franchise-extension plan suffered a quiet delay last week. This concerned Ultomiris’s subcutaneous version, which Alexion said met the primary goal of a phase III study, demonstrating pharmacokinetic non-inferiority versus the approved IV form in 136 PNH subjects previously given Soliris. However, Alexion revealed that a US filing for SC Ultomiris would now not be made until the third quarter of 2021, after the regulator said it wanted 12-month data from an extension trial. This would result in approval in mid-2022, versus an earlier 2021 timeline. While IV delivery is seen as less patient-friendly than SC, a further complication is that Ultomiris in the latter form is given weekly, while the former is injected only once every eight weeks. A separate angle to the Ultomiris story is a phase III trial in Covid-19, measuring 29-day survival, based on anecdotal reports of complement involvement in acute lung injury.