In its move from an ultrarare disease company to one focusing on less-rare diseases, Alexion is attempting a branding and pricing makeover. But the drugmaker still wants to back up blockbuster Soliris, and it needs a few ultrarare indications to do so.
Alexion’s successor drug Ultomiris has snagged a priority review from the FDA to treat atypical hemolytic uremic syndrome (aHUS), an ultrarare disease that can cause progressive damage to vital organs, the drugmaker said Thursday. The FDA’s approval decision date is Oct. 19.
“This acceptance is an important step in our efforts to deliver a potential new standard of care to people living with this devastating disease,” John Orloff, Alexion’s R&D head, said in a statement.
An aHUS label expansion for Ultomiris, a C5 complement inhibitor previously approved in December to treat adult paroxysmal nocturnal hemoglobinuria (PNH), will act as a follow up to Alexion’s pricey Soliris, which was approved in that indication in 2014. Soliris—far and away Alexion’s bestselling therapy—is also approved in PNH and generalized myasthenia gravis.
Alexion is in the middle of a shift from a boutique ultrarare disease drugmaker into one that treats more common ailments, and, in the process, it's hoping to shed its reputation for marketing ultraexpensive therapies. In January, Alexion CEO Ludwig Hantson highlighted a 10% discount on maintenance doses of Ultomiris as a sign of the company’s shift away from a superhigh-price drug strategy.
“This is about a story of innovation and volume,” Hantson said at the J.P. Morgan Healthcare Conference in San Francisco in January. “It is not going to be a story about price and price increases and so on.”
In addition to Soliris and Ultomiris, Alexion also has prospects in amytrophic lateral sclerosis and primary progressive multiple sclerosis currently in the pipeline.
But Alexion has reasons to bring more doctors and patients on board to Ultomiris beyond its reputation makeover. The drug, positioned as Soliris’ successor, could be the face of Alexion’s future with promising PNH switching data under its belt.
As of April 22, 22% of Soliris patients had switched over from the older drug, Alexion said, with the vast majority of new PNH patients starting on Ultomiris. Those results are a positive sign that Ultomiris—which cleared just $24.6 million in first-quarter sales—will hit its 70% conversion target within the next two years. By contrast, Soliris raked in $962 million in global sales in the first quarter.