1. Ultomiris

Alexion's new Ultomiris has won over 22% of U.S. patients who had been using its predecessor, Soliris, putting the launch well on its way to a 70% conversion goal by the end of 2020. (Alexion)

Generic name: ravulizumab-cwvz
Company: Alexion
Disease: paroxysmal nocturnal hemoglobinuria
2024 sales estimate: $3.48 billion 

Alexion has big goals for Ultomiris, the follow-up to its rare-disease standout Soliris. And so far, the company is delivering.

As of April 22, the company had converted 22% of U.S. patients with paroxysmal nocturnal hemoglobinuria (PNH) over to the newer drug, commercial chief Brian Goff said on Alexion’s first-quarter 2019 conference call. That figure marked a significant leap from the 14% executives had quoted a month prior and the 3% recorded in early February.

RELATED: Alexion gets off to the races with key Ultomiris, Soliris launches

It also puts Alexion well on the way to hitting its 70% conversion target by the end of 2020, the deadline it set for reaching that marker. And the way SVB Leerink analyst Geoffrey Porges sees it, the strong start suggests the conversion goal “should be achieved well before” the deadline, he wrote in a note to clients.

One factor that’s helped the switch? Payers have jumped on board, Goff said, erecting “minimal barriers for conversion.” Sixty percent of commercial payers have defined policies for covering Ultomiris, he said during the late April conference call, and Alexion will be looking to mirror that success in Europe and Japan.

RELATED: FDA gifts Alexion quick OK for Soliris follow-up Ultomiris

Alexion’s pricing strategy has no doubt helped win payers' favor. The biotech rolled out Ultomiris at a 10% discount to its notoriously pricey predecessor, whose maintenance doses bear list prices of about $508,000 in PNH, Porges said. And those discounts could grow in other indications if Ultomiris can make its way into Soliris’ other markets.

Ultomiris’ more convenient dosing regimen has also helped woo current Soliris patients, who currently need follow-up doses every two weeks. Ultomiris requires maintenance doses just eight weeks apart.

1. Ultomiris