Alexion snares OK for quicker-infusing Ultomiris formula—and a subQ version is on the horizon

Alexion is also working on a subcutaneous version of Ultomiris, which it plans to file for approval by 2021's third quarter. (Alexion Pharmaceuticals)

Alexion Pharmaceuticals has wasted no time converting patients to its next-generation rare disease med Ultomiris. Now, it's looking to make the switch to its Soliris successor a little easier, thanks to a new and improved version that will replace the old by the middle of next year. 

The FDA on Monday cleared a higher-concentration formulation of Ultomiris to treat the rare blood disease paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS)—an ultra-rare disease that can damage vital organs, Alexion said in a release.

The new formulation is expected to slash infusion times by about 60% compared to the original 10 mg/ml dose, cutting treatments from about two hours to 45 minutes or less. That translates to less than six hours in the infusion chair per year, executive vice president and head of research and development at Alexion, John Orloff, M.D., said in an interview.

Shipments of the new formulation have already gone out and patients should have access in a matter of days, he said. Ultomiris 10 mg/ml will stick around until mid-2021 while patients switch over. After that, Alexion plans to pull the older version from shelves.

While the launch kicks off in the U.S., Alexion has filings for the new formulation under review in the EU and Japan. Armed with an endorsement from the Committee for Medicinal Products for Human Use (CHMP), Alexion expects a decision from the EU in November.

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Since Ultomiris’ first green light in late 2018—initially in the blood disease PNH, followed by an aHUS nod less than a year later—Alexion has campaigned to switch patients over from its rare disease standout Soliris, set to face U.S. biosimilar competition in the second half of the decade.

Ultomiris, dosed every eight weeks, is already more convenient than the biweekly Soliris, and that's helped the new med first gain on, and then surpass, its predecessor, at least in the PNH field. Alexion has already achieved its goal of converting 70% of PNH patients to Ultomiris from Soliris by year-end, Orloff said.

The drug is nearing those numbers in aHUS, too—and the new formulation should make the switch even more attractive, he said.

Ultomiris raked in $389.3 million globally in 2019 compared with Soliris' $3.94 billion, according to the company’s annual report (PDF).

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Elsewhere, the company is pitting Ultomiris against a slate of diseases like neuromyelitis optica spectrum disorder and the neurodegenerative disease amyotrophic lateral sclerosis, with phase 3 trials for both underway. More recently, Alexion has looked to test the drug in COVID-19.

Those trials will continue with the 10 mg/ml dose for now, though the company hopes to eventually swap in the new formulation, Orloff said.

But Alexion isn’t content to simply speed up Ultomiris infusions; the rare disease drugmaker is also working on a subcutaneous version that patients can take at home. If all goes to plan, Alexion will file the Ultomiris shot for approval by 2021’s third quarter.

It’s important to offer patients dosing options, Orloff said. Plus, the company now wields an approval that shows it can deliver the drug at high concentrations safely and effectively.