Alexion Gets CHMP Nod for New Ultomiris Formulation


The Committee for Medicinal Products for Human Use (CHMP) recommendation for ravulizumab (Ultomiris) is for a dosage that involves a less rigorous schedule for administration.

In what may help Alexion to distance itself from eculizumab (Soliris) biosimilar competition, the company has received a positive opinion from Europe’s Committee for Medicinal Products for Human Use (CHMP) for a new formulation of follow-on product Ultomiris (ravulizumab).

Soliris brought Alexion sales of $975.5 million for the second quarter of 2020 vs $980.8 million in the comparable 2019 quarter. Ultomiris netted $251.1 million vs $54.2 million for the respective quarters.

US biosimilar competition for Soliris is looming, aided by a May 2020 court settlement between Alexion and Amgen in which Amgen got the license to start making and distributing an eculizumab biosimilar in the United States beginning March 1, 2025. That bought Alexion time to continue its attempt to transition patients to Ultomiris.

Ultomiris received FDA approval in 2018 for treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) and in 2019 for atypical hemolytic uremic syndrome (aHUS). The European Commission (EC) approved Ultomiris for PNH in 2019 and for aHUS in June 2020. Biosimilars for eculizumab in Europe have been predicted to appear as soon as this year.

The just-announced positive opinion from CHMP paves the way for EC approval of ravulizumab in a new 100-mg/mL intravenous formulation, which Alexion said “would constitute an advancement in the treatment experience for patients with aHUS and PNH by reducing average annual infusion times by approximately 60% compared to Ultomiris 10 mg/mL while delivering comparable safety and efficacy.”

The drug achieves this reduction because it is a long-acting monoclonal anti-C5 antibody. “Importantly, it would mean that most patients would spend only 6 hours or less a year receiving treatment, allowing them to focus on other aspects of their lives,” said John Orloff, MD, executive vice president and head of research and development at Alexion.

PNH is a blood disorder characterized by complement-mediated destruction of red blood cells that can lead to thrombosis, which can cause organ damage and premature death. aHUS is a rare blood disease that causes excessive blood clots to form in blood vessels, leading to progressive injury to vital organs, primarily the kidneys.

Soliris vs Biosimilars

A new Ultomiris formulation coupled with Alexion’s claimed success in switching US patients with PNH from Soliris to Ultomiris presents challenges for eculizumab biosimilar developers. Amgen is developing the biosimilar eculizumab, ABP 959. This biosimilar candidate is currently undergoing a phase 3 randomized study comparing it with Soliris in adults with PNH. Moscow-based Generium last year received Russian approval for an eculizumab biosimilar. Epirus Biopharmaceuticals also has an eculizumab biosimilar in development: BOW080.

In January 2020, Samsung Bioepis announced it would begin a phase 3 trial to assess the clinical safety and efficacy of an eculizumab biosimilar vs Soliris.

Although biosimilars may offer cost savings vs Soliris, Ultomiris is already slightly less costly than Soliris: $6404 per vial vs $6543, according to Pharmaceutical Technology, which in a recent analysis discussed factors that might entice or prevent patients from switching from Soliris to Ultomiris. The discount needs to be sufficient to prompt that move. First-line or second-line placement for eculizumab vs ravulizumab will also play a role, the article stated.

Ultomiris’ 8-week administration schedule may be more attractive to patients than Soliris’ 2-week administration schedule, which would likely be the same schedule for eculizumab biosimilars. Ultomiris also has an ancillary cost-saving advantage as patients will need to visit clinics less.

Additionally, Alexion is working on clinical trials to establish both Soliris and Ultomiris as treatments for coronavirus disease 2019.

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