Drug maker Alexion, developer of the rare disease drug eculizumab (Soliris), announced Friday that the FDA has accepted for priority review its long-acting C5 complement inhibitor, ravulizumab (Ultomiris), which offers less frequent administration than eculizumab.
Drug maker Alexion, developer of the rare disease drug eculizumab (Soliris), announced Friday that the FDA has accepted for priority review its long-acting C5 complement inhibitor, ravulizumab (Ultomiris), which offers less frequent administration than eculizumab. The FDA will review ravulizumab for the treatment of atypical hemolytic uremic syndrome (aHUS), a progressive disease that can lead to irreversible organ damage and premature death.
Alexion expects a regulatory decision by October 2019.
“This acceptance is an important step in our efforts to deliver a potential new standard of care to people living with this devastating disease,” said John Orloff, MD, executive vice president and head of research and development at Alexion. “We look forward to working closely with the FDA to facilitate a rapid review of this application.”
Earlier this year, Alexion announced that its study in 56 patients with aHUS had met its primary end point; in the first 26 weeks of treatment, 53.6% of patients (95% CI, 39.6%-67.5%) demonstrated a complete thrombotic microangiopathy response, defined by hematologic normalization and improved kidney function for at least 28 consecutive days.
Ravulizumab, which is administered every 8 weeks versus every 2 weeks for eculizumab, has already gained approval for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), and Alexion is studying the drug in patient with myasthenia gravis (MG).
Alexion noted in its fourth quarter 2018 earnings call that it hopes to transition patients already receiving the older eculizumab (which carries indications in aHUS, PNH, and MG) onto the newer ravulizumab in the near future. Alexion’s chief commercial officer, Bryan Goff, said that the company is hoping for a 70% conversion from eculizumab to ravulizumab within the next 2 years ahead of biosimilar market entry.
Meanwhile, biosimilars of eculizumab are advancing; last week, drug maker Amgen reported data demonstrating pharmacokinetic and pharmacodynamic equivalence of its proposed biosimilar (ABP 959) and the reference eculizumab, and the biosimilar developer is currently recruiting for its phase 3 clinical trial of the biosimilar in PNH.
Eye on Pharma: Bevacizumab Update; Samsung Bioepis, J&J Settlement; Another EU Trastuzumab
December 6th 2023Outlook Therapeutics provides an update on the development of its bevacizumab candidate for age-related macular degeneration; Samsung Bioepis settles with Johnson & Johnson (J&J) over its ustekinumab biosimilar candidate; and the European Union gains another trastuzumab biosimilar.
Biosimilar Business Roundup for October 2023—Podcast Edition
November 5th 2023On this episode, we discuss the biggest news to come out of October 2023, including 3 regulatory approvals, 2 complete response letters, and new data and industry insights that have the potential to impact the entire US biosimilar industry.
HHS Praises Biosimilars Savings but Opportunities to Reduce Part B Spending Remain
November 28th 2023Although biosimilars have already generated savings for Medicare Part B programs and beneficiaries, opportunities for substantial reductions in spending remain, according to a report from the HHS.
Biosimilars Regulatory Roundup for September 2023—Podcast Edition
October 1st 2023On this episode, we discuss several regulatory updates from around the globe, including some European and Japanese approvals, the FDA’s 2-day workshop on the present science behind clinical efficacy testing for biosimilars, and streamlining biosimilar development.
Eye on Pharma: Adalimumab Updates; New Eylea Biosimilar Lawsuit; Canada Gains Stelara Biosimilar
November 22nd 2023Several companies make moves to further their adalimumab biosimilars, Regeneron sues Celltrion over biosimilar for Eylea (aflibercept), and Health Canada grants marketing authorization for biosimilar referencing Stelara (ustekinumab).