Alexion’s existing C5 complement inhibitor, targeted by biosimilar developers, is used to treat patients with paroxysmal nocturnal hemoglobinuria, but the biologic must be administered every 2 weeks versus ALXN1210’s proposed 8-week intravenous dosing schedule.
This week, Alexion Pharmaceuticals announced that the FDA has accepted for review a Biologics License Application for ALXN1210, a proposed long-acting C5 complement inhibitor, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Alexion says that it expects a regulatory decision on the product in February 2019.
PNH is an ultra-rare blood disorder that causes uncontrolled activation of the complement system and results in hemolysis—the destruction of red blood cells—which, in turn, can cause thrombosis, potentially leading to organ damage and early death.
Alexion’s existing C5 complement inhibitor, eculizumab (Soliris), is currently used to treat patients with PNH, but the biologic must be administered every 2 weeks (versus ALXN1210’s proposed 8-week intravenous dosing schedule, and a proposed once-weekly subcutaneous dosing option that will be the subject of an upcoming phase 3 study).
Click to read more about eculizumab.
Earlier this year, Alexion reported positive topline results from a phase 3 study that determined that patients receiving treatment with eculizumab can be safely and effectively switched to treatment with ALXN1210. In addition to demonstrating noninferiority to eculizumab in terms the primary endpoint—change in lactate dehydrogenase levels—the proposed drug was also noninferior to eculizumab in the proportion of patients with breakthrough hemolysis, change from baseline in quality of life as determined via the Functional Assessment of Chronic Illness Therapy Fatigue Scale, the proportion of patients avoiding transfusion, and the proportion of patients with stable hemoglobin levels.
Alexion’s executive vice president and head of research and development, John Orloff, MD, said in a statement that the company hopes to “establish ALXN1210 as the new standard of care for patients with PNH,” but it will remain to be seen whether payers will agree that a longer duration of action is worth a potentially higher cost; the brand-name Soliris already carries a list price of approximately $500,000 per patient per year, making the biologic one of the most expensive therapies in the world.
However, biosimilars for eculizumab may be on the horizon. The reference eculizumab, which is also approved to treat 2 other ultra-rare disorders, atypical hemolytic uremic syndrome and myasthenia gravis, is expected to lose patent protection in the United States and Europe within 3 years’ time.
The molecule is targeted by at least 1 biosimilar developer; Amgen is currently engaged in a phase 3 study in Spain that evaluates the efficacy and safety of its proposed biosimilar, ABP 959, versus the reference eculizumab in patients with PNH.
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.
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.
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.
Part 3: Study Questions Usefulness of Clinical Efficacy Trials for Oncology Biosimilars in Europe
November 16th 2023In part 3 of a 3-part series for Global Biosimilars Week, The Center for Biosimilars® reviews an analysis investigating whether clinical efficacy studies have an impact on prescribing decisions for oncology biosimilars across Europe.