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.
Enhancing Adoption of Infused Biosimilars for a Sustainable Future
October 30th 2024An IQVIA report highlights challenges to the sustainability of infused biosimilars in the US, citing rebate walls and reimbursement policies, and proposes key solutions to enhance adoption and benefits for all stakeholders.
Biosimilars in America: Overcoming Barriers and Maximizing Impact
July 21st 2024Join us as we explore the complexities of the US biosimilars market, discussing legislative influences, payer and provider adoption factors, and strategies to overcome industry challenges with expert insights from Kyle Noonan, PharmD, MS, value & access strategy manager at Cencora.
Strengthening the Supply Chain: Key Insights From FDA Commissioner Dr Robert Califf
October 25th 2024At the GRx+Biosims conference, FDA Commissioner Robert Califf, MD, stressed the urgent need for data transparency in the global supply chain and the role of collaboration and artificial intelligence in ensuring the resilience of biosimilar and generic drug production.
Exploring the Biosimilar Horizon: Julie Reed's Predictions for 2024
February 18th 2024On this episode of Not So Different, Julie Reed, executive director of the Biosimilars Forum, returns to discuss her predictions for the biosimilar industry for 2024 and beyond as well as the impact that the Forum's 4 new members will have on the organization's mission.
Calling for Unified Biosimilar Standards, Stronger Education at GRx+Biosims
October 23rd 2024At the GRx+Biosims conference, a fireside chat highlighted the need to streamline biosimilar development and strengthen industry collaboration, with Sarah Yim, MD, of the FDA, emphasizing education's key role in building trust and adoption.