CHMP Recommends Saphnelo Subcutaneous Administration for SLE in the EU, Offering Convenient Self-Administration Option
AstraZeneca has announced that its fully human monoclonal antibody Saphnelo (anifrolumab) has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) for subcutaneous (SC) self-administration in adult patients with systemic lupus erythematosus (SLE). The proposed once-weekly pre-filled pen formulation is intended for use on top of standard therapy, offering patients a convenient alternative to intravenous (IV) infusion.
This recommendation comes as part of AstraZeneca’s ongoing commitment to expanding access to innovative therapies for patients with autoimmune conditions, particularly those living with SLE, a chronic, often debilitating disease affecting millions worldwide. The CHMP’s opinion was based on interim results from the Phase III TULIP-SC trial, which demonstrated that subcutaneous administration of Saphnelo produced statistically significant and clinically meaningful reductions in disease activity compared with placebo among participants with moderate to severe, active, autoantibody-positive SLE who were receiving standard therapy.
Clinical Evidence Supporting Subcutaneous Use
The TULIP-SC trial was a multicentre, randomized, double-blind, placebo-controlled Phase III study designed to evaluate the efficacy and safety of SC anifrolumab in adults aged 18–70 years with moderate to severe SLE. Participants were randomly assigned 1:1 to receive 120 mg SC anifrolumab or placebo, administered via a pre-filled, single-use syringe. An interim analysis was performed when the first 220 participants reached week 52, with continued follow-up in an open-label extension for an additional 52 weeks.
The trial’s primary endpoint was reduction in disease activity measured using the British Isles Lupus Assessment Group-based Composite Lupus Assessment (BICLA) at week 52, which evaluates improvements across all organs affected at baseline and the absence of new disease flares. Key secondary endpoints included oral corticosteroid (OCS) use reduction, improvements in skin and joint manifestations, and overall health-related quality of life measures.
Results indicated that SC Saphnelo significantly reduced disease activity, replicating the efficacy observed with IV administration. The safety profile of SC administration was consistent with the known clinical profile of Saphnelo IV, with no new safety signals reported. Common adverse events included upper respiratory tract infections, infusion/injection site reactions, and mild viral infections, mirroring previously observed effects in IV-treated patients.
Expert Perspectives
Professor Thomas Dörner, Rheumatologist at Charité University Hospital, Berlin, and TULIP-SC investigator, highlighted the potential impact for patients:
The positive recommendation for the subcutaneous administration of anifrolumab in the EU is highly encouraging for people living with systemic lupus erythematosus. Many patients still rely on oral corticosteroids, which carry significant long-term side effects and accelerate functional impairment. With updated treatment guidelines emphasizing biologic therapy and early intervention, a self-administered subcutaneous form of anifrolumab could provide broader access while helping to minimize steroid use.
Ruud Dobber, Executive Vice President of AstraZeneca’s BioPharmaceuticals Business Unit, added:
Saphnelo IV has already transformed outcomes for many patients with SLE. The CHMP recommendation for SC administration brings us one step closer to offering a convenient, once-weekly self-administration option, expanding access to the clinically meaningful benefits of Saphnelo. We are also pursuing development in other diseases driven by type I interferon, including cutaneous lupus, lupus nephritis, myositis, and systemic sclerosis.
Understanding Systemic Lupus Erythematosus
SLE is a chronic autoimmune condition in which the immune system attacks healthy tissues, resulting in pain, fatigue, rashes, joint swelling, and fever. The disease predominantly affects women and carries a two- to three-fold increased mortality risk in Europe compared to the general population.
Long-term corticosteroid use is associated with irreversible organ damage in approximately 50% of patients within five years, highlighting the need for steroid-sparing therapies. Even modest reductions in daily OCS dose (e.g., 1 mg/day) can significantly reduce organ damage risk.
Recent EULAR (European Alliance of Associations for Rheumatology) treatment recommendations emphasize treat-to-target approaches, aiming for remission or low disease activity, minimizing oral corticosteroid use, and improving long-term outcomes including reduced organ damage, fewer flares, decreased hospitalizations, and better quality of life. The guidance recommends an OCS-sparing approach with a threshold of 5 mg/day or less whenever feasible.
Subcutaneous Administration: A Convenient Alternative
Since its 2021 approval, Saphnelo IV has been administered by healthcare professionals in clinics or hospitals. The SC formulation introduces a self-administered, once-weekly option, allowing patients and caregivers to administer treatment at home or in outpatient settings. This flexibility may improve adherence, reduce the burden on healthcare resources, and enhance patient quality of life.
Saphnelo binds to subunit 1 of the type I interferon (IFN) receptor, blocking the activity of type I IFNs — key cytokines involved in SLE pathophysiology, including IFN-alpha, IFN-beta, and IFN-kappa. By targeting this upstream inflammatory pathway, Saphnelo addresses the underlying drivers of disease activity, rather than solely mitigating symptoms.
Global Reach and Ongoing Development
Saphnelo IV is approved for moderate to severe SLE in over 70 countries, including the US, EU, and Japan, with regulatory submissions pending in additional markets. More than 40,000 patients globally have received treatment with Saphnelo to date.
The SC formulation is also under regulatory review in multiple countries, signaling a potential broader global rollout. Beyond SLE, Saphnelo is being investigated in Phase III trials for cutaneous lupus erythematosus, lupus nephritis, myositis, and systemic sclerosis, conditions in which type I interferon plays a critical role.
Financial and Collaborative Considerations
AstraZeneca acquired global rights to Saphnelo through an exclusive license and collaboration agreement with Medarex, Inc. in 2004, with subsequent amendments following Medarex’s acquisition by Bristol-Myers Squibb in 2009. AstraZeneca pays BMS a low- to mid-teens royalty, depending on geography, under the agreement.
AstraZeneca’s Leadership in Immune-Mediated Diseases
Saphnelo is part of AstraZeneca’s Respiratory & Immunology portfolio, a key growth driver focused on chronic, debilitating immune-mediated conditions. With a 50-year legacy in respiratory care, the company continues to develop innovative medicines, biologics, and new modalities, targeting previously unreachable biologic pathways. AstraZeneca aims to deliver life-changing therapies to help patients achieve clinical remission, reduce disease burden, and improve overall quality of life.
AstraZeneca’s commitment to addressing unmet needs in autoimmune and inflammatory diseases is exemplified by its continued development of Saphnelo, providing patients with more convenient, effective, and safe treatment options for SLE and beyond.
