PUBLISHER: Grand View Research | PRODUCT CODE: 1771558
PUBLISHER: Grand View Research | PRODUCT CODE: 1771558
The global zilbrysq (zilucoplan) market size is anticipated to reach USD 679.58 million by 2030 and is expected to expand at a CAGR of 28.39% during the forecast period, according to a new report by Grand View Research, Inc. Zilbrysq, a first-in-class self-administered subcutaneous complement C5 inhibitor developed by UCB, is rapidly redefining the treatment paradigm for generalized myasthenia gravis (gMG). Approved in the U.S., EU, Japan, Canada, and South Korea, the therapy's unique delivery model and targeted mechanism have positioned it as a high-value therapeutic in the rare autoimmune neuromuscular segment.
A key driver is the growing prioritization of targeted, mechanism-based therapies in generalized myasthenia gravis (gMG), particularly for patients inadequately managed by corticosteroids, IVIg, or plasma exchange. Zilbrysq's ability to selectively inhibit terminal complement activation provides clinicians with a high-precision intervention that aligns with modern treatment algorithms focused on immunological specificity and long-term safety. In addition, its once-daily subcutaneous formulation disrupts the traditional hospital-dependent model, enabling therapy decentralization and supporting long-term disease control in outpatient and home settings. This has not only improved patient adherence but also reduced infusion-related costs-an increasingly important consideration for payers. The absence of direct SC C5 competitors further enhances its commercial positioning. Moreover, rare disease policy frameworks, including orphan drug incentives, expedited approvals, and national reimbursement prioritization in key markets, have accelerated both access and prescriber uptake. These dynamics, combined with a predictable treatment population and low therapeutic redundancy, continue to reinforce Zilbrysq's strategic value within the gMG treatment landscape.
Zilucoplan works by selectively inhibiting the cleavage of complement component 5 (C5), preventing formation of the membrane attack complex (C5b-9), which plays a central role in neuromuscular junction damage in AChR antibody-positive gMG. Unlike traditional immunosuppressants or IV biologics, Zilbrysq is administered via once-daily subcutaneous injection, allowing patients to self-manage their therapy at home. This significantly reduces treatment burden and improves long-term adherence, particularly among patients transitioning away from hospital-based IV infusions.
As of 2024, Zilbrysq holds 100% indication share for gMG, with no active development in additional indications following the strategic discontinuation of clinical programs in PNH, ALS, and IMNM. However, its strong efficacy profile, convenient SC delivery, and orphan drug exclusivity until 2030 provide a significant commercial moat and sustained revenue potential in its approved population.
Specialty pharmacies serve as the primary distribution channel, accounting for over 70% of dispensed volumes in 2024, reflecting the need for coordinated patient education, adherence monitoring, and cold-chain logistics. Hospital pharmacies continue to play a role in treatment initiation and support for newly diagnosed or complex cases. Meanwhile, home healthcare providers represent the fastest-growing distribution segment, driven by increasing demand for decentralized care and improved at-home treatment infrastructure.
In October 2023, the U.S. FDA approved Zilbrysq, supported by the pivotal RAISE study, which demonstrated statistically significant improvements in MG-ADL and QMG scores versus placebo. Subsequently, the European Commission approved the therapy in December 2023, followed by authorizations in the UK (January 2024), Canada (July 2024), and South Korea (November 2024). Moreover, a 60-week extension study (RAISE-XT) published in May 2024 validated the drug's long-term safety and durability of clinical benefit in gMG.
Further bolstering its real-world readiness, a human factors validation study published in November 2024 confirmed the usability of Zilbrysq's prefilled syringe for patients, caregivers, and healthcare professionals, reinforcing its at-home applicability and alignment with patient-centric care models. As demand for precision therapies rises globally, UCB invests in market expansion across Asia Pacific, Latin America, and the Middle East. Strategic emphasis on supply chain scalability, real-world data generation, and digital adherence platforms is expected to enhance global reach and continuity of care.
The commercial success of Zilbrysq reflects a broader industry shift toward self-administered, mechanism-specific biologics that offer durable disease control without the need for hospital-based delivery. With a strong clinical foundation and limited direct competition in the SC complement inhibitor class, Zilucoplan is poised to remain a market leader in gMG, driving meaningful improvements in patient outcomes and quality of life.