Zilbrysq (Zilucoplan) Market Size, Share & Trends Analysis Report By Indication (Generalized Myasthenia Gravis), By Distribution Channel (Specialty Pharmacies, Hospital/Institution-Based Pharmacies, Home Healthcare Providers), By Region, And Segment Forecasts, 2025 - 2030

Zilbrysq (Zilucoplan) Market Summary

Zilbrysq’s targeted mechanism of action-complement C5 inhibition-positions it as a differentiated treatment for anti-AChR antibody-positive generalized myasthenia gravis (gMG). Its once-daily subcutaneous administration offers a patient-centric alternative to intravenous C5 inhibitors, supporting improved adherence and treatment continuity in outpatient and home-care settings. The drug's approval under orphan drug and accelerated pathways, combined with strong uptake in specialty pharmacy channels, is expected to drive market expansion. As of 2024, gMG remains the sole approved indication, but the product’s novel formulation and favorable safety profile continue to reinforce its competitive positioning within the neuromuscular disease segment.

Zilbrysq’s focused indication for anti-AChR antibody-positive generalized myasthenia gravis (gMG) provides a clearly defined commercial pathway, supported by favorable regulatory designations such as orphan drug status and priority review. Unlike broader immunosuppressive agents, Zilucoplan directly inhibits terminal complement component C5, offering a highly selective mechanism of action. This differentiation is clinically relevant in a market increasingly oriented toward mechanism-specific therapies, particularly for patients with suboptimal response to corticosteroids, IVIg, or plasmapheresis. With long-term treatment needs and a chronic disease burden, gMG represents a high-value therapeutic area, enabling Zilbrysq to command premium pricing and sustained market share over the forecast period.

The self-administered subcutaneous (SC) formulation of Zilucoplan is a key commercial advantage, especially when compared to existing intravenous complement inhibitors such as Soliris and Ultomiris. This formulation allows patients to avoid hospital-based infusions, reducing treatment fatigue and logistical barriers. Given the chronic nature of gMG and the need for sustained disease control, SC dosing enhances patient adherence while aligning with payer preferences for lower administration costs. The convenience of at-home use is expected to drive greater adoption, particularly in the specialty pharmacy channel, which accounted for over 70% of initial distribution in key launch markets. This shift reflects broader industry trends favoring decentralized care and patient-centric delivery models.

As of 2024, Zilbrysq faces limited direct competition within the complement C5 inhibitor space for gMG, with Soliris and Ultomiris primarily administered intravenously and priced at a premium. Meanwhile, therapies like Vyvgart and Rozanolixizumab operate through FcRn inhibition, targeting different immune pathways. This segmentation within the rare neuromuscular disease landscape limits therapeutic redundancy and supports market coexistence. With no biosimilars anticipated before 2030 and limited clinical activity surrounding other SC complement inhibitors, Zilbrysq is expected to retain exclusivity within its mechanism class. Ongoing post-marketing surveillance and potential real-world data will further strengthen its clinical positioning and payer acceptance, reinforcing long-term revenue potential.

Pipeline Analysis for Zilbrysq (Zilucoplan)

Zilbrysq (Zilucoplan), developed by UCB, currently holds approval exclusively for anti-AChR antibody-positive generalized myasthenia gravis (gMG). While initial development aimed to expand into multiple complement-mediated disorders, several programs were discontinued following clinical or strategic reassessment. Notably, the Paroxysmal Nocturnal Hemoglobinuria (PNH) program was halted after Phase 2 results showed suboptimal outcomes in patients previously treated with eculizumab, despite promising data in treatment-naïve cohorts. Similarly, a Phase 2 trial in Amyotrophic Lateral Sclerosis (ALS) under the HEALEY Platform Trial was stopped early for futility. Another trial in Immune-Mediated Necrotizing Myopathy (IMNM) was also terminated, with no further activity observed.

UCB’s pipeline strategy suggests that the company is investing in post-marketing data and real-world evidence to solidify Zilucoplan’s positioning rather than pursue broader clinical expansion at this time.

Patent and Exclusivity Profile of Zilbrysq (Zilucoplan)

Zilbrysq (Zilucoplan), developed by UCB, was granted U.S. FDA approval on October 17, 2023, for treating generalized myasthenia gravis (gMG) in anti-AChR antibody-positive adults. The product benefits from a robust intellectual property portfolio, with nine active U.S. patents covering its composition, formulation, and manufacturing processes. The earliest of these patents is set to expire in October 2027, with the potential for a six-month extension if pediatric exclusivity is granted.

In addition, Zilbrysq has received Orphan Drug Designation, which provides seven years of market exclusivity in the U.S. and will expire in October 2030. This regulatory exclusivity effectively prevents the approval of generic or similar products for the same indication during this period. Given the absence of biosimilar development and the strength of its IP protections, Zilbrysq is expected to retain market exclusivity for gMG through at least 2030, reinforcing its commercial position and supporting sustained revenue potential in the rare neuromuscular disease segment.

Market Concentration & Characteristics

Zilbrysq represents a significant innovation within the neuromuscular disease treatment landscape by offering the first self-administered subcutaneous C5 complement inhibitor approved for generalized myasthenia gravis (gMG). Its ability to inhibit terminal complement activation addresses a key immunopathological mechanism in AChR+ gMG patients, offering targeted immunomodulation without requiring intravenous infusion. The product's once-daily SC formulation enhances treatment convenience and supports long-term outpatient use, distinguishing it from intravenous alternatives like Soliris and Ultomiris. This delivery approach improves patient adherence and reduces healthcare system burden, contributing to its differentiated therapeutic profile.

Entry into the complement inhibition segment, particularly with SC biologics, is limited by high R&D costs, complex protein engineering, and the need for specialized delivery systems. Zilucoplan benefits from orphan drug incentives and regulatory exclusivity through 2030, further reinforcing its competitive position. The requirements for cold-chain distribution, physician familiarity with IV-based standards of care, and limited patient population size compound the challenges for follow-on entrants. UCB’s early commercialization, established specialty distribution network, and growing physician trust represent additional barriers for biosimilar or next-generation competitors.

Zilucoplan received FDA approval under an orphan drug designation and benefited from expedited review pathways due to the unmet gMG need. Regulatory scrutiny remains high, particularly around long-term safety and functional outcomes in chronic neuromuscular conditions. Health Technology Assessment (HTA) bodies in Europe and other regions are expected to evaluate Zilucoplan’s cost-effectiveness against established treatments such as IVIg, corticosteroids, and FcRn inhibitors. Post-marketing commitments, especially related to pharmacovigilance and pediatric investigation plans, may influence future labeling and access.

Zilucoplan competes indirectly with therapies such as IVIg, steroids, plasma exchange, and FcRn inhibitors like Vyvgart and Rozanolixizumab. However, its mechanism-specific targeting of complement C5 differentiates it from both upstream and downstream modulators of immune function. The convenience of subcutaneous administration and its efficacy in reducing disease activity in AChR+ gMG patients position it favorably in treatment algorithms. IV C5 inhibitors remain a substitute, though their hospital-based delivery may limit preference in certain geographies.

Following approvals in the U.S., Japan, the EU, and Canada, UCB is expected to pursue broader geographic expansion across Asia-Pacific, Latin America, and select Middle Eastern markets. Regulatory filings in regions such as Australia and South Korea are anticipated or underway, with future access dependent on local reimbursement frameworks and rare disease infrastructure. The subcutaneous route supports both specialty pharmacy distribution and decentralized care models, enabling adoption in countries with growing outpatient treatment capacity. Strategic partnerships and early physician engagement will accelerate uptake in newly targeted regions.

Indication Insights

The Generalized Myasthenia Gravis (gMG) segment accounted for the entire market share (100%) of Zilbrysq (Zilucoplan) in 2024, reflecting its exclusive regulatory approval and commercial focus. Zilucoplan has rapidly gained traction as a targeted therapy for AChR antibody-positive gMG, offering a differentiated approach through complement C5 inhibition. Unlike traditional immunosuppressants or broad-spectrum therapies, Zilbrysq directly interrupts the terminal complement cascade, a key driver of neuromuscular damage in gMG. Its once-daily subcutaneous administration supports outpatient and home-based care, improving adherence and quality of life for patients requiring long-term treatment. Clinical studies have consistently enhanced muscle strength, disease severity scores, and patient-reported outcomes, reinforcing its role in treatment algorithms. Continued expansion across major pharmaceutical markets, combined with favorable payer acceptance and physician adoption, is expected to sustain its dominant position in the indication over the forecast period.

The “Others” segment, while currently unapproved and non-revenue generating, is expected to represent the fastest-growing potential opportunity if Zilucoplan’s development is re-initiated in adjacent complement-mediated disorders. Earlier clinical efforts had explored indications such as Paroxysmal Nocturnal Hemoglobinuria (PNH), Amyotrophic Lateral Sclerosis (ALS), and Immune-Mediated Necrotizing Myopathy (IMNM). Although these programs were discontinued due to strategic or efficacy-related considerations, the underlying scientific rationale for complement inhibition in chronic inflammatory neuromuscular conditions remains valid. This segment could unlock incremental growth if new trials are launched or life-cycle management strategies resume. Furthermore, the increasing focus on subcutaneous biologics for autoimmune and rare neurological diseases suggests potential re-entry into pipeline programs targeting underserved conditions. While “Others” currently holds no market share, it represents a segment to monitor closely for future expansion initiatives.

Distribution Channel Insights

The Specialty Pharmacies segment dominated the market with a 70.35% revenue share in 2024, driven by the drug’s classification as a specialty biologic and its need for cold-chain logistics, patient education, and adherence support. Specialty pharmacies play a critical role in facilitating access to Zilbrysq, particularly in the outpatient and home-care settings, where self-administration of the subcutaneous formulation is preferred. These pharmacies are equipped to manage complex therapies for rare diseases, offering services such as benefit verification, patient coordination, and refill management-functions essential for chronic, long-term treatment regimens like Zilucoplan. As payer models increasingly favor distribution channels that lower administration costs and improve patient outcomes, specialty pharmacies are expected to maintain their leadership position in the Zilbrysq market throughout the forecast period.

The Home Healthcare Providers segment is projected to witness the fastest growth over the forecast period, supported by increasing demand for decentralized care and improvements in at-home treatment infrastructure. Zilucoplan’s once-daily subcutaneous formulation is well-suited for home administration, either self-administered or through nurse-assisted delivery, particularly in elderly or mobility-limited populations. Expanding telehealth services, patient monitoring platforms, and caregiver networks further facilitate home-based use. While still a smaller contributor to total market share in 2024, this segment is expected to grow significantly as healthcare systems prioritize treatment models that reduce hospital burden, lower costs, and improve patient convenience. As awareness and confidence in home-based biologic administration increase, this channel will likely emerge as a critical growth vector for Zilbrysq.

Regional Insights

North America accounted for the largest share of 77.78% of the Zilbrysq (Zilucoplan) Market in 2024, driven by high clinical awareness of generalized myasthenia gravis (gMG), early regulatory approval, and established specialty pharmacy infrastructure. The U.S. remains the core market, supported by robust diagnostic capabilities and a healthcare environment conducive to rapid adoption of orphan-designated biologics. Physician preference for targeted therapies with subcutaneous administration has contributed to a growing demand for Zilucoplan as a long-term immunomodulatory solution. Specialty pharmacies dominate the distribution landscape, while hospital-based pharmacies support initial therapy initiation and patient transition planning.

U.S. Zilbrysq (Zilucoplan) Market Trends

In the U.S., Zilucoplan's approval for AChR antibody-positive gMG has led to strong prescriber confidence and rapid formulary inclusion across both private and public payers. Combining a once-daily subcutaneous formulation, orphan drug exclusivity, and consistent clinical outcomes has enabled it to secure market share against existing intravenous C5 inhibitors. Specialty pharmacies handle most ongoing dispensing, while hospital settings are important for treatment initiation. Although pediatric use is not yet approved, real-world evidence and post-marketing research could support future market expansion.

Europe Zilbrysq (Zilucoplan) Market Trends

Europe represents a significant share of the Zilbrysq market, following centralized EU approval in December 2023. Major markets such as Germany, France, Italy, and Spain have initiated adoption in hospital-based settings, with growing interest in outpatient transition through specialty pharmacy networks. Reimbursement remains a key variable, with HTA bodies evaluating cost-effectiveness versus IV-based C5 inhibitors. Nonetheless, Zilucoplan’s SC route and strong clinical profile have made it a preferred option in patients seeking alternatives to chronic IV therapy. The shift toward individualized, home-based care is expected to support broader regional uptake.

The UK has seen steady integration of Zilbrysq into NHS pathways for eligible gMG patients. The drug’s SC delivery model aligns with government efforts to reduce hospital burden and enhance chronic disease self-management. Physician preference for therapies that minimize corticosteroid exposure has supported Zilucoplan’s adoption, particularly in adult patients with moderate-to-severe disease. Clinical trial participation and ongoing pharmacovigilance efforts are strengthening its profile across the country.

Germany’s advanced rare disease infrastructure and high diagnostic precision have facilitated early uptake of Zilbrysq. The need for sustained complement inhibition with improved administration convenience drives adoption. Hospital pharmacies lead the initial prescribing pathway, but follow-up dispensing is increasingly shifting to specialty providers. Real-world evidence collection and supportive clinical guidelines have accelerated acceptance among neurologists and tertiary care centers.

France’s centralized healthcare system and prioritization of orphan drugs have contributed to Zilucoplan’s rapid inclusion in treatment protocols for gMG. Adoption is primarily hospital-driven, with growing interest in transitioning patients to home-based self-administration through specialty pharmacies. Physician trust in complement inhibitors and favorable safety outcomes reported in clinical trials has increased utilization across both public and private sectors.

Asia Pacific Zilbrysq (Zilucoplan) Market Trends

The Asia Pacific region is emerging as a high-growth opportunity for Zilbrysq, particularly in countries like Japan and South Korea, where regulatory pathways for rare diseases are well-defined. Subcutaneous administration aligns well with healthcare systems aiming to decentralize chronic disease management. Hospital pharmacies facilitate initiation, while local specialty distributors are positioned to expand access to outpatient populations. Increased investment in rare disease diagnostics and physician education programs is expected to drive further regional market expansion.

Japan was the first country to approve Zilucoplan for gMG, reflecting the country’s proactive approach to managing rare autoimmune disorders. Widespread clinician experience with complement inhibition and government-backed reimbursement for orphan drugs has supported rapid uptake. Hospital-based neurologists are adopting Zilbrysq as an alternative to IV therapies, particularly for patients seeking greater flexibility and independence through SC self-injection.

As of 2025, Zilbrysq is not yet approved in China; however, future entry is anticipated given the country’s expanding rare disease framework and increased biologic innovation. Clinical awareness of gMG is improving, and training initiatives and access to diagnostic testing support this. The drug’s SC formulation and once-daily dosing will likely appeal to patients and providers, particularly in urban tertiary care centers. Regulatory progress and local partnerships will be crucial for entry and scale.

Latin America Zilbrysq (Zilucoplan) Market Trends

In Latin America, Zilucoplan adoption is still in early stages, with market entry focused on countries with defined rare disease pathways and centralized procurement models. Physician awareness of gMG and complement inhibition is growing, and national reference centers are beginning to evaluate new biologics for long-term use. Access challenges remain due to reimbursement constraints and varying regulatory timelines, but specialty pharmacies and hospital-led early access programs are expected to facilitate gradual uptake.

Brazil leads regional interest in Zilbrysq, which is supported by a growing network of neurology specialists and government initiatives to expand access to biologics for rare conditions. Hospital pharmacies handle most administration, with specialty pharmacies supplementing ongoing care for patients transitioning to home-based regimens. Clinical guidelines are evolving to incorporate targeted therapies like Zilucoplan, particularly for steroid-resistant or treatment-refractory gMG patients.

Middle East & Africa Zilbrysq (Zilucoplan) Market Trends

The MEA region presents an emerging opportunity for Zilbrysq, with market development concentrated in Gulf countries and South Africa. Awareness of gMG is increasing through professional training and rare disease initiatives. Hospital infrastructure and public-private collaborations are key enablers of access. While regulatory heterogeneity and pricing remain challenges, countries such as Saudi Arabia and the UAE are investing in expanding access to orphan drugs, creating a foundation for future uptake.

Saudi Arabia is positioning itself as a regional leader in rare disease management, with strategic investments in specialized diagnostic centers and public funding for high-cost biologics. Zilbrysq adoption is expected to grow as clinicians seek alternatives to IV immunosuppressants. The government’s focus on advancing biologic access and integrating new therapies into hospital formularies supports future expansion, particularly in major metropolitan regions.

Key Zilbrysq (Zilucoplan) Company Insights

UCB remains the sole commercial player in the Zilbrysq (Zilucoplan) Market as of 2025, following the product’s approval and launch across major global markets. The company is leveraging its experience in neurology and immunology to expand the drug’s presence through targeted physician education, specialty distribution partnerships, and digital adherence tools. With strong differentiation through subcutaneous delivery and a focused indication in gMG, Zilucoplan faces limited direct competition. UCB strategically emphasizes consolidating its market position in approved regions, generating real-world evidence, and potentially exploring life-cycle management opportunities. The absence of biosimilars or competing SC C5 inhibitors in late-stage development positions UCB to maintain long-term market leadership through at least 2030.

Recent Developments

• In November 2024, UCB Korea received approval from the Ministry of Food and Drug Safety (MFDS) for Zilbrysq (zilucoplan) Home Healthcare Providers Injection, marking it as the first self-administered subcutaneous treatment for generalized myasthenia gravis (gMG) in South Korea. This approval allows adult patients who are anti-acetylcholine receptor antibody-positive to manage their condition with daily subcutaneous injections, offering an alternative to intravenous therapies. The introduction of Zilbrysq in South Korea aligns with UCB's strategy to expand its presence in the region and provide innovative treatment options for rare autoimmune diseases.

• In July 2024, UCB Canada Inc. announced that Health Canada approved ZILBRYSQ™ (zilucoplan injection) for the treatment of adults with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive. This approval positions ZILBRYSQ as the first and only self-administered C5 complement inhibitor available in Canada for gMG. The pivotal Phase III RAISE study supported the decision, demonstrating statistically significant and clinically meaningful improvements in gMG-specific efficacy outcomes. ZILBRYSQ's subcutaneous administration allows patients to self-inject at home using pre-filled syringes, potentially reducing hospital visits and increasing patient independence. UCB Canada anticipates the availability of ZILBRYSQ in the fourth quarter of 2024.

• In November 2024, a human factors validation study published in Expert Review of Medical Devices demonstrated that the Zilbrysq (zilucoplan) Home Healthcare Providers can be safely and effectively used by patients with generalized myasthenia gravis (gMG), their caregivers, and healthcare providers. The study involved 75 participants across five user groups, including injection-naïve and injection-experienced patients and caregivers, as well as healthcare professionals. Participants performed simulated injections into a mock skin pad, first without guidance and then following the provided instructions for use (IFU). Initial results showed that 97.3% of participants successfully administered the injection without critical errors. After consulting the IFU, all participants achieved correct administration, highlighting the effectiveness of the instructional materials. The study also identified areas for improvement in the IFU, leading to enhancements that further reduced user errors. These findings support the usability of the Zilbrysq Home Healthcare Providers for at-home administration, potentially increasing patient adherence and independence in managing gMG.

• In May 2024, long-term data from the RAISE-XT open-label extension study demonstrated that Zilucoplan (Zilbrysq) maintains its safety and efficacy in treating generalized myasthenia gravis (gMG) over a 60-week period. The study included 200 patients who had previously participated in Phase 2 or Phase 3 trials. Those initially receiving Zilucoplan continued their regimen, while participants from the placebo group transitioned to Zilucoplan at a dosage of 0.3 mg/kg once daily. The most common treatment-emergent adverse events (TEAEs) reported were worsening MG (26%) and COVID-19 (25%), with serious TEAEs occurring in 32% of patients. Notably, the need for rescue therapy decreased over time, indicating improved disease stability. Efficacy assessments showed sustained improvements in MG Activities of Daily Living (MG-ADL) scores, with a least squares mean change of –6.06 points in patients originally on Zilucoplan and –6.46 points in those who switched from placebo, maintained through week 60. These findings support the long-term use of Zilucoplan as a safe and effective treatment option for gMG.

• In January 2024, UCB announced that the UK's Medicines and Healthcare products Regulatory Agency (MHRA) granted marketing authorization for Zilbrysq (zilucoplan) as an add-on therapy for adults with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive. Zilucoplan is the first once-daily, subcutaneous, self-administered complement C5 inhibitor approved for this indication in the UK. This approval provides a new treatment option for patients with gMG, a rare autoimmune condition that significantly impacts quality of life. UCB emphasized its commitment to delivering innovative solutions for rare diseases, aiming to improve patient outcomes and reduce the burden of chronic conditions.

• In October 2023, the U.S. Food and Drug Administration (FDA) approved Zilbrysq (zilucoplan) for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive.

• In December 2023, the European Commission approved Zilbrysq® (zilucoplan) as the first once-daily subcutaneous complement component 5 (C5) inhibitor for the treatment of generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) antibody-positive. This approval allows for self-administration, offering patients increased flexibility and convenience. The decision was based on data from the Phase III RAISE study, which demonstrated rapid and statistically significant improvements in gMG-specific outcomes over a 12-week period. Unlike monoclonal antibody C5 inhibitors, zilucoplan, a peptide, can be used concurrently with intravenous immunoglobulin and plasma exchange without needing supplemental dosing. This approval significantly advances the treatment options available for gMG patients in Europe.

Zilbrysq (Zilucoplan) Market Report Scope

Global Zilbrysq (Zilucoplan) Market Report Segmentation

This report forecasts revenue growth at the global, regional, and country levels and provides an analysis of the latest industry trends in each of the sub-segments from 2018 to 2030. For this study, Grand View Research has segmented the global Zilbrysq (Zilucoplan) Market report based on indication, distribution channel, and region:

• Indication Outlook (Revenue, USD Million, 2018 - 2030)

  • Generalized Myasthenia Gravis (gMG)

  • Others

• Distribution Channel Outlook (Revenue, USD Million, 2018 - 2030)

  • Specialty Pharmacies

  • Hospital/Institution-Based Pharmacies

  • Home Healthcare Providers

• Regional Outlook (Revenue, USD Million, 2018 - 2030)

  • North America

    • U.S.

    • Canada

    • Mexico

  • Europe

    • UK

    • Germany

    • France

    • Italy

    • Spain

    • Denmark

    • Sweden

    • Norway

  • Asia Pacific

    • Japan

    • China

    • India

    • Australia

    • South Korea

    • Thailand

  • Latin America

    • Brazil

    • Argentina

  • Middle East & Africa

    • South Africa

    • Saudi Arabia

    • UAE

    • Kuwait