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

U.S. Zilbrysq (Zilucoplan) Market Trends

The U.S. Zilbrysq (Zilucoplan) market size was estimated at USD 60.61 million in 2024 and is projected to grow at a CAGR of 21.64% from 2025 to 2030. The U.S. Zilbrysq (Zilucoplan) industry is witnessing robust growth following its FDA approval in October 2023 for the treatment of anti-AChR antibody-positive generalized myasthenia gravis (gMG). The approval under orphan drug designation and priority review pathways has accelerated its entry into the U.S. neuromuscular therapeutics space, where demand for targeted, mechanism-based treatments continues to rise.

Zilbrysq’s once-daily subcutaneous self-administration offers a compelling alternative to hospital-based intravenous complement inhibitors, supporting long-term disease management in both outpatient and home-care settings. Its differentiated profile has led to rapid adoption among neurologists treating refractory or corticosteroid-intolerant gMG patients. The presence of a well-established specialty pharmacy network, favorable commercial insurance coverage, and prescriber familiarity with complement pathway inhibition further strengthen Zilbrysq’s early market trajectory. While gMG remains the only approved indication in the U.S. as of 2024, its patient-centric formulation and durable efficacy data continue to position the product competitively against both IV C5 inhibitors and emerging FcRn-targeted therapies.

Zilbrysq’s focused label for anti-AChR antibody-positive generalized myasthenia gravis (gMG) provides a clear and differentiated commercial pathway in the U.S., where approximately 85% of diagnosed gMG patients test positive for AChR antibodies. The therapy’s approval under orphan drug designation and priority review by the FDA underscores the high unmet need in this rare, chronic condition. In contrast to broad-spectrum immunosuppressants, Zilucoplan’s selective inhibition of complement C5 directly targets the pathophysiologic cascade driving neuromuscular junction damage. This specificity is especially relevant in a treatment landscape increasingly oriented toward biologics with well-defined molecular targets. With gMG recognized as a lifelong condition requiring sustained control, Zilbrysq’s targeted mechanism and regulatory protections support premium pricing and long-term payer alignment, solidifying its role in treatment sequencing.

The once-daily subcutaneous (SC) formulation of Zilucoplan provides a significant strategic advantage in the U.S. healthcare setting, where patients and payers are actively seeking alternatives to intravenous (IV) therapies that require frequent clinical visits. Unlike Soliris or Ultomiris-which are delivered via infusions administered in hospital or outpatient infusion centers-Zilbrysq can be self-injected at home using a prefilled syringe. This approach aligns with growing demand for decentralized chronic disease management, reducing infusion-related costs and improving treatment adherence. In the U.S., where specialty pharmacies dominate the distribution of rare disease biologics, Zilucoplan’s SC format has enabled rapid integration into payer-preferred pharmacy networks and patient support programs, helping to mitigate initiation barriers and improve real-world outcomes. Its suitability for both commercial and Medicare populations further enhances uptake in a fragmented reimbursement landscape.

As of 2024, Zilbrysq faces limited direct competition in the U.S. for SC C5 complement inhibition, with no biosimilars in development and other approved C5 inhibitors-Soliris and Ultomiris-confined to intravenous administration. While newer FcRn inhibitors such as Vyvgart and Rozanolixizumab offer differentiated mechanisms, they do not overlap with the complement pathway and are often considered complementary rather than substitutive in refractory cases. This segmented competitive environment reduces therapeutic redundancy and supports market coexistence, allowing prescribers to tailor therapies based on individual patient profiles and response history. The lack of direct SC C5 competitors through at least 2030, combined with UCB’s ongoing investments in real-world data collection and provider education, is expected to preserve Zilbrysq’s clinical and economic moat in the U.S. rare neurology market.

Regulatory and Reimbursement Scenario: U.S. Market

Zilbrysq (Zilucoplan) received U.S. FDA approval in October 2023 under the orphan drug designation for the treatment of anti-AChR antibody-positive generalized myasthenia gravis (gMG), granting it seven years of market exclusivity. The approval followed a priority review and was based on results from the pivotal Phase 3 RAISE trial, which demonstrated clinically meaningful improvements in MG-ADL and QMG scores. Given its status as an orphan-designated biologic addressing a high-burden, low-prevalence condition, Zilucoplan has been integrated into U.S. payer formularies with relative speed, particularly among commercial and Medicare Advantage plans.

Coverage typically requires prior authorization, with criteria aligned to label specifications, including confirmed AChR antibody positivity and documented need for advanced immunotherapy. The drug is also listed under the Zilbrysq REMS (Risk Evaluation and Mitigation Strategy) program due to the risk of serious meningococcal infections, requiring vaccination and provider enrollment. From a reimbursement standpoint, specialty pharmacy distribution channels play a central role in ensuring continuity of care and managing benefit verification, co-pay assistance, and adherence support. The availability of a self-administered prefilled syringe format further supports favorable payer alignment, as it reduces infusion-associated costs and appeals to value-based care models increasingly adopted across U.S. health systems.

Market Concentration & Characteristics

Zilbrysq introduces a differentiated therapeutic approach in the U.S. gMG market as the first self-administered complement C5 inhibitor, marking a shift away from infusion-based complement therapies. Its design allows for targeted inhibition of the terminal complement cascade, providing precise immunomodulation for patients with AChR antibody-positive generalized myasthenia gravis. This innovation is especially impactful in the U.S., where healthcare systems increasingly prioritize patient autonomy, reduced clinic dependency, and chronic care management models. By enabling at-home administration through a prefilled syringe, Zilucoplan addresses a major unmet need for convenience in long-term treatment, particularly among working-age and elderly populations managing mobility or transportation constraints.

In the U.S., barriers to entry are intensified by the need for clinical differentiation, payer alignment, and established provider relationships. Developing SC complement inhibitors with sufficient safety and efficacy to meet FDA standards requires extensive investment in biologic manufacturing, large-scale trials, and REMS-compliant safety programs. Zilbrysq’s orphan drug designation and seven-year exclusivity, combined with its early establishment in specialty pharmacy networks, create a high threshold for competing entrants. Additionally, UCB’s direct-to-provider engagement and integration into U.S. neurology treatment algorithms further solidify its lead against potential follow-on candidates.

The U.S. regulatory environment has been highly supportive of Zilbrysq’s launch, with orphan drug designation and priority review facilitating rapid approval. However, the post-approval phase is tightly regulated under the Zilbrysq REMS program, which mandates prescriber registration and meningococcal vaccination due to the elevated infection risk associated with complement inhibition. Furthermore, Zilucoplan’s long-term safety data are under continued FDA review as part of post-marketing obligations. Although the U.S. does not rely on HTA agencies as in Europe, payer assessments of cost-effectiveness and real-world outcomes are increasingly important in determining access tiers and prior authorization criteria.

In the U.S., Zilucoplan faces indirect competition from IVIg, corticosteroids, plasma exchange, and other immunotherapies used in gMG. However, its position is most closely aligned with intravenous C5 inhibitors such as Soliris and Ultomiris. While those agents have strong clinical track records, their infusion-based delivery limits flexibility, especially for patients seeking to avoid infusion centers. FcRn inhibitors like Vyvgart and Rozanolixizumab represent adjacent therapeutic options rather than direct substitutes, targeting a different immunological pathway. This mechanism-based segmentation supports Zilbrysq’s integration as a complementary or stepwise therapy depending on patient response profiles.

In the U.S., Zilucoplan’s growth is being fueled by wider formulary access, expanding neurologist adoption, and increased patient education initiatives. Uptake is strongest in metropolitan areas with high volumes of neuromuscular specialists, but growth in suburban and rural markets is accelerating due to the drug’s SC convenience and specialty pharmacy reach. UCB’s U.S. commercialization strategy includes payer contracting, digital adherence platforms, and REMS-compliant onboarding, helping to reduce barriers to initiation and support continuity of care. Future market depth will depend on expansion into Medicaid and Veterans Health Administration networks and the potential for broader provider education in community neurology practices.

Indication Insights

Generalized Myasthenia Gravis (gMG) accounted for 100% of the U.S. Zilbrysq (Zilucoplan) market in 2024, consistent with its exclusive FDA-approved indication. The domestic patient pool is well-characterized, with a significant proportion of gMG patients testing positive for anti-AChR antibodies-the exact subgroup Zilucoplan targets. This focused indication supports high diagnostic precision and streamlined patient selection, enabling rapid integration into U.S. neurology practice.

Zilbrysq’s complement C5 inhibition offers a direct mechanism to reduce neuromuscular junction damage, making it a preferred option for patients with inadequate response to first-line immunosuppressants or those intolerant to corticosteroids. U.S. clinical adoption has been reinforced by strong Phase 3 trial results, payer coverage under orphan drug frameworks, and a favorable safety profile, particularly in the context of long-term disease management. Given the maturity of the gMG treatment infrastructure in the U.S., including national neuromuscular disease centers and coordinated specialty care, Zilucoplan is expected to retain its market leadership across this indication through 2030.

The “others” segment in the U.S. currently represents an untapped opportunity, as Zilucoplan has no approved or active development programs outside gMG. Earlier U.S.-based trials in PNH, ALS, and IMNM were halted, but interest remains in complement modulation for rare neurological and hematological diseases. Should new domestic trials be initiated or prior programs revisited with revised endpoints or subpopulation targeting, the “Others” segment could deliver incremental volume growth. Furthermore, growing clinician familiarity with SC complement inhibition and patient preference for at-home biologics positions Zilucoplan for potential future expansion-pending clinical validation. U.S. regulatory incentives, such as the Rare Pediatric Disease Priority Review Voucher and Fast Track designation, may further support re-entry into adjacent indications should unmet needs and development economics align.

Distribution Channel Insights

Specialty Pharmacies dominated Zilbrysq distribution in the U.S. in 2024, capturing approximately 70.35% of total dispensed volume. This dominance reflects the product’s classification as a specialty-tier, high-touch biologic, requiring robust care coordination, REMS compliance, and temperature-sensitive logistics. U.S. specialty pharmacy networks are optimized for rare disease therapies and support key services such as prior authorization management, insurance verification, nurse education, and adherence monitoring. These capabilities are essential for ensuring successful initiation and ongoing self-administration of Zilucoplan’s prefilled syringe format. With most U.S. payers preferring specialty pharmacy fulfillment over hospital buy-and-bill for SC biologics, this channel is expected to remain the primary driver of access, especially for patients on commercial and Medicare Advantage plans.

The home healthcare providers segment in the U.S. Zilbrysq (Zilucoplan) industry is poised for the fastest growth, reflecting a broader national trend toward decentralized biologic administration. Zilucoplan’s once-daily SC delivery is compatible with nurse-assisted or self-injection at home, reducing the need for clinic visits and aligning with value-based care priorities. Growth is being further supported by telehealth expansion, remote patient monitoring tools, and home infusion care networks, especially for elderly or mobility-constrained populations. While this segment accounted for a smaller share in 2024, it is gaining traction across integrated delivery networks and Accountable Care Organizations (ACOs) focused on reducing hospital dependency. As confidence in home-based biologic treatment models increases across payers and providers, home healthcare is expected to evolve into a strategic channel for improving both Zilucoplan’s reach and real-world outcomes.

Key U.S. Zilbrysq (Zilucoplan) Company Insights

As of 2025, UCB remains the sole commercial entity operating in the U.S. Zilbrysq (Zilucoplan) market, following the drug’s FDA approval for generalized myasthenia gravis (gMG) in October 2023. Leveraging its longstanding expertise in neurology and immunology, UCB has executed a focused U.S. launch strategy centered on specialist engagement, specialty pharmacy partnerships, and REMS-compliant provider onboarding. The company has prioritized education initiatives across high-volume neuromuscular centers and digital adherence tools to support long-term use of the self-administered subcutaneous formulation.

Zilucoplan’s distinct mechanism as a complement C5 inhibitor, combined with its once-daily at-home administration model, has enabled it to gain early traction in a highly targeted treatment population. With no biosimilars in development and existing C5 therapies (e.g., Soliris, Ultomiris) confined to intravenous delivery, UCB faces minimal direct competition within the subcutaneous complement inhibition segment. The company is actively generating real-world evidence (RWE) in the U.S. to support formulary expansion and reinforce prescriber confidence. Strategic priorities for UCB include deepening penetration into regional neurology practices, expanding payer coverage across commercial and public plans, and strengthening long-term REMS compliance infrastructure. In the absence of late-stage SC-based C5 competitors, UCB is expected to maintain market leadership in the U.S. through at least 2030.

Recent Developments

• In March 2025, interim results from the Phase 3 RAISE-XT extension study were presented at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, highlighting the sustained efficacy of Zilbrysq (zilucoplan) in adults with generalized myasthenia gravis (gMG). After more than two years of daily subcutaneous treatment, patients continued to experience significant clinical benefits. Specifically, 87.7% of participants were MG-ADL responders, and 85.7% were QMG responders at week 120. Additionally, the proportion of patients achieving minimal symptom expression increased from 31.7% at week 24 to 41.4% at week 120. The treatment was generally well-tolerated, with most adverse events being mild or moderate in severity. These findings reinforce Zilbrysq's long-term therapeutic value in managing gMG.

• In January 2024, UCB announced the U.S. availability of Zilbrysq (zilucoplan) for the treatment of generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor antibody positive. Zilbrysq is the first approved once-daily subcutaneous, targeted complement component 5 (C5) inhibitor for gMG. The medication is available by prescription in pre-filled syringes that are self-administered on a once-daily basis. Zilbrysq received Food and Drug Administration (FDA) approval in 2023 based on results from the RAISE study, a phase 3, multicenter, randomized, double-blind, placebo-controlled study that assessed the efficacy, safety, and tolerability of Zilbrysq as a treatment for 174 adult participants with a diagnosis of anti-AChR Ab+ gMG. After 12 weeks, participants in the Zilbrysq group showed a greater reduction in the primary outcome measure of Myasthenia Gravis–Activities of Daily Living (MG-ADL) score compared to those in the placebo group. The prescribing information for Zilbrysq contains a Boxed Warning for serious meningococcal infections; therefore, it is only available to healthcare providers who enroll in the Zilbrysq Risk Evaluation and Mitigation Strategies (REMS) program. Zilbrysq, along with Rystiggo (rozanolixizumab-noli), is available through UCB’s ONWARD program, which provides personalized support to patients who use targeted treatments for gMG.

• In October 2023, UCB announced that 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. Zilucoplan is the first once-daily subcutaneous, targeted C5 complement inhibitor for gMG and the only once-daily gMG-targeted therapy for self-administration. The FDA approval is based on the pivotal Phase 3 RAISE study, which demonstrated that treatment with zilucoplan resulted in statistically significant improvements in MG-specific efficacy outcomes compared to placebo. With this approval, UCB offers the U.S. gMG community the opportunity to benefit from a choice of two new targeted therapies from a single company, as RYSTIGGO (rozanolixizumab-noli) had already been FDA approved and launched in the U.S. UCB's portfolio of two different medicines for gMG, each with a distinct mechanism of action, provides physicians with new and additional choices, embodying the company's commitment to addressing the gMG community's unmet needs.

U.S. Zilbrysq (Zilucoplan) Market Report Scope

U.S. Zilbrysq (Zilucoplan) Market Report Segmentation

This report forecasts revenue growth at the country level 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 U.S. Zilbrysq (Zilucoplan) market report based on indication, and distribution channel:

• 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