The purpose of this study is to assess the steady state (SS) concentrations of zilucoplan (ZLP) and its major metabolites in mature breast milk of healthy study participants following injection of repeated once-daily doses of ZLP.

Start Date09 Jul 2025

Sponsor / Collaborator

UCB Biopharma SRL

NCT06815133

/ Not yet recruitingNot ApplicableIIT

Multicenter Retrospective Study on the Short- and Medium-Term Efficacy and Tolerance of Zilucoplan Therapy in a Cohort of French Patients with Anti-AChR Myasthenia Gravis

This study called "Multicenter Retrospective Study on the Short- and Medium-Term Efficacy and Tolerance of Zilucoplan Therapy in a Cohort of French Patients with Anti-AChR Myasthenia Gravis", is investigating the effects of a new treatment called Zilucoplan (generic name: ZILBRYSQ) on patients in France with a condition known as myasthenia gravis. Myasthenia gravis is an autoimmune disease that causes muscle weakness that worsens throughout the day with activity and impacts the ability to work and perform daily activities. The study aims to see if Zilucoplan can improve muscle strength and quality of life over three months, while also assessing its tolerance and safety by monitoring side effects. Researchers hope this treatment will offer significant benefits for people with this challenging condition.

Start Date01 Mar 2025

Sponsor / Collaborator

Centre Hospitalier Universitaire de Nice

NCT06435312

/ Enrolling by invitationPhase 3

An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Activity of Zilucoplan in Pediatric Study Participants With Acetylcholine Receptor Antibody Positive Generalized Myasthenia Gravis

The purpose of this study is to assess the long-term safety and tolerability of an additional 52 weeks of Zilucoplan treatment administered by subcutaneous injection once daily in pediatric study participants

Start Date19 Nov 2024

Sponsor / Collaborator

UCB Biopharma SRL

100 Clinical Results associated with Zilucoplan

100 Translational Medicine associated with Zilucoplan

100 Patents (Medical) associated with Zilucoplan

Literatures (Medical) associated with Zilucoplan

01 Sep 2025·REVUE NEUROLOGIQUE

Zilucoplan as a fast-acting treatment for severe myasthenic exacerbation: A case series

Letter

Author: Acket, B ; Cointault, O ; Cintas, P ; Espagno, L ; Fabry, V ; Girardie, P

01 Aug 2025·Brain and nerve = Shinkei kenkyu no shinpo

[Myasthenia Gravis Treated with Zilucoplan Prior to Extended Transsternal Thymectomy for the Prevention of Myasthenic Crisis: A Case Report].

Article

Author: Honda, Masaya ; Koga, Michiaki ; Shimizu, Fumitaka ; Tanaka, Toshiki ; Yamanaka, Nanami ; Kanda, So ; Nakamori, Masayuki

A 50year-old female was diagnosed with myasthenia gravis (MG) following aspiration pneumonia. Despite treatment with prednisolone (5mg/day) and intravenous immunoglobulin (IVIg), the bulbar palsy persisted. Additionally, chest CT revealed findings suggestive of invasive thymoma or thymic carcinoma, leading to a planned thymectomy with median sternotomy. This case presents a high-risk of myasthenic crisis due to thymoma-associated MG, persistent bulbar palsy, and the need for highly invasive surgery. Therefore, enhanced immunotherapy was required to prevent this crisis, and zilucoplan was chosen because of its rapid onset of action and compatibility with IVIg. Following the initiation of zilucoplan, there was prompt improvement in the symptoms of MG. Effective preoperative control of MG led to a good clinical course, with no significant postoperative myasthenic crisis or exacerbation of symptoms. This is the first report on the use of zilucoplan for the prevention of perioperative myasthenic crisis. (Received April 14, 2025; Accepted June 3, 2025, Published August 1, 2025).

01 Jul 2025·JOURNAL OF THE NEUROLOGICAL SCIENCES

Efficacy of zilucoplan in patients with generalised myasthenia gravis who have not previously received immunoglobulin or plasma exchange: A subgroup analysis from the Phase 3 RAISE study

Article

Author: Masuda, Masayuki ; Boroojerdi, Babak ; Grimson, Fiona ; Howard, James F ; Utsugisawa, Kimiaki

BACKGROUND:

RAISE (NCT04115293; N = 174) was a randomised, double-blind, placebo-controlled, Phase 3 study of zilucoplan, a macrocyclic peptide complement component 5 inhibitor, in patients with anti-acetylcholine receptor antibody-positive (AChR Ab+) generalised myasthenia gravis (gMG). Zilucoplan showed clinically meaningful improvements in myasthenia gravis (MG)-specific outcomes versus placebo in the overall population. We assessed the profiles of RAISE patients without and with prior immunoglobulin (intravenous and subcutaneous) or plasma exchange (PLEX) use.

METHODS:

Adults with anti-AChR Ab+ gMG were randomised 1:1 to daily, self-administered, subcutaneous zilucoplan 0.3 mg/kg or placebo for 12 weeks. The primary efficacy endpoint was change from baseline (CFB) to Week 12 in Myasthenia Gravis Activities of Daily Living (MG-ADL) score. We conducted a prespecified, descriptive analysis of subgroups without and with prior immunoglobulin/PLEX.

RESULTS:

At baseline, 54 patients had no prior immunoglobulin/PLEX (zilucoplan: n = 29; placebo: n = 25); this subgroup had experienced milder disease and shorter disease duration from diagnosis than the subgroup with prior immunoglobulin/PLEX. Mean (standard error) CFB in MG-ADL score was -4.22 (0.71) with zilucoplan versus -2.61 (0.50) with placebo in the subgroup without prior immunoglobulin/PLEX, and - 4.93 (0.54) versus -2.94 (0.50) in the subgroup with prior immunoglobulin/PLEX. Zilucoplan was well tolerated with a similar safety profile in both subgroups.

CONCLUSIONS:

In the RAISE study, patients without prior immunoglobulin/PLEX, who had milder disease, experienced improvements in MG-specific outcomes with zilucoplan that were comparable to those seen in patients with prior immunoglobulin/PLEX. These findings may support early use of zilucoplan in the gMG treatment paradigm.

News (Medical) associated with Zilucoplan

08 Sep 2025

·www.fiercebiotech.com

Dianthus\' gMG prospect blooms, with phase 2 win setting up pivotal plans

Dosing is central to Dianthus Therapeutics\' plans to differentiate its candidate in a competitive market. \n Dianthus Therapeutics is advancing its challenge for the increasingly competitive generalized myasthenia gravis (gMG) market, outlining plans to move into phase 3 on the back of hits in a midphase study.The phase 2 trial compared two doses of claseprubart, an antibody engineered to selectively target the classical pathway, to placebo in 65 adults with AChR-positive gMG. Patients received claseprubart every two weeks via subcutaneous injection. While the primary endpoint looked at safety and tolerability, the secondary endpoint data provided a glimpse of the efficacy of the drug candidate. At the low dose, Dianthus reported a 4.6-point improvement on MG-ADL, a scale that measures the impact of gMG on daily functions. The improvement rose to 5.4 points on the high dose. Adjusted for placebo, the improvements on the low and high doses were 1.8 points and 2.6 points, respectively.Dianthus shared the results alongside data on QMG, a physician\'s score of disease severity. QMG scores improved 4.4 points and 4.5 points, respectively, on the low and high doses. Scores improved by two points in the placebo arm. Dianthus also reported statistically significant improvements on other measures of gMG. The biotech shared cross-trial comparisons to show how claseprubart performed against AstraZeneca’s Soliris and Ultomiris and UCB’s Zilbrysq. Claseprubart outperformed the competition in the data shared by Dianthus, with the caveat that such cross-trial comparisons can be unreliable. The company found the drug candidate was generally well tolerated, giving it a data set to inform talks with the FDA and preparations for phase 3. Dianthus plans to test two regimens of the low dose in phase 3, with patients receiving the study drug every two or four weeks. Dianthus didn’t test four-week dosing in the phase 2 study, but the lack of statistical difference between the low and high doses, coupled with evidence such as half-life data, led it to speculate that a less-frequent regimen could work. The two-week dose is Dianthus’ target product profile, but CEO Marino Garcia sees an opportunity to go beyond the base case.“Why not go further? Why not push that differentiation? I don\'t see much of a downside to be honest,” Garcia said on a call with investors to discuss the data. Dosing is central to Dianthus’ plans to differentiate its candidate in a competitive market. The company said more than 80% of patients with AChR-positive gMG have yet to switch to a biologic, despite the availability of C5 inhibitors such as Ultomiris and FcRn drugs, including argenx’s Vyvgart. Dianthus has identified intravenous and high-volume subcutaneous dosing as a barrier to uptake of rival drugs.That thinking led Dianthus to develop an autoinjector. The overarching goal is to combine the efficacy of C5 inhibitors such as Ultomiris with the safety of C1 blockers such as Enjaymo and the convenience of Dupixent. Analysts at Evercore ISI said in a note to clients that: “Both doses clearly met all the bull case efficacy bars” the firm had discussed in preview ahead these data. They added that the biotech “convincingly” matched the C5s. It didn’t see a dose response, thus “opening the door to a longer Q4W dosing interval.”

$Dianthus\' gMG prospect blooms, with phase 2 win setting up pivotal plans$

Clinical ResultPhase 2Phase 3

24 Jul 2025

·www.biospace.com

AstraZeneca’s Claims PIII Win With Nanobody Treatment for Myasthenia Gravis

iStock, Robert Way The company didn’t share specific data for the molecule, gefurulimab, but said it hit all endpoints in the Phase III PREVAIL trial and promised to share more at an upcoming scientific meeting. AstraZeneca’s investigational myasthenia gravis treatment gefurulimab cleared a Phase III trial, meeting all primary and secondary endpoints, the company announced Thursday. The company did not release specific numerical data, only noting that gefurulimab “demonstrated a statistically significant and clinically meaningful improvement from baseline” using the Myasthenia Gravis Activities of Daily Living (MG-ADL) scale, a validated measurement tool for symptoms of myasthenia gravis. Analysts at William Blair said in a note Thursday morning on the news that investors should keep in mind “the minimal clinically important difference on the MG-ADL scale is considered to be ≥2 points from baseline.” A comparable approved daily treatment, UCB’s Zilbrysq, got a 4.39-point reduction using that scale in its Phase III trial, the group wrote. Gefurulimab is a complement C5 inhibiting nanobody, a fragment of a normal antibody. The molecule is being tested in the Phase III PREVAIL trial in adults with anti-acetylcholine receptor (AChR) antibody-positive (Ab+) generalized myasthenia gravis, an autoimmune condition that breaks down communications between the brain and muscles. According to AstraZeneca, 85% of patients with myasthenia gravis have this version of the disease. More detailed information will be presented at a future medical meeting, according to AstraZeneca. The William Blair analysts speculated that that presentation could be at the Myasthenia Gravis Foundation of America (MFGA)’s sessions at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting on Oct. 29. Zilbrysq is the only FDA-approved subcutaneous treatment for gMG. Gefurulimab could compete with Zilbrysq given that it is a self-administered weekly drug. However, the William Blair analysts cautioned that argenx’s Vyvgart still leads in the $20 billion myasthenia gravis market, given its two-year head-start, having received FDA approval in June 2023. Both AstraZeneca’s and argenx’s stock prices were up about 1.2% in morning trading following the news. The PREVAIL announcement follows a week of newsmaking for AstraZeneca. The company stumbled last week in another rare disease setting after its antibody anselamimab failed to improve survival or hospitalization in patients with amyloidosis. Earlier this week the company also announced a new $50 billion manufacturing push in the U.S., led by a plant in Virginia that will create the company’s metabolic drugs aimed at cardiovascular conditions and obesity.

Phase 3Clinical ResultDrug Approval

22 May 2025

·www.prnewswire.com

Johnson & Johnson's IMAAVY Gets FDA Approval, Taking on Top Players in Myasthenia Gravis Market | DelveInsight

The FDA has approved Johnson & Johnson's anti-FcRn antibody, nipocalimab, for the treatment of generalized myasthenia gravis. The drug will be sold under the brand name IMAAVY. This approval positions J&J to challenge leading competitors in the myasthenia gravis market, such as AstraZeneca, which markets the monoclonal antibodies SOLIRIS and ULTOMIRIS. J&J will also be competing with Argenx's VYVGART and UCB's RYSTIGGO and ZILBRYSQ. LAS VEGAS, May 22, 2025 /PRNewswire/ -- Myasthenia gravis is an autoimmune disorder in which antibodies disrupt the communication between nerves and muscles, resulting in skeletal muscle weakness. This weakness primarily affects voluntary muscles that control eye movement, facial expression, swallowing, and limb function. The condition is predominantly driven by autoantibodies targeting either the acetylcholine receptor (AChR-Ab) or a receptor-associated protein known as muscle-specific tyrosine kinase (MuSK-Ab). According to DelveInsight's analysis, there were approximately 300,000 diagnosed prevalent cases of myasthenia gravis across the 7MM in 2024. This number is expected to grow steadily over the forecast period (2025–2034), with a projected CAGR. The current myasthenia gravis therapeutics market mainly consists of symptomatic treatments such as acetylcholinesterase inhibitors like Mestinon. However, achieving full clinical remission with symptomatic therapy alone is challenging. As a result, immunosuppressive drugs are frequently introduced. First-line immunotherapy typically involves high-dose oral corticosteroids such as prednisone or prednisolone, which can induce a rapid response within 2 to 4 weeks. To reduce reliance on steroids and improve long-term disease control, nonsteroidal immunosuppressants (NSISTs) are often used. These include antimetabolites (e.g., azathioprine, mycophenolate mofetil, methotrexate, and cyclophosphamide) and calcineurin inhibitors (e.g., tacrolimus and cyclosporine). While NSISTs may be used as standalone treatments, they usually take 6 to 9 months to produce noticeable effects. Immunomodulatory therapies, including intravenous immunoglobulin (IVIg) and plasmapheresis (PLEX), are typically reserved for acute exacerbations or myasthenic crises. They are also employed as chronic treatments in patients who cannot tolerate or do not respond to standard immunosuppressants. Although traditional immunosuppressive strategies can help manage symptoms, achieving long-term, stable remission remains difficult. Additionally, about 15% of patients are considered treatment-refractory, meaning they either fail to respond to or cannot tolerate multiple immunosuppressive options. These patients often face the dual challenge of persistent symptoms and the adverse effects of long-term immunosuppression. Learn more about the myasthenia gravis disease market @ Myasthenia Gravis Treatment Medications The myasthenia gravis treatment market is well-established, with multiple approved drugs for myasthenia gravis, including SOLIRIS, ULTOMIRIS, RYSTIGGO, ZILBRYSQ, and VYVGART, among others. Alexion Pharmaceuticals' SOLIRIS is a monoclonal antibody that targets and inhibits complement protein C5, thereby blocking the formation of the terminal complement complex. This action helps reduce complement-driven hemolysis in PNH and thrombotic microangiopathy in aHUS. Although its exact mechanism in gMG and NMOSD is not fully known, it is believed to decrease complement complex accumulation. Between 2017 and 2023, SOLIRIS gained regulatory approvals in Japan, the US, and the EU for treating gMG, including pediatric indications in Japan. ULTOMIRIS (ravulizumab), also from Alexion Pharmaceuticals, is the first long-acting C5 inhibitor, offering rapid, complete, and sustained suppression of complement activity. It binds to C5, preventing its cleavage and subsequent formation of the membrane attack complex (C5b-9). ULTOMIRIS is effective in managing terminal complement-mediated hemolysis in PNH and thrombotic microangiopathy in aHUS. Although the precise mechanism in gMG and NMOSD remains unclear, it is thought to reduce complement deposition at the neuromuscular junction and block aquaporin-4 antibody-mediated complement activation. It received approvals from the US FDA, EMA, and Japan's PMDA in 2022. Get an in-depth assessment on generalized myasthenia gravis medication for adults @ ULTOMIRIS vs SOLIRIS for Myasthenia Gravis UCB Biopharma's RYSTIGGO is a subcutaneously administered, humanized monoclonal antibody that binds with high affinity to the human neonatal Fc receptor (FcRn). It received regulatory approval from the US FDA and Japan's PMDA in 2023, followed by the EC in January 2024. ZILBRYSQ, also developed by UCB Biopharma, is the first once-daily subcutaneous peptide-based inhibitor specifically targeting complement protein C5. It works by blocking C5 cleavage and the formation of the terminal complement complex C5b-9. Although its precise action in gMG remains uncertain, it is thought to limit C5b-9 accumulation at the neuromuscular junction. ZILBRYSQ gained approval from the US FDA, EMA, and PMDA in 2023. VYVGART, from Argenx, is a human IgG1 antibody fragment that targets FcRn, leading to a decrease in circulating IgG autoantibodies. It is the first FcRn blocker to receive regulatory approval. Argenx's VYVGART HYTRULO (also marketed as VYVDURA) combines efgartigimod alfa, an FcRn inhibitor, with hyaluronidase, an endoglycosidase, for treating adults with generalized myasthenia gravis. This formulation was approved by the US FDA and EMA in 2023 and by the PMDA in January 2024. Uncover the mechanism of FcRn inhibitor myasthenia gravis medication @ Myasthenia Gravis MoA On April 30, 2025, the FDA approved Johnson & Johnson's nipocalimab, branded as IMAAVY, as a new therapy for gMG. This approval puts IMAAVY among the list of approved drugs for myasthenia gravis treatment. The approval, which J&J says encompasses the widest eligible patient population with gMG, includes individuals aged 12 and above who test positive for AChR or anti-muscle-specific kinase (MuSK) antibodies. IMAAVY functions by inhibiting the FcRn protein, which plays a role in maintaining circulating IgG antibodies, key drivers in many autoimmune disorders. By blocking FcRn, IMAAVY significantly reduces harmful IgG autoantibodies while preserving the body's overall immune response, according to clinical data. J&J sees broader potential for IMAAVY beyond gMG, believing its mechanism of action could apply across multiple autoimmune conditions due to IgG's involvement in various diseases. The FDA's decision followed results from the ongoing Phase III Vivacity-MG3 trial, which evaluates IMAAVY in combination with standard care versus placebo and standard care in 199 adult gMG patients, including 153 antibody-positive individuals. Additionally, J&J is conducting a Phase II/III pediatric trial called Vibrance, where IMAAVY, combined with standard care, achieved its primary goal by reducing total serum IgG levels by 69% over 24 weeks. J&J acquired IMAAVY through its USD 6.5 billion acquisition of Momenta Pharmaceuticals in 2020, with the FcRn inhibitor myasthenia gravis serving as the centerpiece of that deal. The company is aiming for blockbuster status, forecasting peak annual sales of over USD 5 billion for the myasthenia gravis medication. Dive deep into the myasthenia gravis treatment cost @ Generalized Myasthenia Gravis Drug with Copay Myasthenia gravis pipeline possesses some drugs in mid- and late-stage development to be approved in the near future. The expected launch of myasthenia gravis treatment medications, such as Batoclimab (Immunovant Sciences GmbH), Descartes-08 (Cartesian Therapeutics), and others, are expected to further create a positive impact on the myasthenia gravis disease market. Discover which therapies are expected to grab major myasthenia gravis therapeutics market share @ Market Analysis on Myasthenia Gravis Batoclimab is a fully humanized monoclonal antibody designed to inhibit neonatal Fc receptors by blocking the interaction between FcRn and IgG, thereby promoting the breakdown of autoantibodies. It has received Orphan Drug Designation (ODD) from both the US FDA and the EMA. In March 2025, Immunovant announced topline results from its Phase III trial evaluating batoclimab in patients with myasthenia gravis. Descartes-08 represents the first RNA-engineered CAR T-cell (rCAR-T) therapy developed for autoimmune conditions. This therapy uses cytotoxic T-cells programmed to target and eliminate pathogenic plasma cells that produce autoantibodies. Created from a patient's own blood, Descartes-08 has also received ODD from the US FDA for treating myasthenia gravis. In December 2024, Cartesian Therapeutics shared encouraging updated findings from its Phase IIb study of Descartes-08 in myasthenia gravis. Furthermore, in January 2025, the FDA agreed to the company's Phase III AURORA trial design under a Special Protocol Assessment, with trial initiation planned for the first half of 2025. Curious about what the new myasthenia gravis treatments are in development? Visit Myasthenia Gravis Clinical Trials The anticipated launch of these emerging myasthenia gravis treatments are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the myasthenia gravis market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the myasthenia gravis market size in the 7MM is expected to grow from USD 5.2 billion in 2024 at a significant CAGR by 2034. According to DelveInsight's analysis, the myasthenia gravis market growth is anticipated with the introduction of emerging therapies, leading to an expansion in the myasthenia gravis market size throughout the forecast period (2025–2034). This anticipated myasthenia gravis market growth is driven by advancements in treatment options, greater healthcare access, and a rising myasthenia gravis prevalence, which together foster higher demand for innovative and effective therapies. DelveInsight's latest published market report titled Myasthenia Gravis Market Insight, Epidemiology, and Market Forecast – 2034 will help you to discover which myasthenia gravis treatment market leader is going to capture the largest market share. The report provides comprehensive insights into the country-specific myasthenia gravis treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The myasthenia gravis market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM, segmented into: Total Diagnosed Prevalent Cases of Myasthenia Gravis Gender-specific Diagnosed Prevalent Cases of Myasthenia Gravis Age-specific Diagnosed Prevalent Cases of Myasthenia Gravis Diagnosed Prevalent Cases of Myasthenia Gravis by MGFA Classification Diagnosed Prevalent Cases of Generalized Myasthenia Gravis by Antibody Serology The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM myasthenia gravis market. Highlights include: 10-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this myasthenia gravis market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the myasthenia gravis market. Also, stay abreast of the mitigating factors to improve your market position in the myasthenia gravis therapeutic space. Related Reports Myasthenia Gravis Epidemiology Forecast Myasthenia Gravis Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted myasthenia gravis epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Myasthenia Gravis Pipeline Myasthenia Gravis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key myasthenia gravis companies, including Harbour BioMed (Guangzhou) Co. Ltd., Kyverna Therapeutics, Cabaletta Bio, Takeda, Hoffmann-La Roche, Immunovant Sciences GmbH, Regeneron Pharmaceuticals, Novartis Pharmaceuticals, Janssen Research & Development, LLC, Momenta Pharmaceuticals, Inc., Amgen, Dianthus Therapeutics, Cartesian Therapeutics, COUR Pharmaceutical Development Company, Inc., Alexion Pharmaceuticals, Inc., among others. Generalized Myasthenia Gravis Market Generalized Myasthenia Gravis Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key gMG companies including Alexion Pharmaceuticals, Argenx, UCB Biopharma, Argenx-Halozyme Therapeutics, Horizon Therapeutics, Hoffmann-La Roche, Janssen Research & Development, LLC, Immunovant Sciences GmbH, Bristol Myers Squibb, Biogen Inc., Pfizer Inc., Novartis AG, Sanofi S.A., Roche Holding AG, Johnson & Johnson, Takeda Pharmaceutical Company Limited, AbbVie Inc., Eli Lilly and Company, Merck & Co., Inc., GlaxoSmithKline plc, AstraZeneca plc, Amgen Inc., Boehringer Ingelheim International GmbH, Genentech, Inc., Gilead Sciences, Inc., Celgene Corporation, Vertex Pharmaceuticals Incorporated , among others. Generalized Myasthenia Gravis Pipeline Generalized Myasthenia Gravis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key gMG companies, including Biocon, Cartesian Therapeutics, UCB, Momenta Pharmaceuticals, HanAll Biopharma, Roche, Alexion, Novartis, Takeda, BioMarin, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

ImmunotherapyPhase 3Drug ApprovalOrphan DrugClinical Result

100 Deals associated with Zilucoplan

External Link

KEGG	Wiki	ATC	Drug Bank
-	Zilucoplan	-	DB15636

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Myasthenia Gravis	Japan	UCB Japan Co. Ltd.	25 Sep 2023

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Amyotrophic Lateral Sclerosis	Phase 3	United States	Ra Pharmaceuticals, Inc.	29 Jul 2020
COVID-19	Phase 2	Belgium	UCB Pharma GmbH	22 May 2020
Hypoxic respiratory failure	Phase 2	Belgium	UCB Pharma GmbH	22 May 2020
Immune-mediated necrotising myopathy	Phase 2	United States	Ra Pharmaceuticals, Inc.	07 Nov 2019
Immune-mediated necrotising myopathy	Phase 2	France	Ra Pharmaceuticals, Inc.	07 Nov 2019
Immune-mediated necrotising myopathy	Phase 2	Netherlands	Ra Pharmaceuticals, Inc.	07 Nov 2019
Immune-mediated necrotising myopathy	Phase 2	United Kingdom	Ra Pharmaceuticals, Inc.	07 Nov 2019
Necrotizing myopathy	Phase 2	United States	Ra Pharmaceuticals, Inc.	07 Nov 2019
Necrotizing myopathy	Phase 2	France	Ra Pharmaceuticals, Inc.	07 Nov 2019
Necrotizing myopathy	Phase 2	Netherlands	Ra Pharmaceuticals, Inc.	07 Nov 2019

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04225871 (RAISE-XT, Pubmed \| J Neurol)	Phase 3	Myasthenia Gravis anti-acetylcholine receptor antibody-positive	200	Zilucoplan 0.3mg/kg	mqoxfnxhsr(anobogqgyj) = puvlmqqtta zumqgthpbj (wybiqlpont )	Positive	01 Jul 2025
NCT05514873 (MG0017, AAN2025)	Phase 3	Myasthenia Gravis acetylcholine receptor autoantibody-positive	26	Zilucoplan	qfgjdgbppb(wkykvgvdao) = mtfahqfmei mzfpqihgww (wejhilslnl ) View more	Positive	07 Apr 2025
NCT04115293 (RAISE, AAN2025)	Phase 3	Myasthenia Gravis acetylcholine receptor autoantibody-positive	-	Zilucoplan 0.3 mg/kg	ecwhwvmnui(ntgsgzztcr): P-Value = 0.0004	Positive	07 Apr 2025
				Placebo
NCT05514873 (MG0017, CTgov)	Phase 3	Myasthenia Gravis	26	Zilucoplan (Zilucoplan 0.3 mg/kg)	oqjvvaqeda = cbywpfkdri cvxlknynjo (wshkwmthra, kwyrjltkuf - ogojvcvawa) View more	-	30 Mar 2025
				Zilucoplan (Zilucoplan 0.3 mg/kg (Overall Treatment Period))	ailnuzcudi(etoeefdpka) = izqtqzpdyt nggdrrrqkd (qafcwohauq, sbundfbrhc - vycecyuhlq) View more
NCT05514873 (MG0017, Pubmed \| Ther Adv Neurol Disord)	Phase 3	Myasthenia Gravis acetylcholine receptor autoantibody-positive	26	Zilucoplan 0.3 mg/kg	hpqajtwady(uuzguhriuh) = sbosstggmn ghmttiixff (vfqtuokkmt ) View more	Positive	01 Jan 2025
NCT04225871 (RAISE-XT, AAN2024)	Phase 3	Myasthenia Gravis acetylcholine receptor autoantibody-positive (AChR Ab+)	200	Zilucoplan 0.3 mg/kg	agvlzralqk(qikhiqtowb) = ciwcujqwve boukbncdrl (ujrsrgyhvf ) View more	Positive	09 Apr 2024
NCT05514873 (AAN2024)	-	Myasthenia Gravis	8	泽勒普肽	mrdcbvyppx(wgltpjucsn) = fngyjqecdg diqcshmfwq (imgvexaidv ) View more	Positive	07 Mar 2024
NCT04225871 (RAISE-XT, MDA2024) Manual	Phase 3	Myasthenia Gravis acetylcholine receptor autoantibody-positive	200	continued zilucoplan or placebo swtiched to zilucoplanzilucoplan	tckfsirjip(ydgoavgrgg) = dfixscdbxb karzzseycw (xsgvqyluvs ) View more	Positive	03 Mar 2024
				Zilucoplan 0.3 mg/kg	xeydytcngb(wonjmtyvcp) = ubffkgteas jvrjrovjfg (cfmxdiisqt ) View more
NCT04115293 (RAISE, FDA) Manual	Phase 3	Myasthenia Gravis	174	ZILBRYSQ	uxclbwhfcs(txriprzmkd) = qhsglugkki sblafdswpq (zydmchwsns, -5.28 to -3.50) View more	Positive	17 Oct 2023
				Placebo	uxclbwhfcs(txriprzmkd) = mvirvzhlse sblafdswpq (zydmchwsns, -3.17 to -1.43) View more
NCT04382755 (ZILU-COV, CTgov)	Phase 2	Hypoxic respiratory failure \| COVID-19	81	Zilucoplan® (Group A (Active))	hcdtwbighg(qfkbteggne) = prtzoarjvz lpmtrbnpxs (dkewmbzulc, dzsgrochun - nxdwiarzvi) View more	-	14 Sep 2023
				Placebo (Group B (Control))	hcdtwbighg(qfkbteggne) = hcbqzoqifx lpmtrbnpxs (dkewmbzulc, qmddujoksb - kgvocchjhr) View more

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Biosimilar

Competitive landscape of biosimilars in different countries/locations. Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

AI Agents Built for Biopharma Breakthroughs

Accelerate discovery. Empower decisions. Transform outcomes.

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis