An Open-label, Single-arm Study Evaluating the Activity, Safety, and Pharmacokinetics of Rozanolixizumab in Pediatric Study Participants With Moderate to Severe Generalized Myasthenia Gravis

The purpose of the study is to assess the safety and tolerability of subcutaneous (sc) administration of rozanolixizumab in pediatric participants aged ≥2 to <18 years with generalized Myasthenia Gravis (gMG).

开始日期2024-05-15

申办/合作机构

UCB Biopharma SRL

NCT05681715 / Active, not recruiting临床3期

An Open-label, Crossover Study to Evaluate Rozanolixizumab Self-administration by Study Participants With Generalized Myasthenia Gravis

The purpose of this study is to evaluate the ability of study participants with generalized Myasthenia Gravis (gMG) to successfully self-administer rozanolixizumab after training in the self-administration technique using the syringe driver and manual push methods.

开始日期2023-04-17

申办/合作机构

UCB Biopharma SRL

NCT05643794 / Active, not recruiting临床2期

A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Phase 2A, Proof-Of-Concept Study to Evaluate the Efficacy and Safety of Rozanolixizumab to Treat Adult Study Participants With Severe Fibromyalgia Syndrome

The purpose of the study is to evaluate efficacy and safety of rozanolixizumab to treat adult study participants with severe fibromyalgia syndrome (FMS).

开始日期2022-12-21

申办/合作机构

UCB Biopharma SRL

100 项与罗扎诺利珠单抗相关的临床结果

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100 项与罗扎诺利珠单抗相关的转化医学

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100 项与罗扎诺利珠单抗相关的专利（医药）

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项与罗扎诺利珠单抗相关的文献（医药）

2024-01-11·CNS drugs

Myasthenia Gravis Treatment: From Old Drugs to Innovative Therapies with a Glimpse into the Future.

Review

作者: Salvatore Crisafulli ; Brigida Boccanegra ; Massimo Carollo ; Emanuela Bottani ; Paola Mantuano ; Gianluca Trifirò ; Annamaria De Luca

Myasthenia gravis (MG) is a rare autoimmune disease that causes debilitating muscle weakness due to impaired neuromuscular transmission. Since most (about 80-90%) MG patients present autoantibodies against the acetylcholine receptor, standard medical therapy consists of symptomatic treatment with acetylcholinesterase inhibitors (e.g., pyridostigmine). In addition, considering the autoimmune basis of MG, standard therapy includes immunomodulating agents, such as corticosteroids, azathioprine, cyclosporine A, and cyclophosphamide. New strategies have been proposed for the treatment of MG and include complement blockade (i.e., eculizumab, ravulizumab, and zilucoplan) and neonatal Fc receptor antagonism (i.e., efgartigimod and rozanolixizumab). The aim of this review is to provide a detailed overview of the pre- and post-marketing evidence on the five pharmacological treatments most recently approved for the treatment of MG, by identifying both preclinical and clinical studies registered in clinicaltrials.gov. A description of the molecules currently under evaluation for the treatment of MG is also provided.

2023-12-01·Journal of clinical neuromuscular disease

What Is in the Neuromuscular Junction Literature?

Article

作者: David Lacomis

ABSTRACT:

This update covers several articles on diagnosis and misdiagnosis of myasthenia gravis (MG), the role of complement in MG, and then an impressive number of recent treatment trials. There is a negative study on any corticosteroid-sparing effect of intravenous immunoglobulin. A number of positive studies are reviewed. Open-label extension studies of phase 3 trials showed benefit regarding quality of life with efgartigimod and in functional measures with ravulizumab. The phase 3 RAISE trial of zilucoplan, a self-administered complement C5 inhibitor, is covered as well as the MyCarinG trial of rozanolixizumab. The notion of using fast-acting therapies early in the course of MG is addressed. The last sections center on MG and Lambert-Eaton myasthenic syndrome as a consequence of immune checkpoint inhibitor therapy.

2023-09-01·Drugs

Rozanolixizumab: First Approval.

Review

作者: Sheridan M Hoy

Rozanolixizumab (rozanolixizumab-noli; RYSTIGGO^®) is a high affinity humanized immunoglobulin G4 monoclonal antibody directed against human neonatal Fc receptor (FcRn). Administered subcutaneously, it is being developed by UCB Pharma for the treatment of autoimmune diseases and received its first approval on 27 June 2023 in the USA for the treatment of generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) or anti-muscle-specific kinase (MuSK) antibody positive. Rozanolixizumab is the first agent to be approved in the USA for both anti-AChR and anti-MuSK antibody-positive gMG. A regulatory assessment of rozanolixizumab for the treatment of gMG is currently underway in the EU and Japan. Clinical development is ongoing for the treatment of leucine-rich glioma-inactivated 1 autoimmune encephalitis, myelin oligodendrocyte glycoprotein (MOG) antibody disease and severe fibromyalgia syndrome. This article summarizes the milestones in the development of rozanolixizumab leading to this first approval for the treatment of gMG in adults who are anti-AChR or anti-MuSK antibody positive.

项与罗扎诺利珠单抗相关的新闻（医药）

2024-04-02

·FierceBiotech

J&J targets rare maternal-fetal diseases: 'We're going for this. No one else has'

Last week, the FDA granted fast track status for Johnson & Johnson’s investigational monoclonal antibody nipocalimab in the rare blood disorder FNAIT. Just once in history has a drug been approved in maternal fetal immunology, specifically in hemolytic disease of the fetus and newborn (HDFN). “And it was approved in 1968," says Katie Abouzahr, M.D., Johnson & Johnson’s vice president and autoantibody and maternal fetal immunology disease area leader, in an interview with Fierce Biotech. “That program was revolutionary." The drug is Rh immunoglobulin (RhoGAM), a medication used to manage and treat Rh-negative pregnancies, which is when the mother’s immune system treats Rh-positive fetal cells as if they were foreign cells and destroys them. The blood product is delivered by intramuscular injection to prevent individuals from forming the alloantibodies that cause HDFN. RhoGAM was originally discovered by J&J and now sold by Kedrion. Now, more than 50 years after RhoGAM’s approval, the J&J leader is hoping to build off the pharma’s legacy and bring another maternal fetal immunology treatment to market. “The unmet need is unequivocal,” Abouzahr said. Last week, the FDA granted fast track status for J&J’s investigational monoclonal antibody (mAb) nipocalimab in another rare blood disorder called fetal and neonatal alloimmune thrombocytopenia (FNAIT). According to J&J, nipocalimab is the only investigational therapy currently in clinical development to treat the potentially life-threatening condition in which a pregnant person's immune system attacks fetal platelets and subsequently runs the risk of severe bleeding complications for the baby. The fast track designation follows a phase 2 proof-of-concept data drop for nipocalimab in HDFN, an alloimmune disease of pregnancy that has a similar disease mechanism to FNAIT. The mAb is designed to block IgG binding to FcRn in the placenta and prevent transfer of maternal alloantibodies to the fetus. The investigational therapy also previously snagged orphan drug designation in FNAIT, an indication in which no approved therapies specifically designed to solely manage the condition exist. The freshly obtained fast-track status is for alloimmunized pregnant people at risk of FNAIT. The agency’s tag is made to speed up timelines related to drug development and the review process. “These are desperate situations,” Abouzahr said, explaining that severe cases of FNAIT can lead to intracranial hemorrhage and even death for the newborn. “It's rare but it's cataclysmic for these families.” FNAIT often occurs in a person’s first pregnancy and there isn’t any routine screening conducted for the alloantibodies that cause the condition, Abouzahr said. That means diagnoses aren’t delivered until a FNAIT pregnancy or birth occurs. So, what options do patients have? “If you've got access to a good center—a specialized center that can handle this—treatment can include intravenous immunoglobulin, so IVIG with or without steroids, because you're trying to suppress this immune response,” Abouzahr said. But IVIG comes with several challenges. “IVIG hasn't been studied for approvals. There are no clinical trials; people use all different doses because there's no sort of agreement around it,” the J&J leader explained. The pooled antibody treatment has black box warnings—the FDA’s warning to consumers that a product could cause serious adverse reactions that could lead to severe injury or even death. The Big Pharma is currently testing the mAb among alloimmunized pregnant individuals with FNAIT in a pivotal phase 3 program, according to the company. More information on the global FREESIA trial will be available in the coming weeks when the study’s listing will go up on ClinicalTrials.gov, according to Abouzahr. But J&J isn’t limiting nipocalimab just to maternal/fetal diseases. The New Jersey-based pharma giant believes the investigational therapy could hold the answer for dozens of conditions. “This FcRn blocking approach has the potential to affect any autoantibody-driven disease—there are maybe around 80,” Abouzahr said. J&J splits up these diseases across three segments: rare autoantibody, maternal/fetal and prevalent rheumatology diseases. The company is currently evaluating nipocalimab in a handful of trials that fall under those three umbrellas, including generalized myasthenia gravis, chronic inflammatory demyelinating polyneuropathy, warm autoimmune hemolytic anemia, idiopathic inflammatory myopathies, rheumatoid arthritis, Sjögren's disease and systemic lupus erythematosus. We're going for this. No one else has. Yes, it is more challenging. There's no trodden path that we can follow for regulatory approval." — Katie Abouzahr, M.D. J&J acquired nipocalimab back in 2020 when Momenta Pharmaceuticals was bought out for $6.5 billion. The deal secured J&J a challenger to now-approved drugs from Argenx (Vyvgart for generalized myasthenia gravis) and UCB (Rystiggo also in myasthenia gravis). While other companies have gone after FcRn blocking, no one else has publicly disclosed any attempts in the fetal/maternal category. “We're going for this. No one else has,” Abouzahr said. “Yes, it is more challenging. We're studying two patients every time. There's no trodden path that we can follow for regulatory approval. And it's an incredibly important and delicate area that we want to approach with the care, the respect, the thought that it requires. But I almost feel like we have a duty to go forward, given we have an investigational therapy that could potentially be transformative.” Editor's note: This article was updated at 1:30 p.m. ET on April 2 to clarify RhoGAM's current therapeutic uses.

免疫疗法快速通道临床2期上市批准孤儿药

2024-03-20

·FiercePharma

#FierceMadness is back: Get voting in the 2024 drug name tournament

This year we’re taking the branded drug names that were officially approved by the FDA in 2022 and 2023 - now it's your turn to vote for your winner. LAUNCHED: Wednesday, March 20, 7:00 a.m. ET It’s March, so it’s time for some #FierceMadness. Last year, we channeled the NCAA tournament for the best pharma ad campaigns, but, this year, we’re looking for the best of the best in drug names. Here’s how it works. We’ve chosen 64 of the most distinctive drug names to come out of the FDA’s 2022 and 2023 crop of new approvals. Whittling down to 64 from the 92 drug names available was our staff challenge, but now it’s up to you, readers, to deliberate and vote down to the final winner. Remember, this isn’t about the drugs themselves—neither how well they may work (or not) nor any controversies in how they may have been approved. Our goal is to assess their marketing suitability, considering how well they perform in their respective field and against competitors. Ready to play? Check out the bracket here (PDF) and see the full Madness 64 list below. Then vote is via our poll here. You can vote from now until Friday, March 22, at 6 p.m. ET. Check back here Monday, March 25, for your results, and the chance to vote in the next round. After that, we’ll be back with a new round each Wednesday and Monday through Monday, April 8, when you can vote for your championship winner. Any remote robo-voting will be noted, and those votes will be discarded. Please play fair. Don’t forget to tell us why you picked what you picked in the voting poll comments section—and what you think each drug name really sounds like. We’ll include the funniest and most out-of-the-box comments in our recaps every round, so make 'em good! New this year: Our sister publication Fierce Biotech is also running its own Madness tournament for the best of the best in biotech company names. Check out and vote in its championship here. Good luck, and happy Madness! #FierceMadness 2024: The full bracket of 64 Jesduvroq vs. Lamzede Jesduvroq Maker: GSK Indication: Anemia due to chronic kidney disease Sounds like: When you hit your finger with a hammer, but it’s in front of your children so you’re trying not to swear Lamzede Maker: Chiesi Indication: Non-central nervous system manifestations of alpha-mannosidosis Sounds like: A vegan meal Pluvicto vs. Lunsumio Pluvicto Maker: Novartis Indication: Prostate-specific membrane antigen-positive metastatic castration-resistant prostate cancer following other therapies Sounds like: A new planet Lunsumio Maker: Roche Indication: Relapsed or refractory follicular lymphoma, a type of non-Hodgkin lymphoma Sounds like: A Japanese wrestler Briumvi vs. Camzyos Briumvi Maker: TG Therapeutics Indication: Relapsing forms of multiple sclerosis Sounds like: A sixth-generation cheese Camzyos Maker: Bristol Myers Squibb Indication: Obstructive hypertrophic cardiomyopathy Sounds like: A Spanish holiday resort Voquezna vs. Mounjaro Voquezna Maker: Phathom Pharmaceuticals Indication: Helicobacter pylori infection Sounds like: A sports drink with a kick Mounjaro Maker: Eli Lilly Indication: Lowering blood sugar levels in Type 2 diabetes Sounds like: A legendary medieval sword fighter Vtama vs. Amvuttra Vtama Maker: Dermavant Indication: Plaque psoriasis Sounds like: A mouthful Amvuttra Maker: Alnylam Indication: Polyneuropathy of hereditary transthyretin-mediated amyloidosis Sounds like: A pope’s gift Xenpozyme vs. Daxxify Xenpozyme Maker: Sanofi Indication: Acid sphingomyelinase deficiency Sounds like: An alien protein Daxxify Maker: Revance Indication: The treatment of cervical dystonia and for the temporary improvement of frown lines Sounds like: A ray gun to make you sassy Fruzaqla vs. Sotyktu Fruzaqla Maker: Takeda Pharmaceuticals Indication: Refractory, metastatic colorectal cancer Sounds like: A European swear word Sotyktu Maker: Bristol Myers Squibb Indication: Moderate to severe plaque psoriasis Sounds like: Gesundheit! Terlivaz vs. Imjudo Terlivaz Maker: Mallinckrodt Pharmaceuticals Indication: Improve kidney function in adults with hepatorenal syndrome with rapid reduction in kidney function Sounds like: A Hungarian dance Imjudo Maker: AstraZeneca Indication: Unresectable hepatocellular carcinoma Sounds like: A self-confident martial artist Elahere vs. Vabysmo Elahere Maker: ImmunoGen Indication: Recurrent ovarian cancer that is resistant to platinum therapy Sounds like: An inebriated attempt at Hawaiian Vabysmo Maker: Roche Indication: Neovascular (wet) aged-related macular degeneration and diabetic macular edema Sounds like: A new way of saying fantastic Pyrukynd vs. Rystiggo Pyrukynd Maker: Agios Indication: Hemolytic anemia in pyruvate kinase deficiency Sounds like: A drunk Scrabble game Rystiggo Maker: UCB Indication: Generalized myasthenia gravis in adults who are anti-acetylcholine receptor- or anti-muscle-specific tyrosine kinase antibody-positive Sounds like: An Italian village Beyfortus vs. Xdemvy Beyfortus Maker: AstraZeneca Indication: To prevent respiratory syncytial virus lower respiratory tract disease Sounds like: A medieval stronghold Xdemvy Maker: Tarsus Pharmaceuticals Indication: Demodex blepharitis Sounds like: Elon Musk’s drunk attempt at renaming his business Tzield vs. Rezlidhia Tzield Maker: Provention Bio Indication: Delay the onset of stage 3 Type 1 diabetes Sounds like: An electronic barrier Rezlidhia Maker: Rigel Pharmaceuticals Indication: Relapsed or refractory acute myeloid leukemia with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation Sounds like: A new Disney princess Krazati vs. Leqembi Krazati Maker: Mirati Therapeutics Indication: KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer in adults who have received at least one prior systemic therapy Sounds like: A crazy party Leqembi Maker: Eisai Indication: Alzheimer’s disease Sounds like: An inspirational French aphorism Cibinqo vs. Brenzavvy Cibinqo Maker: Pfizer Indication: Refractory, moderate to severe atopic dermatitis Sounds like: A game of cheeky bingo Brenzavvy Maker: TheracosBio Indication: Glycemic control in adults with Type 2 diabetes mellitus as an adjunct to diet and exercise Sounds like: Heard in a copy of "Pirates of the Caribbean" Jaypirca vs. Skyclarys Jaypirca Maker: Eli Lilly Indication: Relapsed or refractory mantle cell lymphoma in adults who have had at least two lines of systemic therapy, including a BTK inhibitor Sounds like: A strong coffee Skyclarys Maker: Biogen Indication: Friedreich’s ataxia Sounds like: A high-rise building Zavzpret vs. Paxlovid Zavzpret Maker: Pfizer Indication: Migraine treatment Sounds like: A Turkish fast-food restaurant Paxlovid Maker: Pfizer Indication: Mild to moderate COVID-19 in adults at high risk for progression to severe COVID-19 Sounds like: A bizarre Pac-Man sequel Inpefa vs. Columvi Inpefa Maker: Lexicon Pharmaceuticals Indication: Heart failure Sounds like: An anti-fascist offshoot group Columvi Maker: Roche Indication: Diffuse large B-cell lymphoma, not otherwise specified, or large B-cell lymphoma arising from follicular lymphoma after two or more lines of systemic therapy Sounds like: An ancient Roman detective Daybue vs. Zynyz Daybue Maker: Acadia Indication: Rett syndrome Sounds like: How my five-year-old insists debut is spelled Zynyz Maker: Incyte Indication: Metastatic or recurrent locally advanced Merkel cell carcinoma Sounds like: Drug naming companies may be running out of ideas Rezzayo vs. Qalsody Rezzayo Maker: Melinta Therapeutics Indication: Candidemia and invasive candidiasis Sounds like: A phrase used when you’ve had a great nap in the day Qalsody Maker: Biogen Indication: Amyotrophic lateral sclerosis in adults who have a SOD1 gene mutation Sounds like: From the makers of “Better Call Saul” comes “better Qalsody” Elfabrio vs. Veozah Elfabrio Maker: Chiesi Indication: Fabry disease Sounds like: The lost Spanish Mario brother Veozah Maker: Astellas Indication: Moderate to severe hot flashes caused by menopause Sounds like: A Broadway play Epkinly vs. Zurzuvae Epkinly Makers: AbbVie/Genmab Indication: Relapsed or refractory diffuse large B-cell lymphoma and high-grade B-cell lymphoma after two or more lines of systemic therapy Sounds like: Someone who watches too many streaming episodes Zurzuvae Makers: Sage/Biogen Indication: Postpartum depression Sounds like: A mouthful Izervay vs. Talvey Izervay Maker: Iveric Bio Indication: Geographic atrophy secondary to age-related macular degeneration Sounds like: A new app for scanning the land Talvey Maker: Johnson & Johnson Indication: Relapsed or refractory multiple myeloma who have received at least four prior therapies Sounds like: A little too close to talc… Elrexfio vs. Veopoz Elrexfio Maker: Pfizer Indication: Relapsed or refractory multiple myeloma who have received at least four prior lines of therapy Sounds like: A new dinosaur found in Mexico Veopoz Maker: Regeneron Pharmaceuticals Indication: Patients 1 year old and older with CD55-deficient protein-losing enteropathy Sounds like: Double-plus good! Zilbrysq vs. Ojjaara Zilbrysq Maker: UCB Indication: Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive Sounds like: Absolute zero Ojjaara Maker: GSK Indication: Intermediate or high-risk myelofibrosis in adults with anemia Sounds like: “What’s my name?” Exxua vs. Pombiliti Exxua Maker: Fabre-Kramer Pharmaceuticals Indication: Major depressive disorder Sounds like: Someone who’s always leaving Pombiliti Maker: Amicus Indication: Late-onset Pompe disease Sounds like: The ability to treat Pompe disease (this feels a little on the nose) Rivfloza vs. Velsipity Rivfloza Maker: Novo Nordisk Indication: Urinary oxalate levels in patients 9 years and older with primary hyperoxaluria type 1 and relatively preserved kidney function Sounds like: An exuberant DJ Velsipity Maker: Pfizer Indication: Moderately to severely active ulcerative colitis in adults Sounds like: A deodorant brand you wouldn’t want to buy Bimzelx vs. Omvoh Bimzelx Maker: UCB Indication: Moderate to severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy Sounds like: Throwing away unwanted antacids Omvoh Maker: Eli Lilly Indication: Ulcerative colitis Sounds like: A new age prayer Loqtorzi vs. Defencath Loqtorzi Maker: Coherus Biosciences Indication: Recurrent or metastatic nasopharyngeal carcinoma when used together with or following other therapies Sounds like: How you close the door in a magical kingdom Defencath Maker: CorMedix Indication: Reduce the incidence of catheter-related bloodstream infections in adults with kidney failure receiving chronic hemodialysis Sounds like: A Dungeons and Dragons character Augtyro vs. Ryzneuta Augtyro Maker: Bristol Myers Squibb Indication: ROS1-positive non-small cell lung cancer Sounds like: A virtual reality game where you’re the bad guy Ryzneuta Maker: Acrotech Biopharma Indication: Neutropenia Sounds like: Getting rid of someone’s charisma Truqap vs. Ogsiveo Truqap Maker: AstraZeneca Indication: Breast cancer that meets certain disease criteria Sounds like: A limit on truth Ogsiveo Maker: SpringWorks Indication: Progressing desmoid tumors who require systemic treatment Sounds like: Oh, give us a go! Quviviq vs. Ztalmy Quviviq Maker: Idorsia Indication: Insomnia Sounds like: A producer asking a biotech founder turned politician to get ready for his TV appearance Ztalmy Maker: Marinus Indication: Seizures in cyclin-dependent kinase-like 5 deficiency disorder Sounds like: A nefarious Yevgeny Zamyatin character Opdualag vs. Wainua Opdualag Maker: Bristol Myers Squibb Indication: Unresectable or metastatic melanoma Sounds like: A Russian prison Wainua Maker: AstraZeneca Indication: Polyneuropathy of hereditary transthyretin-mediated amyloidosis Sounds like: A Premier League footballer

2024-03-14

·医药地理

2023年日本批准新药情况

2023年度，日本独立行政法人医药品医疗器械综合机构（PMDA）公布的批准新药数为106个（PMDA对于新药的定义是其有效成分、数量、用法、剂量、功效等与已批准药物有明显区别的药物，主要是含有新活性成分药物、新复方药物、新给药途径药物、新适应症药物、新剂型药物、新剂量药物等）。其中，有新活性成分新药的数量为30个。2023年的新活性成分新药中，疫苗和肿瘤药占总数的1/3，按照PMDA分类批准的各类别的新药情况如下图所示（左边列表审查类别按从多到少排序）。图1：2023各审查类别新药情况来源：Pharma ONE智能药物大数据分析平台，中国医药工业信息中心对比2022年度的新活性成分新药情况，可以看出，2023年度批准的新活性成分新药要比2022年度少，在疫苗、血制品、泌尿药、肛肠药、医疗用复方药类别中，2023年的新药数量比2022年多。图2：新活性成分新药审查类别情况来源：Pharma ONE智能药物大数据分析平台，中国医药工业信息中心在新活性成分新药中，有8个新药由PMDA批准指定为孤儿药。其中，胆酸是ReqMed和CTRS(现Theravia)共同开发用于治疗先天性胆汁酸合成障碍，CTRS社的胆酸曾在2013获得欧盟批准用于治疗先天性胆汁酸合成障碍的3β-HSD缺乏症。UCB开发的罗泽利昔珠单抗用于治疗全身型重症肌无力，是抗FcRn的人源化IgG4单克隆抗体制剂，抑制FcRn介入IgG循环路径，来降低致病性IgG浓度。PMDA批准的新活性成分孤儿药具体品类见下表。表1：2023年PMDA批准的新活性成分孤儿药除了孤儿药外，有3个PMDA优先审批通过的新活性成分新药，如下。表2：2023年PMDA优先审批批准的新活性成分新药2023年度PMDA批准的新活性成分新药中，UCB开发的齐芦克布仑为全球首次批准，用于治疗全身型重症肌无力。Eli Lilly开发的米吉珠单抗为全球首次批准上市，用于治疗现有治疗效果不理想的中度至重度溃疡性结肠炎。Genmab开发的艾可瑞妥单抗为全球首次上市，用于治疗复发或难治性弥漫性大B细胞淋巴瘤和复发或难治性滤泡性淋巴瘤。2023年度PMDA批准的全部新活性成分新药具体如下表所示。表3：2023年PMDA批准的新活性成分新药END如需获取更多数据洞察信息或公众号内容合作，请联系医药地理小助手微信号：pharmadl001

孤儿药上市批准优先审批

100 项与罗扎诺利珠单抗相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
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研发状态

适应症	最高研发状态	国家/地区	公司
重症肌无力	批准上市	欧盟更多	UCB, Inc. 更多
-	申请上市	中国更多	UCB, Inc. 更多
特发性血小板减少性紫癜	临床3期	比利时更多	UCB SA 更多
抗髓鞘少突胶质细胞糖蛋白免疫球蛋白Ｇ抗体相关疾病	临床3期	比利时更多	UCB SA 更多
原发性血小板减少症	临床3期	中国更多	优时比贸易（上海）有限公司更多

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临床结果

适应症

分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03052751 (Pubmed) 人工标引	临床2期	重症肌无力	43	Rozanolixizumab	(構願觸鬱簾構鏇選觸糧) = 淵獵願鏇簾憲獵遞構壓淵襯積簾遞衊憲願膚鹹 (廠構醖餘膚夢鹽繭鬱衊 ) 更多	积极	2021-02-09
				Placebo	(構願觸鬱簾構鏇選觸糧) = 顧鬱顧衊壓衊淵獵構壓淵襯積簾遞衊憲願膚鹹 (廠構醖餘膚夢鹽繭鬱衊 ) 更多
NCT03052751 (S43.001, AAN2019)	临床2期	重症肌无力 IgG autoantibodies \| anti-AChR antibody	43	Rozanolixizumab 7mg/kg	(網獵淵艱構蓋蓋願糧積) = During Period-1, 16/21 (76.2%) and 0/21 patients receiving rozanolixizumab and 16/22 (72.7%) and 2/22 (9.1%) taking placebo reported ≥1 TEAE and SAE, respectively. By D99, 36/43 (83.7%) rozanolixizumab-treated patients reported ≥1 TEAE, and 5/43 (11.6%) reported ≥1 SAE; no deaths occurred. As expected, headache was more frequent (57.1%) versus placebo (13.6%) (Period-1); all were manageable and resolved with standard therapies. 齋衊醖遞鬱築鬱壓鹽繭 (願餘鏇構憲淵觸鏇獵繭 ) 更多	积极	2019-04-09
				Placebo
NCT03971422 (CTgov)	临床3期	重症肌无力	200	Placebo	築艱衊鏇糧艱齋繭鑰遞(觸構範網選觸衊鹽構鏇) = 選壓夢蓋膚築觸夢鹽選觸淵蓋壓餘鑰鬱鑰窪艱 (醖選積簾觸憲窪繭製夢, 簾膚淵蓋積築製鏇網衊 ~ 選醖醖構鏇網艱鏇範醖) 更多	-	2023-08-21
NCT02718716 (EHA2019)	临床2期	特发性血小板减少性紫癜	66	4 mg/kg rozanolixizumab	積壓夢糧網蓋齋窪窪壓(齋醖觸齋範鏇壓艱衊顧) = 膚觸遞選簾繭鹽窪醖遞製艱廠窪觸齋範齋鑰餘 (憲繭窪鹽鏇夢艱積鹽夢, 21.9 ~ 68.6)	-	2019-06-15
				7 mg/kg rozanolixizumab	積壓夢糧網蓋齋窪窪壓(齋醖觸齋範鏇壓艱衊顧) = 鹹構淵網簾築壓簾選廠製艱廠窪觸齋範齋鑰餘 (憲繭窪鹽鏇夢艱積鹽夢, 29.5 ~ 65.5)
NCT02220153 (Pubmed \| Sci Transl Med) 人工标引	临床1期	-	49	Rozanolixizumab	-	积极	2017-11-01
				Placebo
NCT03971422 (MycarinG, MDA2023) 人工标引	临床3期	重症肌无力 MuSK Ab Positive \| AChR Ab Positive	200	Rozanolixizumab 7mg/kg	(網遞鏇簾憲築蓋選鬱蓋) = 願糧衊網範鏇膚構築廠艱衊襯鑰艱觸憲鹹鬱繭 (簾襯憲鹹衊蓋衊築選壓 )	积极	2023-03-19
				Rozanolixizumab 10mg/kg	(網遞鏇簾憲築蓋選鬱蓋) = 襯鏇廠獵窪鹽積簾窪網艱衊襯鑰艱觸憲鹹鬱繭 (簾襯憲鹹衊蓋衊築選壓 )
NCT03971422 (FDA) 人工标引	临床3期	重症肌无力	200	RYSTIGGO (7 mg/kg)	(築築觸廠選選網廠蓋獵) = 繭鹽鬱鬱範蓋餘廠積憲網獵鹽壓繭鑰衊製選製 (壓構網壓襯範鬱膚觸鬱, 0.5) 更多	积极	2023-06-26
				RYSTIGGO (10 mg/kg)	(築築觸廠選選網廠蓋獵) = 壓選膚鑰餘築積廠顧顧網獵鹽壓繭鑰衊製選製 (壓構網壓襯範鬱膚觸鬱, 0.5) 更多
NCT04596995 (CTgov)	临床3期	特发性血小板减少性紫癜	43	Rozanolixizumab	艱製淵鑰網醖鹹簾鏇鑰(網觸積壓鏇廠積獵糧壓) = 壓網顧襯遞膚選鹹鑰膚鑰遞廠膚製艱鹹醖淵糧 (鑰淵鏇餘廠糧簾廠糧衊, 襯餘蓋願積衊繭淵範醖 ~ 壓蓋遞範製憲糧夢製憲) 更多	-	2024-02-05
NCT02718716 (CTgov)	临床2期	特发性血小板减少性紫癜	66	UCB7665 (UCB7665 4 mg/kg (SS))	選窪獵獵壓網選積鑰糧(鹽醖選選積醖顧簾積糧) = 憲壓蓋鑰積膚憲選鹽範鏇窪鏇製艱範鏇鏇淵窪 (衊齋齋繭鑰襯範糧製廠, 構鹽糧築範遞選顧憲選 ~ 糧廠餘艱壓網繭衊網蓋) 更多	-	2024-01-29
				UCB7665 (UCB7665 7 mg/kg (SS))	選窪獵獵壓網選積鑰糧(鹽醖選選積醖顧簾積糧) = 繭憲壓積範糧繭顧窪鏇鏇窪鏇製艱範鏇鏇淵窪 (衊齋齋繭鑰襯範糧製廠, 鏇構餘鹽鬱觸餘築製膚 ~ 糧鹽襯製顧餘簾蓋窪齋) 更多
NCT02718716 (Pubmed \| Blood Adv) 人工标引	临床2期	特发性血小板减少性紫癜	66	Rozanolixizumab	(觸夢窪顧範膚構鑰糧願) = 蓋憲構憲築齋夢壓鬱網簾鬱艱簾廠網網壓憲鹹 (範鹹窪壓構憲壓淵築簾 ) 更多	积极	2020-09-08