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Cited by 295Myasthenia Gravis and ThymomaPeripheral Neuropathies and DisordersAutoimmune Bullous Skin Diseases

Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study

Bril, Vera, Drużdż, Artur, Grosskreutz, Julian, Habib, Ali A, Mantegazza, Renato, Sacconi, Sabrina, Utsugisawa, Kimiaki, Vissing, John +283 more·2023·The Lancet Neurology
DOI 10.1016/s1474-4422(23)00077-7
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Abstract

Background Generalised myasthenia gravis is a chronic, unpredictable, and debilitating autoimmune disease. New treatments for this disease are needed because conventional therapies have limitations, such as side-effects (eg, increased infection risk) or inadequate control of symptoms. Rozanolixizumab is a neonatal Fc receptor blocker that might provide a novel therapeutic option for myasthenia gravis. We aimed to assess the safety and efficacy of rozanolixizumab for generalised myasthenia gravis. Methods MycarinG is a randomised, double-blind, placebo-controlled, adaptive phase 3 study done at 81 outpatient centres and hospitals in Asia, Europe, and North America. We enrolled patients (aged ≥18 years) with acetylcholine receptor (AChR) or muscle-specific kinase (MuSK) autoantibody-positive generalised myasthenia gravis (Myasthenia Gravis Foundation of America class II-IVa), a Myasthenia Gravis Activities of Daily Living (MG-ADL) score of at least 3 (non-ocular symptoms), and a quantitative myasthenia gravis score of at least 11. Patients were randomly assigned (1:1:1) to receive subcutaneous infusions once a week for 6 weeks of either rozanolixizumab 7 mg/kg, rozanolixizumab 10 mg/kg, or placebo. Randomisation was stratified by AChR and MuSK autoantibody status. Investigators, patients, and people assessing outcomes were masked to random assignments. The primary efficacy endpoint was change from baseline to day 43 in MG-ADL score, assessed in the intention-to-treat population…

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Crossref · 10.1016/s1474-4422(23)00077-7

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  • These characteristics of rozanolixizumab may explain its fast onset of action and sustained maintenance of pharmacodynamic effects (IgG reduction) in the MG patient population.

    “These characteristics of rozanolixizumab may explain its fast onset of action and sustained maintenance of pharmacodynamic effects (i.e., IgG reduction) in the MG patient population [24].”

    From The uptake, intracellular trafficking and recycling of FcRn-blocking therapeutics in human endothelial cells in vitro (2026) · cites this paper as [24]

  • For rozanolixizumab, rapid reductions in mean maximum total IgG levels were observed at Day 8 and sustained to Day 43, with a reduction of approximately 70% for both rozanolixizumab doses studied.

    “For rozanolixizumab, rapid reductions in mean maximum total IgG levels were observed at Day 8 and sustained to Day 43, with a reduction of approximately 70% for both rozanolixizumab doses studied [24].”

    From The uptake, intracellular trafficking and recycling of FcRn-blocking therapeutics in human endothelial cells in vitro (2026) · cites this paper as [24]

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Bril, V., Drużdż, A., Grosskreutz, J., Habib, A. A., Mantegazza, R., Sacconi, S., Utsugisawa, K., Vissing, J., Vu, T., Boehnlein, M., Bozorg, A., Gayfieva, M., Greve, B., Woltering, F., Kaminski, H. J., Genge, A., Massie, R., Berube, M., Bril, V., Daniyal, L., Mannan, S., Ng, E., Raman, R. R. R., Sarpong, E., Alcantara, M., Dionne, A., Siddiqi, Z., Blackmore, D., Hussain, F., Matte, G., Botez, S., Tyblova, M., Jakubikova, M., Junkerova, J., Vissing, J., Witting, N., Holm-Yildiz, S., Stemmerik, M., Andersen, H., Obál, I., Solé, G., Mathis, S., Violleau, M.-H., Tranchant, C., Messai, S., Chanson, J.-B., Nadaj-Pakleza, A., Verloes, A., Zaidi, L., Sacconi, S., Gambella, M., Cavalli, M., Stojkovic, T., Demeret, S., Le Guennec, L., Querin, G., Weiss, N., Masingue, M., Magy, L., Ghorab, K., Rukhadze, I., Tsiskaridze, A., Janelidze, M., Margania, T., Then Bergh, F., Hänsel, E., Kalb, A., Meilick, B., Reuschel, M., Teußer, L.-M., Unterlauft, A., Goedel, C., Hagenacker, T., Totzeck, A., Stolte, B., Blaes, F., Bindler, C., Tsoutsikas, V., Roediger, A., Geis, C., Schmidt, J., Zschüntzsch, J., Schwarz, M., Meyer, S., Kummer, K., Glaubitz, S., Zeng, R., Wiendl, H., Klotz, L., Lammerskitten, A., Lünemann, J., Diószeghy, P., Mantegazza, R., Maggi, L., Rinaldi, E., Gastaldi, M., Mazzacane, F., Businaro, P., Iorio, R., Antonini, G., Fionda, L., Rinaldi, R., Rossi, S., Habetswallner, F., Tuccillo, F., Umehara, H., Uenaka, E., Takahashi, M., Higashi, K., Kinoshita, M., Yoneda, E., Nakamura, N., Fujita, S., Kubota, T., Ono, M., Yamamoto, S., Hatano, T., Oikoshi, K., Yokoyama, K., Oji, Y., Tomizawa, Y., Uzawa, A., Yasuda, M., Akita, S., Ozawa, Y., Onishi, Y., Takaki, M., Yamada, H., Minemoto, K., Sanko, M., Izawa, N., Nakayama, M., Masuda, M., Tsuji, R., Ido, N., Hyodo, Y., Okubo, Y., Minohara, A., Haraguchi, N., Naito, M., Yoshida, S., Fukushige, Y., Tsujino, A., Nagaoka, A., Miyazaki, T., Yoshimura, S., Hirayama, T., Shima, T., Okamoto, N., Matsumoto, R., Sekiguchi, K., Ueda, T., Chihara, N., Kirimura, M., Sunagawa, E., Suzuki, A., Suzuki, S., Wada, A., Ishizuchi, K., Suzuki, Y., Yata, M., Komatsu, Y., Tsukita, K., Watanabe, G., Sato, K., Kawasaki, E., Yamamoto, N., Ono, H., Tsuda, T., Ohashi, S., Utsugisawa, K., Fujisawa, Y., Yokota, Y., Nagane, Y., Ayumi, K., Takematsu, Y., Naito, H., Sugimoto, T., Kuwada, K., Rejdak, K., Szklener, S., Kitowska, M., Derkacz, K., Druzdz, A., Berkowicz, T., Budzinska, P., Halas, M., Zaslavskiy, L., Skornyakova, E., Kotov, S., Novikova, E., Sidorova, O., Goldobin, V., Alekseeva, T., Isabekova, P., Malkova, N., Korobko, D., Djordjevic, G., Stojanov, A., Peric, S., Lavrnic, D., Bozovic, I., Palibrk, A., Casasnovas, C., Nedkova-Hristova, V., Vidal Fernández, N., Cortés Vicente, E., Querol Gutiérrez, L., Salvadó Figueras, M., Canovas Segura, A., Juntas Morales, R., Sanchez Tejerina, D., Saiz, A., Blanco Morgado, Y., Llufriú Durán, S., Sepúlveda Gázquez, M., Martínez Hernández, E. M., Gutiérrez Gutiérrez, G., Iniesta, P., Meca Lallana, J., Guo, Y.-C., Chiu, H.-C., Yeh, J.-H., Chen, Y. H., Lee, M. F., Lee, Y.-C., Lai, K. L., Beydoun, S., Akhter, S., Vu, T., Lam, L., Thomas, A., Rivner, M., Quarles, B., Lange, D., Holzberg, S., Pavlakis, P., Goutham, A., Kaminski, H., Aly, R., Ashworth, L., Bender, K., Bond, K., Buckner, J., Byerly, S., Caress, J., Clemons, J., Farmer, A., Franklin, C., Harris, S., Hiatt, M., Gandhi Mehta, R., Miller, G., Smith, L., Smith, R., Strittmatter, B., Mozaffar, T., Habib, A. A., Hernandez, I., Moulton, K., Karam, C., Ravikumar, P., Lomen-Hoerth, C., Rosow, L., George, H., Irodenko, V., Kang, M., Denny, C., Hanson, B., Klein, S., Martinez-Thompson, J., Naddaf, E., Padgett, D., Sorenson, E., L Sultze, J., Weis, D., Rezania, K., Thonhoff, J., Shroff, S., Pascuzzi, R., Micheels, A., Bodkin, C., Comer, A., Baras, G., Wagner, R., Mahuwala, Z., Ryan, S., Su, K., Sharma, K., Brown, A., & Liow, K.
Affiliations
  • University of Lübeck (DE)
  • UCB Pharma (Germany) (DE)
  • UCB Pharma (United States) (US)
  • UCB Pharma (United Kingdom) (GB)
  • George Washington University (US)
  • General Hanamaki Hospital (JP)
ORCID
Venue
The Lancet Neurology
Publisher
Elsevier BV
Published
2023-05
Volume
22
Issue
5
Pages
383-394
ISSN
1474-4422 (ISSN-L)
DOI
10.1016/s1474-4422(23)00077-7
OpenAlex
W4365457148
Cited by
295
References
0
Citations by year
2026: 85 · 2025: 94 · 2024: 87 · 2023: 28
FWCI
49.53
Type
journal article
Language
en
Update notices
erratum (2023-10-01)
Open access
closed
Licence
Locations
  • The Lancet Neurology (publishedVersion)
  • PubMed (publishedVersion)
  • IRIS Research product catalog (Sapienza University of Rome) (submittedVersion)
  • Research at the University of Copenhagen (University of Copenhagen) (submittedVersion)
  • VBN Forskningsportal (Aalborg Universitet) (submittedVersion)
Funders
UCB Pharma
Topics
  • Health Sciences › Medicine › Neurology › Myasthenia Gravis and Thymoma
  • Health Sciences › Medicine › Neurology › Peripheral Neuropathies and Disorders
  • Health Sciences › Medicine › Pathology and Forensic Medicine › Autoimmune Bullous Skin Diseases
Keywords
generalized myasthenia gravis, rozanolixizumab, acetylcholine receptor antibodies, MG-ADL score, treatment-emergent adverse events, drug safety, autoimmune disease therapy, subcutaneous infusion, adaptive trial design, open-label extension
MeSH terms
Activities of Daily Living, Adolescent, Adult, Autoantibodies, Double-Blind Method, Humans, Infant, Newborn, Myasthenia Gravis, Receptors, Cholinergic, Treatment Outcome, Antibodies, Monoclonal, Humanized

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