Drugs / Zilucoplan
Regulatory milestones approvals, filings & regulatory actions · 5 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed generalized myasthenia gravis 3 milestones | |||||
| Filed | US (FDA) | — | generalized myasthenia gravis | 2022-11-14 | “today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for its investigational treatment, zilucoplan.” ucb.com ↗ “a NDA for zilucoplan – a subcutaneous self-administered peptide inhibitor of complement component 5 (C5 inhibitor) has recently been filed with the U.S. FDA” ucb.com ↗ “the FDA’s acceptance of a New Drug Application (NDA) and the EMA’s validation of the MAA for zilucoplan in the same indication.” ucb.com ↗ “These pivotal Phase 3 trials supported U.S., European, and Japanese regulatory filings of both rozanolixizumab and zilucoplan.” ucb.com ↗ “These two Phase 3 trials supported U.S., EU, and Japanese regulatory filings of both rozanolixizumab-noli and zilucoplan.” ucb.com ↗ |
| Filed | EU (EMA) | — | generalized myasthenia gravis | — | “This acceptance follows the recent European Medicines Agency (EMA) validation of the Marketing Authorization Application (MAA) for zilucoplan” ucb.com ↗ “the FDA’s acceptance of a New Drug Application (NDA) and the EMA’s validation of the MAA for zilucoplan in the same indication.” ucb.com ↗ “These pivotal Phase 3 trials supported U.S., European, and Japanese regulatory filings of both rozanolixizumab and zilucoplan.” ucb.com ↗ “These two Phase 3 trials supported U.S., EU, and Japanese regulatory filings of both rozanolixizumab-noli and zilucoplan.” ucb.com ↗ |
| Filed | Japan (PMDA) | — | generalized myasthenia gravis | — | “These pivotal Phase 3 trials supported U.S., European, and Japanese regulatory filings of both rozanolixizumab and zilucoplan.” ucb.com ↗ “These two Phase 3 trials supported U.S., EU, and Japanese regulatory filings of both rozanolixizumab-noli and zilucoplan.” ucb.com ↗ “Regulatory applications for rozanolixizumab and zilucoplan for the treatment of gMG have also been filed in Japan.” ucb.com ↗ |
| Approved Indication not stated 2 milestones | |||||
| Approved | EU (EMA) | Zilbrysq | — | 2023-12-01 | europa.eu ↗ |
| Approved | US (FDA) | ZILBRYSQ | — | 2023-10-17 | fda.gov ↗ |
Trials 17
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 12 trials · 1 met primary | ||||||
| Phase 1 | NCT06961747 | Jul 2025 → Mar 2027 expected | — | UCB Biopharma SRL | Recruiting | No outcome recorded |
| Phase 1 | NCT06511076 | Aug → Nov 2024 | — | UCB Biopharma SRL | Completed | Met primary |
| Phase 25 trials · 2 missed | ||||||
| Phase 2 | NCT04025632 | Nov 2019 → Mar 2021 | immune-mediated necrotizing myopathy | Ra Pharmaceuticals | Terminated | Missed primary |
| Phase 2 | NCT03315130 | Oct 2017 → Dec 2018 | generalized myasthenia gravis | Ra Pharmaceuticals | Completed | Missed primary |
| Phase 2 | NCT03225287 | Jul 2017 → Sep 2021 | paroxysmal nocturnal hemoglobinuria | Ra Pharmaceuticals | Terminated | No outcome recorded |
| Phase 2 | NCT03030183 | Apr 2017 → Mar 2018 | paroxysmal nocturnal hemoglobinuria | Ra Pharmaceuticals | Completed | No outcome recorded |
| Phase 2 | NCT03078582 | Mar 2017 → Mar 2018 | paroxysmal nocturnal hemoglobinuria | Ra Pharmaceuticals | Completed | No outcome recorded |
| Phase 2/33 trials · 2 missed | ||||||
| Phase 2/3 | NCT06055959 | Oct 2024 → Nov 2026 expected | generalized myasthenia gravis | UCB Biopharma SRL | Recruiting | No outcome recorded |
| Phase 2/3 | NCT04436497 | Jul 2020 → Apr 2022 | amyotrophic lateral sclerosis | Merit E. Cudkowicz, MD | Completed | Missed primary |
| Phase 2/3 | NCT04297683 | Jun 2020 → Jul 2027 expected | amyotrophic lateral sclerosis | Merit E. Cudkowicz, MD | Recruiting | Missed primary |
| Phase 37 trials · 1 met primary · 1 missed | ||||||
| Phase 3 | NCT07215949 | Jan 2026 → Dec 2027 expected | generalized myasthenia gravis | Miriam Freimer | Recruiting | No outcome recorded |
| Phase 3 | NCT06435312 | Nov 2024 → Oct 2027 expected | generalized myasthenia gravis | UCB Biopharma SRL | Enrolling by invitation | No outcome recorded |
| Phase 3 | NCT06471361 | Aug 2024 → Jan 2025 | generalized myasthenia gravis | UCB Biopharma SRL | Completed | No outcome recorded |
| Phase 3 | NCT05514873 | Oct 2022 → Mar 2024 | generalized myasthenia gravis | UCB Biopharma SRL | Completed | No outcome recorded |
| Phase 3 | NCT04590586 | Nov 2020 → Aug 2021 | COVID-19 | Amgen | Completed | Missed primary |
| Phase 3 | NCT04225871 | Dec 2019 → Jul 2026 overdue | generalized myasthenia gravis | UCB Biopharma SRL | Active not recruiting | No outcome recorded |
| Phase 3 | NCT04115293 | Sep 2019 → Dec 2021 | generalized myasthenia gravis | Ra Pharmaceuticals | Completed | Met primary |
| Comparator or background therapy1 trial | ||||||
| Phase 2 | NCT04382755 Comparator | May → Dec 2020 | COVID-19 | University Hospital, Ghent | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 12
| Date | Issuer | Release |
|---|---|---|
| 2026-07-08 | UCB Pharma | Results UCB presents latest data from its targeted generalized myasthenia gravis treatment portfolio at International Congress on Neuromuscular Diseases 2026 ucb.com ↗
A usability study supports appropriate and effective use of the zilucoplan pre-filled pen in gMG, with 90% of adult participants, including injection‑naïve patients and caregivers, successfully injecting on their first untrained attempt |
| 2026-03-26 | UCB Pharma | Regulatory UCB receives positive CHMP opinion for new ZILBRYSQ[®]▼ (zilucoplan) pre-filled pen in EU for adults living with generalized myasthenia gravis ucb.com ↗ |
| 2024-05-14 | UCB Pharma | Results New data published in the Journal of Neurology show clinically meaningful improvement of fatigue in generalized myasthenia gravis (gMG) with ZILBRYSQ[®]▼ (zilucoplan) ucb.com ↗
The results showed that patients with gMG treated with once daily subcutaneous injections of the complement C5 inhibitor zilucoplan experienced clinically meaningful improvements in fatigue sustained for more than a year. |
| 2023-12-04 | UCB Pharma | Regulatory UCB announces European Commission approval of ZILBRYSQ[®]▼ (zilucoplan) for the treatment of adults with generalized Myasthenia Gravis ucb.com ↗
European approval of ZILBRYSQ ® (zilucoplan) granted as an add-on to standard therapy for the treatment of generalized Myasthenia Gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody-positive1 |
| 2023-10-17 | UCB Pharma | Regulatory UCB announces U.S. FDA approval of ZILBRYSQ[®] (zilucoplan) for the treatment of adults with generalized myasthenia gravis ucb.com ↗
FDA approval of ZILBRYSQ ® (zilucoplan) has been granted for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody-positive |
| 2023-09-25 | UCB Pharma | Regulatory UCB announces approval of RYSTIGGO[®] (rozanolixizumab) and ZILBRYSQ[®] (zilucoplan) for the treatment of adult patients with generalized myasthenia gravis in Japan ucb.com ↗
Japanese Ministry of Health, Labour and Welfare (MHLW) approves two UCB treatments, RYSTIGGO ® (rozanolixizumab) and ZILBRYSQ ® (zilucoplan), for the treatment of generalized myasthenia gravis (gMG) in adult patients (only for patients who inadequately respond to steroids or other immunosuppressants). |
| 2023-09-15 | UCB Pharma | Regulatory UCB receives CHMP positive opinion of zilucoplan for the treatment of adults with generalized myasthenia gravis in Europe ucb.com ↗
The CHMP’s positive opinion is now being reviewed by the European Commission, which grants centralized marketing authorizations for medicinal products in the EU. |
| 2023-04-13 | UCB Pharma | Results UCB’s Rozanolixizumab and Zilucoplan Phase 3 Generalized Myasthenia Gravis studies published in prestigious The Lancet Neurology Journal ucb.com ↗
RAISE publication describes clinically meaningful and statistically significant improvements in MG-specific efficacy outcomes, as well as a favorable safety profile, for zilucoplan, the first C5 complement inhibitor to be self-administered at home by adult patients with AChR-Ab+ gMG. |
| 2022-11-14 | UCB Pharma | Regulatory UCB announces U.S. FDA acceptance of new drug application and EMA MAA validation for zilucoplan for the treatment of generalized myasthenia gravis in adult patients ucb.com ↗
UCB, a global biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for its investigational treatment, zilucoplan. |
| 2022-05-10 | UCB Pharma | Results UCB presents efficacy and safety results for zilucoplan and rozanolixizumab in generalized myasthenia gravis ucb.com ↗
Phase 3 RAISE and MycarinG studies in generalized myasthenia gravis (gMG) show zilucoplan and rozanolixizumab improve gMG-specific outcomes with consistent statistical significance and clinical relevance |
| 2022-02-04 | UCB Pharma | Results UCB announces positive data in myasthenia gravis with zilucoplan phase 3 study results ucb.com ↗
The primary endpoint of the trial was met; a clinically meaningful and statistically significant improvement from baseline in Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) total score at Week 12 was observed for the zilucoplan treatment group vs placebo. |
| 2021-04-22 | UCB Pharma | Results UCB’s zilucoplan shows no relevant effect in immune-mediated necrotizing myopathy (IMNM) ucb.com ↗
The phase 2 study with zilucoplan , a peptide inhibitor of complement component 5 (C5), in immune-mediated necrotizing myopathy (IMNM) did not show a meaningful effect of zilucoplan for people living with IMNM. |
All press releases naming this drug 12 releases
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 20 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Zilucoplan | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06435312 ↗ |
| Known as | RA101495 | “The purpose of the study is to evaluate the safety and efficacy of zilucoplan (RA101495) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).” NCT03078582 ↗5“Drug: Zilucoplan (RA101495)” NCT03030183 ↗ “Drug: zilucoplan (RA101495)” NCT05514873 ↗ “Zilucoplan (RA101495)” NCT03225287 ↗ “zilucoplan (RA101495)” NCT04225871 ↗ “zilucoplan (RA101495)” NCT04115293 ↗ |
| Action | Inhibit | “macrocyclic peptide inhibitor of complement component 5” PMID 32065623 ↗ May 2020 |
| Modality | Peptide | “subcutaneously (SC) self-administered macrocyclic peptide inhibitor” PMID 32065623 ↗ May 2020 |
| Route | Subcutaneous | “0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC” NCT03030183 ↗ |
| Target | C5 | “complement C5 inhibitor” PMID 37059508 ↗ May 2023 |