Database for
Therapeutic Antibodies
Therapeutic Antibodies
| Entry ID | 16 |
| INN | Ravulizumab |
| Status | Approved |
| Drug code(s) | ALXN1210 |
| Brand name | Ultomiris |
| mAb sequence source | mAb humanized |
| General Molecular Category | Naked monospecific |
| Format, general category | Full length Ab |
| Format details | None |
| Isotype (Fc) | IgG2/4 |
| Light chain isotype | kappa |
| Linker | None |
| Ave. DAR | None |
| Conjugated/fused moiety | None |
| Discovery method/technology | None |
| Target(s) | Complement C5 |
| Indications of clinical studies | Neuromyelitis Optica Spectrum Disorder, Generalized myasthenia gravis, Paroxysmal Nocturnal Hemoglobinuria, Atypical Hemolytic Uremic Syndrome |
| Primary therapeutic area | Immune-mediated / inflammatory disorders |
| Most advanced stage of development (global) | Approved EU, US, Japan, Australia |
| Status | Active |
| Start of clinical phase (IND filing or first Phase 1) | August 15, 2014 |
| Start of Phase 2 | November 15, 2015 |
| Start of Phase 3 | September 15, 2016 |
| Date BLA/NDA submitted to FDA | June 18, 2018 |
| Year of first approval (global) | 2018 |
| Date of first US approval | December 21, 2018 |
| INN, US product name | Ravulizumab, ravulizumab-cwvz |
| US or EU approved indications | May 10, 2023 I Ultomiris (ravulizumab) has been approved in the European Union (EU) for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder. European Commission has approved ULTOMIRIS® (ravulizumab)—the first and only long-acting C5 complement inhibitor administered every eight weeks*—for the treatment of adults and children with a body weight of 10 kg or above with atypical hemolytic uremic syndrome (aHUS) who are complement inhibitor treatment-naïve or have received SOLIRIS® (eculizumab) for at least three months and have evidence of response to eculizumab. ULTOMIRIS is approved in the United States (U.S.), European Union (EU) and Japan as a treatment for adults with paroxysmal nocturnal hemoglobinuria (PNH) and in the U.S. for atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA) in adult and pediatric (one month of age and older) patients. Ultomiris was approved in the US in April 2022 and Japan in August 2022 for certain adults with generalised myasthenia gravis. Sep 2022: Ultomiris (ravulizumab) has been approved in Europe as an add-on to standard therapy for the treatment of adult patients with generalised myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive. |
| Company | AstraZeneca |
| Licensee/Partner | None |
| Comments about company or candidate | AstraZeneca acquired Alexion (formerly Syntimmune) in 2021. EU approval announced July 3, 2019. Phase 3 study in generalized myasthenia gravis started in April 2019. BLA submission announced June 19, 2018; The submission uses a rare disease priority review voucher, which designates the BLA for an expedited eight-month review by the FDA. Company plans global regulatory filings in the U.S. and EU in mid-2018, followed by Japan later in the year. US orphan designation for hemolytic uremic syndrome. July 2017: Enrollment is complete in a Phase 3 trial comparing ALXN1210 administered intravenously every eight weeks to Soliris in complement inhibitor treatment-naive patients with PNH. Alexion expects to report data from this study in the second quarter of 2018. Alexion initiated a Phase 3 PNH Switch study of ALXN1210 administered intravenously every eight weeks compared to patients currently treated with Soliris in the second quarter of 2017. The Company expects to complete enrollment in this study in the third quarter of 2017. Patients are being dosed in a Phase 3 trial with ALXN1210 administered intravenously every eight weeks in complement inhibitor treatment-naive adolescent and adult patients with aHUS. Enrollment is expected to be complete in early 2018. Alexion expects to initiate a Phase 3 trial of ALXN1210 in pediatric patients with aHUS in the third quarter of 2017. ALXN1210, a longer-acting anti-C5 antibody designed to treat paroxysmal nocturnal hemoglobinuria, has received orphan drug status from the FDA and EMA. NCT02946463 Phase 3 recruiting as of Oct 2016. Start of Phase 1 announced Jan 29, 2015. NCT05288660 Phase 1 started in Aug 2014. |
| Full address of company | Cambridge, United Kingdom Europe United Kingdom https://www.astrazeneca.com/our-company/contact-us.html |
Next-generation Soliris candidate; a longer-acting anti-C5-antibody suitable for monthly dosing; uses Xencor Xtend technology. Ravulizumab (ALXN1210) differs from eculizumab by 4 amino acid substitutions, and like eculizumab is a complement component C5 inhibitor. Two of the amino acid substitutions are M428L and N434S (LS mutation) in the Fc region; these modulate the affinity to FcRn. The remaining two amino acid substitutions are in the complementarity-determining region; these substitutions modulate the affinity to the antigen.
| Anticipated events | None |
| Factor(s) contributing to discontinuation | None |