Database for
Therapeutic Antibodies
Therapeutic Antibodies
| Entry ID | 1730 |
| INN | Narsoplimab |
| Status | Regulatory review |
| Drug code(s) | OMS721 |
| Brand name | (Pending) |
| mAb sequence source | mAb human |
| General Molecular Category | Naked monospecific |
| Format, general category | Full length Ab |
| Format details | None |
| Isotype (Fc) | IgG4 |
| Light chain isotype | lambda |
| Linker | None |
| Ave. DAR | None |
| Conjugated/fused moiety | None |
| Discovery method/technology | None |
| Target(s) | MASP-2 |
| Indications of clinical studies | Steroid-dependent IgA nephropathy, Thrombotic Microangiopathies, Atypical hemolytic uremic syndrome (Phase 1 in healthy volunteers) |
| Primary therapeutic area | Cardiovascular / hemostasis disorders |
| Most advanced stage of development (global) | Regulatory review US |
| Status | Active |
| Start of clinical phase (IND filing or first Phase 1) | May 15, 2013 |
| Start of Phase 2 | August 15, 2014 |
| Start of Phase 3 | February 23, 2017 |
| Date BLA/NDA submitted to FDA | |
| Year of first approval (global) | None |
| Date of first US approval | |
| INN, US product name | Narsoplimab |
| US or EU approved indications | None |
| Company | Omeros Corporation |
| Licensee/Partner | None |
| Comments about company or candidate | Oct 2023: Omeros has stopped a phase 3 trial of narsoplimab in immunoglobulin A (IgA) nephropathy patients after an interim analysis found it was destined to fail, adding another plot line to its epic, ongoing attempt to get the candidate to market. Aug 2023 press release: In May 2023 we had a Type B meeting with the review division at FDA to discuss the planned resubmission of our Biologics License Application (“BLA”) for narsoplimab in hematopoietic stem cell transplant-associated thrombotic microangiopathy (“TA-TMA”). Based on the agency’s feedback we expect to submit to FDA early next month a detailed plan for analysis of survival data from already-identified external sources. Nov 2022: FDA's decision proposes the resubmission of the narsoplimab BLA including a comparison of the existing response data from the completed pivotal trial to a threshold derived from an independent literature analysis and evidence of increased survival from patients in the pivotal trial compared to an appropriate historical control group. It also notes that persuasive evidence of superior survival versus a well-matched historical control group could be sufficient even in the absence of the independent literature analysis. The specific approach to resubmission and its details would be determined through discussion with the review division. June 2022, Omeros submitted to the United States Food and Drug Administration (FDA) a request for Formal Dispute Resolution regarding the Complete Response Letter (CRL) issued by FDA last year regarding the Company’s biologics license application (BLA) for narsoplimab in the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA). Formal dispute resolution is an official pathway that enables a sponsor to appeal a decision by an FDA division to a higher authority within FDA, in this case the Office of New Drugs (OND). Last month, in accordance with the standard dispute resolution procedure, Omeros had a formal meeting with the OND official assigned to decide the dispute. A decision is expected in August 2022. November 18, 2020 I Omeros Corporation announced that it has completed the rolling submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for narsoplimab for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA). Narsoplimab targets mannan-binding lectin-associated serine protease-2 (MASP-2), the effector enzyme of the lectin pathway of complement, and has received breakthrough therapy designation and orphan drug designation from FDA for HSCT-TMA. March 2020: In recent meetings with FDA focused on clinical as well as chemistry, manufacturing and controls (CMC) data, FDA confirmed important aspects of Omeros’ rolling Biologics License Application (BLA) for narsoplimab in HSCT-TMA. The BLA continues on its clear path to completion. Rolling BLA started in Oct 2019. Feb 2019: FDA will consider not only accelerated approval but also regular (full) approval for OMS721 in HSCT-TMA, with the determination based on the submitted data. A rolling BLA submission is appropriate for OMS721 in this indication. Omeros plans to submit first the nonclinical sections. Oct 2018: OMS721 has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy. Aug. 28, 2018-- Omeros Corporation today announced that the European Commission (EC) has adopted a decision designating OMS721 as an Orphan Medicinal Product in the European Union (EU) for treatment in hematopoietic stem cell transplantation (HSCT). The adoption by the EC follows a positive opinion for orphan designation of OMS721 in this indication by the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP). April 2018: FDA granted Breakthrough therapy designation. Feb. 15, 2018-- Omeros Corporation today announced new results from the company’s ongoing Phase 2 study of OMS721 evaluating patients with hematopoietic stem cell transplant-associated thrombotic microangiopathy (HCT-TMA). The data demonstrate an increase in median overall survival in HCT-TMA patients treated with OMS721 compared to a matched historical control. Omeros is scheduled and expects to meet with the U.S. Food and Drug Administration and with the European Medicines Agency to discuss the most expeditious path to approval for OMS721 in HCT-TM. Aug. 4, 2017-- Omeros Corporation today announced that OMS721 has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of Immunoglobulin A (IgA) nephropathy June 2017: The FDA has granted breakthrough therapy designation to Omeros' OMS721 as a treatment for IgA nephropathy. March 2017: Omeros met with FDA recently to discuss these data and Phase 3 development. Following review of the data, FDA suggested that Omeros apply for breakthrough therapy designation in IgAN. The discussion included accelerated approval and an expedited approach to full approval based on an endpoint of proteinuria, which could be faster than accelerated approval. Omeros is preparing its breakthrough application and a Phase 3 protocol for discussion with FDA. The Phase 3 trial in IgAN is expected to begin this year. Fast track designation granted in March 2016. Start of Phase 3 program announced on March 8, 2016; Phase 3 enrollment is expected to begin later this year and patients currently being treated in the Phase 2 trial are likely to be included in the Phase 3 program. The OMS721 Phase 3 program will consist of one clinical trial – a single-arm (i.e., no control arm), open-label trial in patients with newly diagnosed or ongoing aHUS.The FDA has awarded OMS721 both orphan drug designation for the treatment of TMAs and fast-track status for the treatment of aHUS. Omeros' breakthrough therapy OMS721, indicated to treat patients with the kidney disorder Berger's disease, has been granted orphan drug status by the FDA |
| Full address of company | 201 Elliott Avenue West, Seattle, WA 98119 North America United States of America https://www.omeros.com/contact-us/ |
Target is mannan-binding lectin-associated serine protease-2. https://www.bbmt.org/article/S1083-8791(16)30736-4/pdf. S228P hinge mutation.
| Anticipated events | Sponsor response to FDA CRL |
| Factor(s) contributing to discontinuation | None |