Database for
Therapeutic Antibodies
Therapeutic Antibodies
| Entry ID | 112 |
| INN | Emapalumab |
| Status | Approved |
| Drug code(s) | NI-0501 |
| Brand name | Gamifant |
| mAb sequence source | mAb human |
| General Molecular Category | Naked monospecific |
| Format, general category | Full length Ab |
| Format details | None |
| Isotype (Fc) | IgG1 |
| Light chain isotype | lambda |
| Linker | None |
| Ave. DAR | None |
| Conjugated/fused moiety | None |
| Discovery method/technology | Phage display-derived |
| Target(s) | IFN gamma |
| Indications of clinical studies | Primary Hemophagocytic Lymphohistiocytosis; Phase 1 in heathy volunteers |
| Primary therapeutic area | Immune-mediated / inflammatory disorders |
| Most advanced stage of development (global) | Approved US |
| Status | Active |
| Start of clinical phase (IND filing or first Phase 1) | September 15, 2011 |
| Start of Phase 2 | |
| Start of Phase 3 | January 15, 2013 |
| Date BLA/NDA submitted to FDA | March 20, 2018 |
| Year of first approval (global) | 2018 |
| Date of first US approval | November 20, 2018 |
| INN, US product name | Emapalumab, emapalumab-lzsg |
| US or EU approved indications | Treatment of paediatric (newborn and older) and adult patients with primary haemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance to conventional HLH therapy. |
| Company | NovImmune SA |
| Licensee/Partner | Swedish Orphan Biovitrum AB |
| Comments about company or candidate | November 13, 2020 I Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) today announced that the Committee for Medicinal Products for Human use (CHMP) has adopted a negative opinion recommending a refusal of the marketing authorisation for emapalumab for the treatment of primary haemophagocytic lymphohistiocytosis (pHLH) in children under 18 years of age in Europe. The negative opinion was given after the re-examination requested by Sobi after the initial opinon in July 2020. EMA's negative opinion appealed: The applicant (SFL Regulatory Services GmbH) for Gamifant (emapalumab) have requested re-examination of the Committee's negative opinions for these medicines adopted at the July 2020 meeting. The CHMP will now re-examine the opinions and issue final recommendations. Aug 2019: Included in Committee for medicinal products for human use (CHMP) Agenda of CHMP written procedure* 19-22 August 2019. June 2019: Swedish Orphan Biovitrum acquires emapalumab from Novimmune. August 2018: MAA submitted to EMA. June 2018: Swedish Orphan Biovitrum AB and Novimmune SA have entered into an exclusive licence agreement for the perpetual global rights to emapalumab, a late-stage orphan drug candidate for the treatment of primary Haemophagocytic lymphohistiocytosis (HLH), developed by Novimmune. BLA submitted in March 2018; PDUFA date is Nov 20 2018. NCT03312751 Phase 3 study not yet recruiting as of Mar 28 2018. Sep 2017: Novimmune SA, a privately held Swiss biopharmaceutical company, announced that the Food and Drug Administration of the US (FDA) has granted Rare Pediatric Disease Designation to its lead compound emapalumab for the treatment of primary HLH as a drug for a “rare pediatric disease”. Priority review vouchers are awarded upon the FDA approval of an eligible drug for a rare pediatric disease. EMA PRIME designation granted June 2016. Breakthrough therapy designation. NCT01818492 Phase 2 study recruiting as of June 2014; Changed to Phase 2/3 study in June 2016 |
| Full address of company | 15B Chemin du Pré-Fleuri, CH-1228 Plan-Les-Ouates, Switzerland Europe Switzerland https://www.lightchainbio.com/contact/contact-us.html |
IgG1 lambda1
| Anticipated events | None |
| Factor(s) contributing to discontinuation | None |