Database for
Therapeutic Antibodies
Therapeutic Antibodies
| Entry ID | 114 |
| INN | Concizumab |
| Status | Approved |
| Drug code(s) | NNC172-2021, NN7415, mAb2021 |
| Brand name | Alhemo™ |
| mAb sequence source | mAb humanized |
| General Molecular Category | Naked monospecific |
| Format, general category | Full length Ab |
| Format details | None |
| Isotype (Fc) | IgG4 |
| Light chain isotype | kappa |
| Linker | None |
| Ave. DAR | None |
| Conjugated/fused moiety | None |
| Discovery method/technology | None |
| Target(s) | Tissue factor pathway inhibitor |
| Indications of clinical studies | Haemophilia A and B With and Without Inhibitors, Hemophilia A |
| Primary therapeutic area | Cardiovascular / hemostasis disorders |
| Most advanced stage of development (global) | Approved Australia, Canada |
| Status | Active |
| Start of clinical phase (IND filing or first Phase 1) | October 15, 2010 |
| Start of Phase 2 | June 08, 2017 |
| Start of Phase 3 | October 21, 2019 |
| Date BLA/NDA submitted to FDA | |
| Year of first approval (global) | 2023 |
| Date of first US approval | |
| INN, US product name | Concizumab |
| US or EU approved indications | None |
| Company | Novo Nordisk |
| Licensee/Partner | None |
| Comments about company or candidate | Aug 2023: Health Canada has approved a new indication for Alhemo (concizumab injection) for the treatment of Canadian adolescent and adult patients with hemophilia A (congenital factor VIII [FVIII] deficiency) who have FVIII inhibitors. Alhemo was first approved by Health Canada in March 2023 for the treatment of patients who have FIX inhibitors and require routine prophylaxis to prevent or reduce the frequency of bleeding episodes April 24, 2023, Novo Nordisk received a complete response letter from the FDA regarding the therapy, an anti-tissue factor pathway inhibitor antibody called concizumab. Alhemo™ was approved by Health Canada on March 10, 2023. Health Canada approved Alhemo™, the first anti-tissue factor pathway inhibitor (TFPI) and subcutaneous prophylactic treatment for people living with hemophilia B with inhibitors.1 Alhemo™ is indicated for the treatment of patients over the age of 12 with hemophilia B who have FIX inhibitors and require routine prophylaxis to prevent or reduce the frequency of bleeding episodes.1 MAA evaluation started on Jan 26, 2023. July 2022: Novo Nordisk expects to submit concizumab for regulatory approval for the prophylactic treatment of haemophilia A or B with inhibitors in the second half of 2022 in the US and Japan, and in 2023 in the EU and the UK. Aug 2020: Phase 3 studies resumed. March 2020: Novo Nordisk announced that two clinical trials in the concizumab Phase III programme (Explorer7 and Explorer8) and one clinical trial in the Phase II programme (Explorer5) have been paused. The three clinical trials were investigating concizumab prophylaxis treatment in haemophilia A and B patients regardless of inhibitor status. These results are certainly negative for concizumab and coupled with the previous failure of four TFPI inhibitors, is a major set-back for the TFPI inhibitor class. Novo Nordisk is yet to hold a press release on the progress of the drug but have suspended all ongoing clinical trials until further review. FDA granted concizumab Breakthrough Therapy designation for prophylaxis treatment of people with hemophilia B with inhibitors towards Factor VIII or Factor IX, and Orphan Drug designation for hemophilia A and B. Concizumab was granted Orphan Drug designation in the EU for treatment of hemophilia B. Q3 2019 earnings release: In October 2019, Novo Nordisk initiated the explorer7 phase 3 clinical trial with concizumab in patients with haemophilia A or B with inhibitors towards FVIII or FIX. The objective of the trial is to establish the safety and efficacy of once-daily subcutaneous concizumab as a pen device based on prophylactic treatment to reduce the number of bleeds. A parallel phase 3 trial in haemophilia A or B patients without inhibitors, explorer8, is being initiated in November 2019. The trials are expected to enrol approximately 265 patients from 32 countries. The trials are the first pivotal trials with concizumab and the results are expected to form the basis for regulatory approval for oncedaily subcutaneous prophylaxis treatment with concizumab in all haemophilia patients with and without inhibitors. NCT04082429 Phase 3 Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia Without Inhibitors (explorer8) recruiting as of Nov 13, 2019; NCT04083781 explorer7 study recruiting as of Oct 2019. Jan Astermark presented data at ISTH 2019, concluding that the “Phase 2 trial results confirmed the proof of concept, with no safety signals, and has guided selection of the phase 3 dosing regimen. Further development of concizumab as a prophylactic treatment for all hemophilia patients is supported.” As part of this important work, breakthrough therapy designation has been granted by the FDA based on the HBwI data. Phase 1b study results published (J Thromb Haemost. 2018 Nov;16(11):2184-2195. doi: 10.1111/jth.14272. Epub 2018 Sep 30). Listed as Phase 2 in Novo Nordisk pipeline dated June 20, 2019. EudraCT Number 2016-000510-30 and 2016-000614-29 (Explorer 4 and Explorer 5) started in Sweden in June 2017. NCT02490787 Explorer™3 Phase 1 study recruiting as of Sep 15. Phase 1 NCT01631942 terminated as of January 2014 due to the need for changes in the trial design requiring a new re-designed multiple dosing phase 1 trial. Initiation of this new trial awaits additional non-clinical data. Listed in Novo Nordisk pipeline as of May 2014 |
| Full address of company | Novo Alle 1, 2880 Bagsværd, Denmark Europe Denmark https://www.novonordisk.com/contact-us.html |
Hinge stabilized S228P
| Anticipated events | Sponsor response to FDA CRL, Regulatory review EU (positive opinion in Oct 2024), US, Japan- anticipate approval |
| Factor(s) contributing to discontinuation | None |