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Crizanlizumab

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Crizanlizumab
Monoclonal antibody
TypeWhole antibody
SourceHumanized
Targetselectin P
Clinical data
Trade namesAdakveo
Other namesSEG101, SelG1, crizanlizumab-tmca
AHFS/Drugs.comMonograph
MedlinePlusa620010
License data
Pregnancy
category
Routes of
administration		Intravenous
ATC code
Legal status
Legal status
Identifiers
CAS Number
DrugBank
ChemSpider
  • none
UNII
KEGG
Chemical and physical data
FormulaC6458H9948N1712O2050S58
Molar mass146232.04 g·mol−1

Crizanlizumab, sold under the brand name Adakveo among others, is a monoclonal antibody medication that binds to P-selectin.[3] It is a medication used to reduce the frequency of vaso-occlusive crisis in people aged 16 years and older who have sickle cell anemia.[3][4][6] It is given by injection into a vein.[3][4]

The most common side effects include joint pain, nausea, back pain, fever and abdominal (belly) pain.[4]

Crizanlizumab was approved for medical use in the United States in November 2019.[6][7][8] The EU's EMA withdrew authorization in May 2023 based on no significant effects from a phase 3 trial.[9] The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.[10]

Medical uses

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Crizanlizumab is indicated for the prevention of recurrent vaso occlusive crises in sickle cell disease patients aged 16 years and older.[4] It can be given as an add on therapy to hydroxyurea/hydroxycarbamide (HU/HC) or as monotherapy in patients for whom HU/HC is inappropriate or inadequate.[3][4]

Vaso-occlusive crisis is a common and painful complication of sickle cell disease that occurs when blood circulation is obstructed by sickled red blood cells (red cells are usually round and flexible, but sometimes many red cells in a person with sickle cell anemia will become rigid and crescent-shaped due to polymerization of hemoglobin).[11]

Pathophysiology

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P-selectin molecules are present on the surface of activated platelets and vascular endothelial cells and have been linked to sickle cell vaso-occlusive crises.[12][13][14]

History

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The US Food and Drug Administration (FDA) approved crizanlizumab based on evidence from one clinical trial (Trial 1/NCT01895361) of 132 participants with sickle cell diseases who had a history of vaso-occlusive crisis.[8] The trial was conducted at 60 sites in the United States, Brazil and Jamaica.[8]

The FDA granted the application for crizanlizumab priority review, breakthrough therapy designation, and orphan drug designation.[6] The FDA granted approval of Adakveo to Novartis.[6][8]

The European Medicines Agency's human medicines committee (CHMP) has recommended the withdrawal of Adakveo (crizanlizumab), a medicine for preventing vaso-occlusive crises in patients with sickle cell disease, due to the lack of sufficient benefits outweighing the risks.[15] The STAND phase III study showed that Adakveo does not effectively reduce the number of painful crises requiring healthcare visits or treatment at home compared to a placebo, and it exhibits a higher rate of severe side effects.[16][17]

References

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  1. ^ a b "AusPAR: Crizanlizumab". Therapeutic Goods Administration (TGA). 24 August 2021. Retrieved 4 September 2021.
  2. ^ a b "Adakveo". Therapeutic Goods Administration (TGA). 16 April 2021. Retrieved 6 September 2021.
  3. ^ a b c d e "Adakveo- crizanlizumab injection". DailyMed. 8 September 2022. Retrieved 4 March 2023.
  4. ^ a b c d e f "Adakveo EPAR". European Medicines Agency. 20 July 2020. Retrieved 5 March 2023. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  5. ^ "Adakveo Product information". Union Register of medicinal products. Retrieved 3 March 2023.
  6. ^ a b c d "FDA approves first targeted therapy to treat patients with painful complication of sickle cell disease". U.S. Food and Drug Administration (FDA) (Press release). 15 November 2019. Archived from the original on 21 November 2019. Retrieved 20 November 2019.Public Domain This article incorporates text from this source, which is in the public domain.
  7. ^ "Drug Approval Package: Adakveo (crizanlizumab-tmca)". U.S. Food and Drug Administration (FDA). 17 December 2019. Retrieved 22 January 2020.
  8. ^ a b c d "Drug Trials Snapshots Adakveo". U.S. Food and Drug Administration (FDA). 15 November 2019. Archived from the original on 24 January 2020. Retrieved 26 January 2020. Public Domain This article incorporates text from this source, which is in the public domain.
  9. ^ "Novartis sickle cell drug's approval formally revoked by EU regulators". BioPharma Dive. Retrieved 19 August 2023.
  10. ^ "New Drug Therapy Approvals 2019". U.S. Food and Drug Administration. 31 December 2019. Retrieved 15 September 2020.
  11. ^ Darbari DS, Sheehan VA, Ballas SK (September 2020). "The vaso-occlusive pain crisis in sickle cell disease: Definition, pathophysiology, and management". European Journal of Haematology. 105 (3): 237–246. doi:10.1111/ejh.13430. PMID 32301178. S2CID 215801719.
  12. ^ Manwani D, Frenette PS (December 2013). "Vaso-occlusion in sickle cell disease: pathophysiology and novel targeted therapies". Blood. 122 (24): 3892–3898. doi:10.1182/blood-2013-05-498311. PMC 3854110. PMID 24052549.
  13. ^ Manwani D, Frenette PS (December 2013). "Vaso-occlusion in sickle cell disease: pathophysiology and novel targeted therapies". Hematology. American Society of Hematology. Education Program. 2013: 362–369. doi:10.1182/asheducation-2013.1.362. PMC 3854110. PMID 24319205.
  14. ^ Riley TR, Boss A, McClain D, Riley TT (July 2018). "Review of Medication Therapy for the Prevention of Sickle Cell Crisis". P & T. 43 (7): 417–437. PMC 6027858. PMID 30013299.
  15. ^ EMA (26 May 2023). "EMA recommends revocation of authorisation for sickle cell disease medicine Adakveo". European Medicines Agency (Press release). Retrieved 26 May 2023.
  16. ^ "Novartis provides update on Phase III STAND trial assessing crizanlizumab". Novartis (Press release). Retrieved 26 May 2023.
  17. ^ "EMA recommends revocation of authorisation for sickle cell disease medicine Adakveo". European Medicines Agency (Press release). 26 May 2023. Retrieved 26 May 2023.
[edit]
  • Clinical trial number NCT01895361 for "Study to Assess Safety and Impact of SelG1 With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients With Pain Crises (SUSTAIN)" at ClinicalTrials.gov
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