Therapeutic Luspatercept antibody from the original Reblozyl® commercial drug.
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|API type||Luspatercept is produced in Chinese Hamster Ovary (CHO) cells by recombinant DNA technology.|
|Pharmacotherapeutic group||Antianaemic preparations, other antianaemic preparations|
|Target of antibody||TGF-beta|
|General function||The active substance in Reblozyl, luspatercept, regulates the maturation of red blood cells. It does this by blocking a signalling pathway called Smad2/3 that slows down the maturation of red blood cells and is overactive in patients with beta thalassaemia and myelodysplastic syndromes. Blocking Smad2/3 increases the production of red blood cells and allows them to develop normally.|
(Mechanism of action; Source EMA document)
|Luspatercept, an erythroid maturation agent, is a recombinant fusion protein that binds selected transforming growth factor-β (TGF-β) superfamily ligands. By binding to specific endogenous ligands (e.g. GDF-11, activin B) luspatercept inhibits Smad2/3 signalling, resulting in erythroid maturation through differentiation of late-stage erythroid precursors (normoblasts) in the bone marrow. Smad2/3 signalling is abnormally high in disease models characterised by ineffective erythropoiesis, i.e. MDS and β-thalassaemia, and in the bone marrow of MDS patients.|
|Pharmacodynamic properties (Pharmacodynamic effects; Source EMA document)||The efficacy and safety of luspatercept were evaluated in a Phase 3 multicentre, randomised, double-blind, placebo-controlled study MEDALIST (ACE-536-MDS-001) in adult patients with anaemia requiring RBC transfusions (≥ 2 units/8 weeks) due to International Prognostic Scoring System-Revised (IPSS-R) very low-, low- or intermediate-risk MDS who have ring sideroblasts (≥ 15%). Patients were required to have either received prior treatment with an erythropoiesisstimulating agent (ESA) with inadequate response, to be ineligible for ESAs (determined to be unlikely to respond to ESA treatment with serum erythropoietin (EPO) > 200 U/L), or intolerant to ESA treatment. Patients with deletion 5q (del5q) MDS were excluded from the study.
Patients in both arms were treated for 24 weeks, then continued treatment if they had demonstrated clinical benefit and absence of disease progression. The study was unblinded for analyses when all patients had at least received 48 weeks of treatment or discontinued treatment.
A treatment effect in favour of luspatercept over placebo was observed in most subgroups analysed using transfusion independence ≥12 weeks (during week 1 to week 24), including patients with high baseline endogenous EPO level (200-500 U/L) (23.3% versus 0%, explorative analysis). Only limited data are available for the group with transfusion burden of ≥ 8 units/8 weeks. Safety and efficacy have not been established in patients with a transfusion burden of > 12 units/8 weeks.
|Original license holder|
|Marketing authorisation numbers||EU/1/20/1452/001-002
|Marketing authorisation holder||Bristol-Myers Squibb Pharma EEIG
Blanchardstown Corporate Park 2
Dublin 15, D15 T867
|Name of the manufacturer of the biological active substance||Lonza Biologics Tuas Pte Ltd.
35 Tuas South Ave. 6,
Singapore, Singapore 637377
Biogen MA Inc.
5000 Davis Dr
Research Triangle Park, NC
|Name and address of the manufacturer(s) responsible for batch release||Celgene Distribution B.V.
3528 BD Utrecht
|Max shelf life||48 months
|Storage conditions||2°C – 8°C
|List of excipients||Citric acid monohydrate (E330)
Sodium citrate (E331)
Hydrochloric acid (for pH adjustment)
Sodium hydroxide (for pH adjustment)