Pharmaceutical drug
Pharmaceutical compound
Luspatercept , sold under the brand nameReblozyl , is a medication used for the treatment of anemia inbeta thalassemia andmyelodysplastic syndromes .[ 5]
The USFood and Drug Administration (FDA) considers it to be afirst-in-class medication .[ 8]
Luspatercept[ 9] indicated for the treatment of adults with transfusion-dependent anemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts, who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy.[ 6]
Luspatercept is indicated for the treatment of adults with transfusion-dependent anaemia associated with beta thalassaemia.[ 6]
Possible adverse effects include temporary bone pain, joint pains (arthralgias), dizziness, elevated blood pressure (hypertension) and elevated uric acid levels (hyperuricemia ). There was also an increased risk ofthrombosis (blood clots) in patients who have risk factors for thrombosis who are taking luspatercept.[ 10]
Structure and mechanism [ edit ] Luspatercept is arecombinant fusion protein derived fromhuman activin receptor type IIb (ActRIIb) linked to a protein derived fromimmunoglobulin G .[ 11] transforming growth factor beta ) superfamilyligands to reduceSMAD signaling. The reduction in SMAD signaling leads to enhancederythroid maturation.[ 10]
Phase III trials evaluated the efficacy of luspatercept for the treatment of anemia in the hematological disorders beta thalassemia[ 12] [ 13]
It wasdeveloped byAcceleron Pharma in collaboration withCelgene .[ 14]
The U.S.Food and Drug Administration (FDA) granted approval for luspatercept–aamt in November 2019, for the treatment of anemia (lack of red blood cells) in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions.[ 15] [ 16] [ 17] [ 18] [ 6]
The U.S.Food and Drug Administration (FDA) awardedorphan drug status in 2013, andfast track designation in 2015.[ 19]
Luspatercept is being evaluated for use in adults with non-transfusion dependent beta thalassemia.[ 20]
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This article incorporates text from this source, which is in thepublic domain.