FDA Approves Luspatercept-Aamt for Anemia in Adults With MDS 

April 07, 2020

On April 3, 2020, the U.S. Food and Drug Administration (FDA) approved luspatercept-aamt (https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-luspatercept-aamt-anemia-adults-mds) (Reblozyl®) for the treatment of anemia failing an erythropoiesis-stimulating agent and requiring two or more red blood cell (RBC) units over eight weeks in adult patients with very low- to intermediate-risk myelodysplastic syndromes with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T).  

FDA Approves Luspatercept-Aamt for Anemia in Adults With MDS 

Efficacy was demonstrated in the MEDALIST trial (NCT02631070), a randomized, multicenter, double-blind, placebo-controlled trial in 229 patients with Revised International Prognostic Scoring System very low-, low-, or intermediate-risk myelodysplastic syndromes who had ring sideroblasts and required RBC transfusions (two or more RBC units over eight weeks). Patients were randomized two to one to receive luspatercept-aamt or placebo. All patients received best supportive care, which included RBC transfusions.  

The main efficacy endpoint in MDS-RS and MDS-RS-T was the proportion of patients who were RBC-transfusion independent (RBC-TI), defined as the absence of any RBC transfusion during any consecutive eight-week period between weeks 1 and 24.  

Of the 153 patients who received luspatercept-aamt, 58 (37.9%; 95% CI = 30.2, 46.1) were RBC-TI for at least eight weeks, compared to 10 patients (13.2%; 95% CI = 6.5, 22.9) who received placebo (treatment difference = 24.6% [95% CI = 14.5, 34.6; p < 0.0001]).  

The most common (> 10%) adverse reactions to luspatercept-aamt are fatigue, headache, musculoskeletal pain, arthralgia, dizziness or vertigo, nausea, diarrhea, cough, abdominal pain, dyspnea, and hypersensitivity.  

The recommended starting dose of luspatercept-aamt is 1 mg/kg once every three weeks by subcutaneous injection. Review hemoglobin results prior to each administration.  

View full prescribing information for luspatercept-aamt. (https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/761136orig2lbl.pdf) 

FDA granted luspatercept-aamt orphan drug and fast-track designation. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics (https://www.fda.gov/regulatory-information/search-fda-guidance-documents/expedited-programs-serious-conditions-drugs-and-biologics).  

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System (https://www.accessdata.fda.gov/scripts/medwatch/index.cfm) or by calling 800-FDA-1088.  

For assistance with single-patient oncology investigational new drugs, healthcare professionals may contact OCE’s Project Facilitate (https://www.fda.gov/about-fda/oncology-center-excellence/project-facilitate) at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov (mailto:OncProjectFacilitate@fda.hhs.gov).  

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