Luspatercept–aamt

What is Luspatercept-aamt?

• Luspatercept-aamt (Reblozyl) is an erythroid maturation agent.
• It is a receptor fusion protein consisting of a modified extracellular domain of the human activin receptor type IIB linked to a human IgG1 Fc domain with a calculated molecular mass of approximately 76 kD.
• It is produced in Chinese hamster ovary cells by recombinant DNA technology.

What are the uses of this medicine?

• It is indicated for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions.
• It is indicated for the treatment of anemia failing an erythropoiesis stimulating agent and requiring 2 or more red blood cell units over 8 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).

How does this medicine work?

• Luspatercept-aamt is a recombinant fusion protein that binds several endogenous TGF-β superfamily ligands, thereby diminishing Smad2/3 signaling.
• Luspatercept-aamt promoted erythroid maturation through differentiation of late-stage erythroid precursors (normoblasts) in mice.
• In models of β-thalassemia and MDS, luspatercept-aamt decreased abnormally elevated Smad2/3 signaling and improved hematology parameters associated with ineffective erythropoiesis in mice.

Who Should Not Use this medicine?

• It is not indicated for use as a substitute for RBC transfusions in patients who require immediate correction of anemia.

What are the brand names and dosage forms of this medicine?

• Brand name: Reblozyl; NDC Codes: 59572-711-01, 59572-775-01
• Dosage form:

1. Injection: 25 mg white to off-white lyophilized powder in a single-dose vial for reconstitution.
2. Injection: 75 mg white to off-white lyophilized powder in a single-dose vial for reconstitution.

Is this medicine FDA approved?

• It is approved by US FDA on April 3, 2020.

What are the active and inactive ingredients in in this medicine?

• Active ingredient: Luspatercept-aamt
• Inactive ingredients:

1. Citric acid monohydrate
2. Trisodium citrate dihydrate
3. Polysorbate 80
4. Sucrose
5. Hydrochloric acid
6. Sodium hydroxide
7. Water

How should this medicine be used?

1. Your healthcare provider will prescribe REBLOZYL in a dose that is right for you.
2. REBLOZYL is given as an injection under your skin (subcutaneous) in the upper arm, thigh or stomach (abdomen) by your healthcare provider.
3. Your healthcare provider will do regular blood tests to check your hemoglobin to monitor if your anemia is getting better before each injection and during your treatment with REBLOZYL.
4. Your healthcare provider may adjust your dose or stop treatment depending on how you respond to REBLOZYL.
5. If your scheduled REBLOZYL dose is delayed or missed, your healthcare provider will give your dose of REBLOZYL as soon as possible and continue your treatment as prescribed with at least 3 weeks between doses.

What side effects can this medication cause?

• Blood clots: Blood clots in the arteries, veins, brain, and lungs have happened in people with beta thalassemia during treatment with REBLOZYL. The risk of blood clots may be higher in people who have had their spleen removed or who take hormone replacement therapy or birth control (oral contraceptives).
• High blood pressure
• Most common side effects include:

1. tiredness
2. muscle or bone pain
3. dizziness
4. diarrhea
5. stomach (abdominal) pain
6. allergic reactions
7. headache
8. joint pain (arthralgia)
9. nausea
10. cough
11. trouble breathing

What to do in case of emergency/overdose?

• In case of overdose, call the poison control helpline of your country. In the United States, call 1-800-222-1222.

• Overdose related information is also available online at poisonhelp.org/help.
• In the event that the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services. In the United States, call 911.

Can this medicine be used in pregnancy?

• Based on findings in animal reproduction studies, REBLOZYL may cause fetal harm when administered to a pregnant woman.

Can this medicine be used in children?

• Safety and effectiveness in pediatric patients have not been established. Based on findings in juvenile animals, REBLOZYL is not recommended for use in pediatric patients.

What should I know about storage and disposal of this medication?

• Store vials refrigerated at 2°C to 8°C (36°F to 46°F) in original carton to protect from light.
• Do not freeze.

• Dailymed label info on Luspatercept–aamt
• FDA Luspatercept–aamt

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