FDA Approves Luspatercept-aamt for Anemia in Patients with Beta Thalassemia

November 8, 2019
From the American Society of Clinical Oncology in cooperation with the Food and Drug Administration (FDA) and as a service to our members, ASCO will periodically distribute information about newly approved therapies for cancer patients. This helps FDA inform oncologists and professionals in oncology-related fields about recent approvals in a timely manner. Included in the email from the FDA will be a link to the product label, which will provide the relevant clinical information on the indication, contraindications, dosing, and safety. In sending this information, ASCO does not endorse any product or therapy and does not take any position on the safety or efficacy of the product or therapy described. The following is a message from the Director of the FDA Oncology Center of Excellence, Dr. Richard Pazdur:
On November 8, 2019, the Food and Drug Administration approved luspatercept-aamt (REBLOZYL®, Celgene Corp.) for treatment of anemia in adult patients with beta thalassemia who require regular red blood cell transfusions.
Efficacy was evaluated in the BELIEVE trial (NCT02604433), a multicenter, randomized, double-blind, placebo-controlled trial enrolling 336 adult patients with beta thalassemia requiring regular red blood cell (RBC) transfusions. Patients were randomized 2:1 to luspatercept-aamt (224) or placebo (112). Luspatercept-aamt was administered subcutaneously once every 3 weeks as long as a reduction in transfusion requirement was observed or until unacceptable toxicity. 
The primary efficacy outcome measure was the proportion of patients achieving RBC transfusion burden reduction from baseline of at least 33%, with a reduction of at least 2 units from week 13 to week 24. Of the patients who received luspatercept-aamt, 21.4% achieved the primary endpoint compared with 4.5% of those who received placebo (risk difference 17.0; 95% CI 10.4, 23.6; p<0.0001). 
The most common adverse reactions (>10%) in patients with beta thalassemia were headache, bone pain, arthralgia, fatigue, cough, abdominal pain, diarrhea, and dizziness.
The recommended starting dose is 1 mg/kg once every 3 weeks by subcutaneous injection.
FDA granted this application fast track and orphan drug designations. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for 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 or by calling 1-800-FDA-1088.
For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov.

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