According to Pharmacy Times, the FDA recently granted Priority Review status to luspatercept-aamt (Reblozyl). The therapy, for which its developer has submitted a supplemental biologics license application (sBLA), is designed to treat adult patients with non-transfusion dependent beta thalassemia. In particular, luspatercept-aamt works to address anemia within this patient population.
Priority Review is granted to drugs that show the potential to significantly improve the safety and efficacy of treating, diagnosing, or preventing serious conditions as compared to the current standards-of-care. With Priority Review, the FDA aims to take action on that application within 6 months.
Luspatercept-aamt
The National Cancer Institute (NCI) defines luspatercept-aamt as:
A soluble, recombinant fusion protein composed of a modified form of the extracellular domain of human activin receptor type IIb (ActRIIb) and linked to the human IgG1 Fc domain, with red blood cell stimulating activity. This agent ultimately enhances red blood cell production and prevents anemia.
In the Phase 2 BEYOND clinical study, researchers evaluated the therapy for 145 patients with non-transfusion dependent beta thalassemia. They found that patients taking luspatercept-aamt saw a 77.1% mean hemoglobin increase from baseline, despite a lack of transfusions. Alternately, the group taking the placebo saw a 0% increase.
Beta Thalassemia
HBB gene mutations cause beta thalassemia, a genetic blood disorder characterized by lowered hemoglobin levels. Normally, hemoglobin helps carry oxygen throughout the body in red blood cells. Without enough hemoglobin, negative side effects and adverse health reactions can occur. Beta thalassemia may be minor, intermediate, or major, depending on severity. In the major form, many patients require life-long medical care. Symptoms and characteristics associated with this condition include:
- Anemia (low red blood cell count)
- Blood clots
- Jaundice (yellowing of the skin, eyes, and mucous membranes)
- Rapid heart rate
- Abdominal swelling
- Slowed growth
- Shortness of breath
- Fatigue and/or general malaise
- Dizziness
- Headache
- Pale skin
- Enlarged spleen
Learn more about beta thalassemia.
