FDA Grants Priority Review to Luspatercept-aamt for Anemia in Adults With NTD β-Thalassaemia
The FDA has accepted and granted priority review to the supplemental biologics license application (sBLA) for Luspatercept-aamt (Reblozyl) to treat anemia in adult patients with non-transfusion dependent (NTD) β- thalassaemia, according to a press release by Bristol Myers Squibb (BMS).
The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of March 27, 2022. In addition, the European Medicines Agency (EMA) has validated the Type II variation for Reblozyl inNTD β-thalassaemia.
“Patients with non-transfusion dependent β-thalassaemia may not require lifelong blood transfusions for survival, but their need for effective treatment options is significant as they face a range of clinical complications due to chronic anemia and iron overload,” said Noah Berkowitz, MD, PhD, Senior Vice President, Hematology Development, Bristol Myers Squibb, in a press release. ´´Reblozyl is an important therapy approved for anemia associated with β-thalassaemia and lower-risk myelodysplastic syndromes in multiple countries, including the United States and within the European Union. Along with our partners at Merck, we are committed to continuing to advance our clinical program for Reblozyl and look forward to working with the FDA during its review of our application for this underserved patient population”, Mr Berkowitz added.
In the phase 2, double-blind, randomized, placebo-controlled, multicenter BEYOND study (NCT03342404), Luspatercept 1 mg/kg administered every 3 weeks was combined with best supportive care (BSC) and compared with an arm of subcutaneous placebo administered every 21 days plus BSC.
The first results from the study showed that in 145 patients, 77.7% of patients who received Luspatercept achieved a hemoglobin increase compared with none of the patients in the placebo arm (P < .0001). The primary endpoint of ≥ 1.0 g/dL mean increase in haemoglobin was met. The study also showed positive results for the key secondary endpoint: 52.1% of patients in the Luspatercept/BSC arm achieved a mean hemoglobin increase of ≥ 1.5 g/dL compared to baseline. In comparison, no patients in the placebo/BSC arm achieved an increase in hemoglobin from baseline (P < .0001). Notably, changes from baseline in patient-reported outcomes were associated with increases in haemoglobin. Overall, 89.6% of patients who were treated with Luspatercept remained transfusion-free at weeks 1through 24 compared with only 67.3% of the placebo group (P = .0013).
Updated findings from the study will be presented during the upcoming 63rd American Society of Hematology (ASH) Annual Meeting and Exposition, to take place December 11-14, 2021.
Luspatercept-aamt (Reblozyl, Bristol Myers Squibb) is an erythroid maturation agent. The agent is approved in the United States, Europe, Canada, and Australia for treatment of anemia among adults with β–thalassaemia who require regular red blood cell transfusions. It also is approved for the treatment of certain patients with anemia associated with lower-risk myelodysplastic syndrome.
