Celgene and Acceleron Announce FDA Approval of First-in-Class Erythroid Maturation Agent REBLOZYL for Adult Patients with Beta Thalassemia

On November 12, 2019, VCBeat (WeChat ID: vcbeat) learned from foreign media reports that biotechnology company Celgene and pharmaceutical company Acceleron Pharma announced that their erythroid maturation agent REBLOZYL (luspatercept-aamt) had received FDA approval for marketing, for the treatment of adult patients with beta-thalassemia who require regular red blood cell (RBC) transfusions.

Celgene, founded in 1986 and headquartered in California, USA, is a global leading biotechnology company. The company is dedicated to developing innovative therapies and advancing their commercialization for the treatment of cancer, immune system disorders, and inflammatory diseases. Celgene went public on the NASDAQ Stock Exchange in 1987 under the ticker symbol CELG.

Acceleron Pharma, Inc., founded in 2004 and headquartered in Cambridge, Massachusetts, USA, is a biopharmaceutical company. The company is primarily engaged in the discovery, development, and commercialization of novel biologic therapies, as well as the development of anticancer drugs and treatments for rare diseases. Acceleron went public on the NASDAQ Stock Exchange in 2013 under the ticker symbol XLRN.

β-Thalassemia is a rare hereditary blood disorder caused by hemoglobin defects. The disease typically manifests as abnormal erythropoiesis and a reduced count of healthy red blood cells, often leading to severe anemia and complications such as organ damage. β-Thalassemia is a severe, chronic, progressive anemia, and patients rely on blood transfusions for survival. Generally, patients require 6–20 units of packed red blood cells (RBCs) every six months; the interval between transfusions must not exceed 35 days, and the transfusion cycle shortens progressively with age.

REBLOZYL is the first and only FDA-approved erythroid maturation agent, as well as the first therapeutic drug for beta-thalassemia. This medication modulates the activity of late-stage erythroid cells, helping patients reduce their red blood cell (RBC) transfusion burden and providing them with a novel treatment option. REBLOZYL has also received FDA approval for the treatment of anemia associated with very low- to intermediate-risk myelodysplastic syndromes (MDS).

REBLOZYL has passed the safety and efficacy evaluation of the Phase III BELIEVE clinical trial, a randomized, double-blind, placebo-controlled, multicenter study. Conducted across 65 sites in 15 countries worldwide, the study achieved significant clinical milestones. Following the completion of the Phase III trial, Celgene will conduct a five-year follow-up of patients treated with REBLOZYL to monitor the drug’s long-term efficacy and adverse reactions.

Nadim Ahmed, President of Celgene’s Global Hematology and Oncology Business Unit, stated, “The FDA approval of REBLOZYL represents a significant milestone for Celgene, underscoring our continued commitment to addressing hematologic diseases. As a novel therapy, REBLOZYL provides an effective treatment option for patients with beta-thalassemia who are chronically dependent on red blood cell (RBC) transfusions.”

Habib Dable, President and Chief Executive Officer of Acceleron Pharma, said, “We are delighted that REBLOZYL, a treatment for beta-thalassemia, has received its initial FDA approval, providing patients with this condition with a novel therapeutic option. We are deeply grateful to the patients, families, and caregivers who participated in and supported our research; their contributions were instrumental in bringing REBLOZYL to market.”

(Compiled by Xu Xiaoxue)