The approval of REBLOZYL® for beta-thalassemia, which received a Priority Review designation from the FDA, is based on results from the pivotal, Phase 3, randomized, double-blind, placebo-controlled, multicenter BELIEVE trial evaluating the safety and efficacy of REBLOZYL® for the treatment of anemia in adult patients with beta-thalassemia who require regular RBC transfusions.  All patients were eligible to receive the best supportive care, which included: RBC transfusions and iron-chelating agents, as well as use of: antibiotic, antiviral, and antifungal therapy and/or nutritional support, as needed.

The trial results achieved clinically meaningful and statistically significant improvement in the primary endpoint. 

The study also met key secondary endpoints, including transfusion burden reduction.    

Efficacy endpoints included transfusion burden reduction of ≥50% (with a reduction of at least 2 units) during weeks 13-24 and weeks 37-48. A ≥50% reduction in transfusion burden was observed in 7.6% of patients receiving REBLOZYL® vs. 1.8% of patients in the placebo arm at weeks 13-24, and 10.3% of patients vs. 0.9% of patients at weeks 37-48.

The Product REBLOZYL®

REBLOZYL® is a first-in-class erythroid maturation agent that promotes late-stage red blood cell maturation in animal models. 

Celgene and Acceleron are jointly developing REBLOZYL® as part of a global collaboration.

The FDA is also evaluating luspatercept-aamt for the treatment of anemia in adults with very low- to intermediate-risk myelodysplastic syndromes (MDS) who have ring sideroblasts and require RBC transfusions. A Prescription Drug User Fee Act (PDUFA), or target action date, is set for April 4, 2020 for this indication.

In Europe, Celgene’s Marketing Authorization Application for the treatment of anemia in adults with beta thalassemia or MDS is currently under review.

REBLOZYL® is indicated for anemia in adult patients with beta thalassemia who require RBC transfusions. The product has not been approved as safe and effective for use in patients with MDS or myelofibrosis in any country and is not indicated for use as a substitute for RBC transfusions in patients who require immediate correction of anemia 

Safety and Tolerability:  

It is important for the reader to learn about the detailed warnings, precautions and other adverse effects of REBLOZYL®. Please read the firm’s press release at the Celgene’s website.  

Ongoing Trials Include:  

• A Phase 2 trial (BEYOND) in adult patients with non-transfusion-dependent beta thalassemia;
• A Phase 3 trial (COMMANDS) in ESA-naïve, lower-risk MDS patients, and  
• A Phase 2 trial in myelofibrosis patients  

For more information please visit, www.clinicaltrials.gov.

Prohost Observations

Again Celgene is demonstrating its high value and its capability to increase the number of its approved products; hence, the continued boosting of its drugs’ sales revenues. We are still waiting for a possible finalization of Bristol-Myers’ (BMY) acquisition of Celgene, which, if it materializes, would mean BMY could add a wealth to its pipeline investigational products and enlarge its approved products’ market.    

Regarding Acceleron Pharma, REBLOZYL® approval is this firm’s first drug approval. The firm’s stock, XLRN, is trading right now at $45.81 UP $0.84 with a market Cap. of $2.37 billion. Its 52-week high is $56.91 and its 52-week low is $37.

We will further examine and evaluate Acceleron Pharma in the very near future. 

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