Acceleron Pharma & Bristol Myers Squibb / Reblozyl
In November 2019, Reblozyl became the only FDA-approved erythroid maturation agent, representing an entirely new class of medicine that aids in late-stage red blood cell maturation and helps reduce transfusion burden in adult patients with beta-thalassemia who require regular red blood cell transfusions. Just five months later, Reblozyl was approved for the treatment of anemia in certain patients with myelodysplastic syndromes (MDS), increasing the number of individuals potentially benefiting from this novel therapy by thousands. And most recently, the drug got a nod from regulators in the EU in both indications, expanding its reach outside of the U.S. and to an area of the world where blood disorders, particularly beta-thalassemia, are more prevalent.
Reblozyl was discovered by scientists at Acceleron Pharma, who have spent decades developing the leading TGF-beta-based pipeline in the industry in an effort to benefit patients with both serious and rare diseases. This superfamily of proteins has an incredible capacity to regulate cell growth and repair, with research suggesting that erythropoiesis is regulated by ligands and proteins belonging to TGF-beta.
For a patient population that previously had limited treatment options, Reblozyl is a new opportunity for physicians and patients, transforming the treatment paradigm for individuals dependent on regular red blood cell transfusions.