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Celgene Files for FDA Approval of Blood Disease Drug

Xconomy New York — 

An experimental blood disease drug cited as one of Celgene’s key pipeline assets has been filed for FDA approval.

Summit, NJ-based Celgene (NASDAQ: CELG) said Friday that it submitted the application for luspatercept as a treatment for patients who have anemia, or low red blood cell counts, caused by either of two blood diseases: myelodysplastic syndromes (MDS) or beta thalassemia. Anemia caused by these diseases requires patients to receive regular red blood cell transfusions. Luspatercept was developed to reduce the need for transfusions, and Bristol-Myers Squibb (NYSE: BMY) identified it as one of the close-to-commercialization products that justified its $74 billion deal to acquire Celgene.

MDS is a type of cancer characterized by abnormalities in the blood-forming cells in the bone marrow. These abnormalities lead to low numbers of blood cells, including red blood cells. Beta thalassemia is an inherited disorder that leads to low levels of hemoglobin, the oxygen-carrying protein in red blood cells.

Luspatercept is an injectable drug developed to regulate the late-stage maturation of red blood cells. It originated in the labs of Cambridge, MA-based Acceleron Pharma (NASDAQ: XLRN). Celgene and Acceleron have been drug development partners since 2008.

Celgene and Acceleron say the FDA submission for luspatercept is based on results of Phase 3 studies in both MDS and beta thalassemia. The companies plan to file for approval of the drug in Europe in the second quarter of this year.

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