Acceleron receives orphan drug designations

Cambridge biopharmaceutical company Acceleron Pharma Inc. said Tuesday that the Food and Drug Administration granted orphan drug designation for ACE-536 as a potential treatment for two rare blood disorders.

The disorders are beta-thalassemia and myelodysplastic syndromes, the company said in a press release.

Orphan designation is granted to advance the evaluation and development of therapies for the treatment of rare diseases or conditions affecting fewer than 200,000 people in the US. A company that receives orphan status can qualify for tax advantages as well as for seven years of market exclusivity in the United States following marketing approval of the treatment by the FDA.

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The granting of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval.

In a statement, Acceleron chief medical officer Matthew Sherman M.D. said: “ACE-536 has shown the potential to address a significant unmet medical need in the treatment of these rare hematological disorders. We are excited about the prospect of bringing a new therapeutic option to these underserved patient populations and continue to work closely with our clinical investigators on both of our recently initiated phase 2 clinical studies of ACE-536, one in patients with beta-thalassemia and a second in patients with MDS.”

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