Regulatory Focus™ > News Articles > FDA Approves Orphan Drug Application for Pfizer CML Drug

FDA Approves Orphan Drug Application for Pfizer CML Drug

Posted 05 September 2012 | By Alexander Gaffney, RAC 

The US Food and Drug Administration (FDA) has approved Pfizer's new orphan drug application for Bosulif (bosutinib), a tyrosine kinase inhibitor used to treat chronic myelogenous leukemia (CML).

CML is a bone marrow and blood disorder that affects more than 5,000 adults in the US each year, and is generally associated with patients with a chromosomal abnormality known as the Philadelphia chromosome. Patients with this chromosome over-produce abnormal white blood cells, which prevent the body from adequately fighting off infections.

Bosulif treats this condition by blocking tyrosine kinase signals that would otherwise cause the malformed white blood cells to be produced, but is intended only as an advanced-stage or last-resort treatment for patients unable to tolerate other CML therapies.

Richard Pazdur, director of FDA's Office of Hematology and Oncology Products (OHOP), said in a statement that understanding the root causes of CML have allowed drug manufacturers to develop newer and better drugs to treat the condition. Bosulif is the fourth CML drug to be approved since 2001, FDA noted.

The drug was approved on 4 September 2012 based on a 546-person clinical trial with chronic, accelerated or blast phase CML who either did not respond to or could not tolerate other CML therapies. All patients received Bosulif, and 51.4 percent of patients experienced a major cytogenetic response lasting at least nine months. Thirty-three percent of patients achieved a complete hematologic response, and a further 55% achieved normal blood counts with no evidence of leukemia. The most common side effects to treatment included diarrhea, nausea, anemia, fever and vomiting.

Read more:

FDA - FDA approves new orphan drug for chronic myelogenous leukemia

FDA - Bosutinib tablets

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