FDA Grants Approval For New Treatment For Adults With Rare Cancer Diseases

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On Thursday, the FDA approved Besponsa for the treatment of adults with relapsed or refectory B-cell precursor acute lymphoblastic leukemia. “For adult patients with B-cell ALL whose cancer has not responded to initial treatment or has returned after treatment, life expectancy is typically low,” stated Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “These patients have few treatments available and today’s approval provides a new, targeted treatment option.”

B-cell precursor ALL is an aggressive type of cancer that results in the bone marrow makes too many B-call lymphocytes, an immature white blood cell. According to the National Cancer Institute, there are roughly 5,970 Americans that will be diagnosed with ALL in 2017 and almost 1,440 will die from the disease.

Besponsa is a focused therapy that is thought to work by fusing to B-cell ALL cancer cells that express the CD22 antigen, prohibiting the growth of cancerous cells.

Besponsa conducted a random study to determine the safety and efficacy of the treatment, in which 326 patients participated and all received one or two of the last treatments. Patients were chosen at random to receive treatment with Besponsa or an alternative chemotherapy schedule. The trial measured the percentage of patients with no evidence of disease and full recovery of blood counts following treatment (complete remission or CR). Out of the 218 patients that were evaluated, 35.8 percent who received Besponsa experienced CR for an average 8 months; of the patients who was treated alternative chemotherapy, 17.4 percent experienced CR for an average of 5 months.

Normal side effects of Besponsa include low levels of platelets (thrombocytopenia), low levels of specific white blood cells infection, low levels of red blood cells, fatigue, severe bleeding, fever, nausea, headache, low levels of white blood cells with, liver damage, abdominal pain and high levels of bilirubin in the blood.

On the box for the Besponse it includes a warning label that it can cause severe liver damange, including blockage of veins in the liver occurred in some patients who took Besponsa. Other serious side effects of Besponsa entail an drop in blood cell and platelet production, infusion-related reactions and problems with the heart’s electrical pulses. Women who are pregnant or breastfeeding should refrain from taking Besponsa because it may cause damage to a developing fetus or a newborn baby.

The FDA approved this application Priority Review and Breakthrough Therapy. Also, Besponsa also got Orphan Drug designation that provides incentives to help and support the development of drugs for rare diseases. The FDA granted the approval of Besponsa to Pfizer Inc. (PFE).

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