U.S. FDA approves cancer drug to treat rare genetic disorder

08:17, November 02, 2010      

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The U.S. Food and Drug Administration (FDA) said Monday that it has approved the cancer drug Afinitor (everolimus) to treat patients with subependymal giant cell astrocytoma (SEGA) associated with tuberous sclerosis ( TS), a rare genetic disorder. This approval was for treatments of SEGA that can not be treated with surgery.

TS causes benign (non-cancerous) tumors to grow in the brain and in other parts of the body including the eyes, lungs, liver, heart, skin and kidneys. TS occurs as a result of genetic mutations that lead to the development of tumors and results in a variety of possible symptoms including learning and developmental disabilities, skin abnormalities, seizures, and lung and kidney disease.

SEGAs are considered a major diagnostic feature of TS and are slow growing tumors, seen in six percent to nine percent of patients. The disease can be fatal for patients who develop complications with tumor growth on the brain. Surgery is used to remove the tumor growths in some patients.

"Patients with this disease currently have limited treatment options beyond surgical intervention," said Richard Pazdur, director of the Office of Oncology Drug Products in the FDA's Center for Drug Evaluation and Research "It is important for research to continue in rare diseases where patients have few or no existing drug treatment options."

Afinitor was first approved in March 2009 to treat kidney cancer. It is marketed by East Hanover, N.J.-based Novartis.

A single study of 28 patients was used to evaluate the safety and effectiveness of Afinitor to treat SEGA. At six months into the study, nine patients (32 percent) had a greater than 50 percent reduction in space the tumor occupied (tumor volume) of their largest SEGA tumor lesion. The length of time from when a patient's tumor visibly shrank and then remained stable (duration of response) for these nine patients ranged from about three months to two and one-half years with a median of 266 days. Seven of these patients retained the greater than 50 percent reduction in space the tumor occupied at time of last follow up.

Patients participating in the study did not develop any new tumors. However, no tumor resolved completely. Four of the patients on this study had previous surgery, but their tumor grew back. After receiving Afinitor, three of these patients experienced greater than 50 percent reduction in tumor volume.

In patients treated with Afinitor for SEGA, the most commonly reported side effects included upper respiratory tract infections, sinus and ear infections, mouth sores, and fever. Common laboratory test abnormalities included liver enzyme elevations, high blood cholesterol and triglycerides (hyperlipidemia), high blood sugar, and decreases in white blood cells, red blood cells ( anemia), and platelets. In patients with kidney cancer (renal cell carcinoma), lung inflammation (pneumonitis) and decreases in kidney (renal) function also have been reported.

Source: Xinhua

(Editor:张茜)

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