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FDA approves Kanuma, the first treatment for patients with lysosomal acid lipase.
On Dec. 8, 2015, FDA approved Kanuma (sebelipase alfa) as the first treatment for patients with a rare disease known as lysosomal acid lipase (LAL) deficiency, produced by Alexion Pharmaceuticals.
FDA granted Kanuma orphan drug designation because it treats a rare disease affecting fewer than 200,000 patients in the United States. The drug was granted breakthrough therapy designation, as it is the first and only treatment available for Wolman disease. The Kanuma application was also given a priority review, which is granted to drug applications that show a significant improvement in safety or effectiveness in the treatment of a serious condition. The drug’s manufacturer was granted a rare pediatric disease priority review voucher––a provision intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.
Patients with LAL deficiency (also known as Wolman disease and cholesteryl ester storage disease [CESD]) have little or no LAL enzyme activity. This results in a build-up of fats within the cells of tissues that can lead to liver and cardiovascular disease.
Wolman disease presents during infancy and is rapidly progressive. Patients with the disease rarely survive beyond the first year of life. CESD is a milder, later-onset form of LAL deficiency, and presents in early childhood or later. Life expectancy of patients with CESD depends on the severity of the disease and associated complications. Kanuma can be used to treat patients with Wolman disease and CESD.
Kanuma is produced in the egg whites of genetically engineered chickens. The chickens contain an rDNA construct producing rhLAL protein in their egg whites. These egg whites are then refined to extract the rhLAL protein that is eventually used to produce Kanuma.
“We reviewed all of the data to ensure that the hens do produce rhLAL in their egg whites, without suffering any adverse health effects from the introduced rDNA construct,” said CVM Director Bernadette Dunham, DVM, PhD. “The company has taken rigorous steps to ensure that neither the chickens nor the eggs will enter the food supply, and we have confirmed their containment systems by inspecting the manufacturing facilities.”