U.S. FDA Approves Use Of Transgenic Chickens To Produce A Drug For Treatment Of Rare Enzyme Disorder

FDA front building — The U.S. FDA has approved Kanuma as a first line of treatment for patients with a rare enzyme disease called LAL deficiency. Reuters/Jason Reed

The United States Food and Drug Administration (FDA) has approved the use of transgenic chickens to produce a drug called Kanuma (sebelipase alfa) for treatment of people with a rare disease called lysosomal acid lipase (LAL) deficiency.

People with LAL deficiency, also known as cholesteryl ester storage disease (CESD) or Wolman disease, have limited or not LAL enzymatic activity. As a result, fat gets accumulated in cells belonging to different tissues. The extra deposition of fat might lead to serious health conditions, including cardiovascular disorders and fatty liver disease.

The Center for Veterinary Medicine (CVM), an FDA Center, approved the use of recombinant DNA technology to create genetically modified chickens. The GE chickens are able to produce human lysosomal acid lipase (rhLAL) protein in their egg whites.

Kanuma is then extracted from the egg whites, provided that the purified form is effective and safe for use in people with LAL deficiency. The approval for the use of Kanuma as a human therapeutic came from The Center for Drug Evaluation and Research (CDER), another FDA Center.

“LAL deficiency is a rare inherited genetic disorder that can lead to serious and life-threatening organ damage, especially when onset begins in infancy,” said CDER Director Janet Woodcock, in a press release. “Using this technology, these patients for the first time ever have access to a treatment that may improve their lives and chances of survival.”

The rhLAL protein derived from chicken's egg white functions as inactive or partially active LAL enzyme in people with its deficiency. Kanuma is administered to the patients through intravenous infusion once in a week.

LAL deficiency is a rapidly progressive disease that often affects infants. Patients with severe form of the disease do not survive beyond the first year of life. CESD affects 25 infants in nearly one million births and Wolman disease affects one or two infants per million births.