(HealthDay News) — Kanuma (sebelipase alfa) has been approved by the U.S. Food and Drug Administration as the first treatment for lysosomal acid lipase (LAL) deficiency (Wolman disease or cholesteryl ester storage disease).
Patients with LAL deficiency produce little or none of the enzyme LAL. This causes fat build-up in the body that can lead to liver and cardiovascular disease and other complications. The disease, affecting up to 2 people per million births, often presents during the first 2 to 4 months of infancy, the FDA said in a news release.
Life expectancy depends on the disease’s severity and its complications, according to the agency.
Kanuma is human LAL that is produced by genetically modified chickens in their egg whites. Neither the chicken or its eggs are allowed in the food supply, the FDA said.
The injected drug is given once weekly or once every other week.
Of 9 infant clinical trial participants treated with Kanuma, 6 were alive at 12 months of age, compared with no surviving infants among the 21 given a placebo, the FDA said. The most common human side effects of the drug included diarrhea, vomiting, fever, rhinitis, anemia, cough, headache, constipation and nausea.
No adverse outcomes were noted in the chickens.
Kanuma is produced by Cheshire, Connecticut-based Alexion Pharmaceuticals.
- FDA approves first drug to treat a rare enzyme disorder in pediatric and adult patients [news release]. Silver Spring, MD: U.S. Food and Drug Administration; December 8, 2015. http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm476013.htm. Accessed December 9, 2015.