FDA Approves Kanuma for Lysosomal Acid Lipase Deficiency

Share this content:
FDA Approves Kanuma for Lysosomal Acid Lipase Deficiency
FDA Approves Kanuma for Lysosomal Acid Lipase Deficiency

TUESDAY, Dec. 8, 2015 (HealthDay News) -- Kanuma (sebelipase alfa) has been approved by the U.S. Food and Drug Administration as the first treatment for LAL deficiency (Wolman disease or cholesteryl ester storage disease).

Patients with LAL deficiency produce little or none of the enzyme lysosomal acid lipase (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 two people per million births, often presents during the first two-to-four months of infancy, the FDA said in a news release. Life expectancy depends on the disease's severity and its complications, the agency said.

Kanuma is human LAL that's 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 nine infant clinical trial participants treated with Kanuma, six 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, Conn.-based Alexion Pharmaceuticals.

More Information

Share this content:

is free, fast, and customized just for you!




Already a member?

Sign In Now »

Trending Activities

All Professions

Drug Lookup

Browse drugs by: BrandGenericDisease

More in Home

NIH Panel Will No Longer Review Gene Therapy Experiments

NIH Panel Will No Longer Review Gene Therapy ...

FDA will now assess gene therapy experiments and products as it does with other treatments, drugs

Doctors Remove Contact Lens Embedded in Eyelid for Years

Doctors Remove Contact Lens Embedded in Eyelid for ...

Lens was encapsulated within the upper eyelid soft tissue since childhood eye trauma

CDC: Brucellosis in Dogs Remains a Public Health Risk

CDC: Brucellosis in Dogs Remains a Public Health ...

Infection is under-recognized, may remain a threat without stronger intervention measures

is free, fast, and customized just for you!




Already a member?

Sign In Now »