Drug for Lysosomal Acid Lipase Deficiency Wins FDA Nod

The US Food and Drug Administration (FDA) gave a green signal to Alexion Pharmaceutical Inc’s drug on Tuesday to treat rare and fatal genetic disorder.

This incredibly rare disease occurs when the body does not produce enough active lysosomal acid lipase (LAL or LIPA) enzyme, which is essential for breaking down fatty material in the body. This results in a build-up of fats within the cells of various tissues, which can lead to liver and cardiovascular disease and other complications. “In clinical studies, 67% of infants who received enzyme replacement therapy survived beyond 12 months of age, and children and adults had meaningful improvements in multiple disease-related liver and lipid abnormalities”. CESD is a milder, later-onset form of LAL deficiency and presents in early childhood or later. The life expectancy of patients with CESD depends on the severity of the disease and on associated complications. Wolman disease affects one to two infants per million births, and CESD affects 25 individuals per million births.

Kanuma is an enzyme replacement therapy that is a recombinant form of the human LAL protein (rhLAL).

Kanuma is developed from the egg whites of genetically engineered chickens and required approval from both the FDA’s veterinary division and its normal drug evaluation division. The whites are then refined and the rhLAL protein extracted and purified.

The second trial, double-blinded and placebo-controlled, involved 66 pediatric and adult patients with CESD.

Six of the nine Wolman disease patients survived to 1 year of age, the FDA said, compared with none in a cohort of historical controls.

The most common human side effects of the drug included diarrhea, vomiting, fever, nasal inflammation, anemia, cough, headache, constipation and nausea. The FDA says that the chickens are not likely to accidentally enter the food supply or adversely affect the environment, because they are raised in indoor facilities. “No adverse outcomes were noted in the chickens”, the agency said. “We look forward to bringing Kanuma to patients with LAL-D and their physicians in the United States”.

At the time, Kanuma was already under priority review with the FDA and had won accelerated consideration of its marketing authorization application by the EMA, where it was subsequently approved. The manufacturer of Kanuma was granted a rare pediatric disease priority review voucher — a provision meant to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

The Cheshire, Connecticut-based company’s shares were up 3.6 percent at $185.7 on the Nasdaq on Tuesday.

In addition to the approvals in the US and European Union, the new drug application for Kanuma is under review with the Japan’s Ministry of Health, Labour and Welfare.