Patients living with Wolman disease may soon have a new treatment option with the U.S. Food and Drug Administration's (FDA) approval of Kanuma (sebelipase alfa).
Wolman disease, also known as lysosomal acid lipase (LAL) deficiency, is a rare disease in which fats build up in the cells of the body's tissues because of reduced or completely absent LAL enzyme activity. The disease often leads to liver and heart disease; it is typically diagnosed when the patient is an infant and progresses rapidly.
The prognosis for the disease is dire — most infants diagnosed with Wolman disease don't survive their first year.
Kanuma contains a recombinant form of human lysosomal acid lipase (rhLAL) protein, extracted from the whites of eggs produced by genetically engineered chickens.
“LAL deficiency is a rare inherited genetic disorder that can lead to serious and life-threatening organ damage, especially when onset begins in infancy,” CDER Director Dr. Janet Woodcock said. “Using this technology, these patients for the first time ever have access to a treatment that may improve their lives and chances of survival.”
Kanuma is administered via a once-weekly, intravenous infusion for those with advanced LAL deficiencies; all other patients receive the medication once every two weeks.
Testing for the drug's safety and effectiveness was done on nine infants. Six of them lived beyond their first birthdays, while all 21 infants in a control group died.
CESD is a form of Wolman disease that occurs later in life and is not as severe. It can still be fatal, though: the life expectancy varies from patient to patient. This condition is rare, affecting just 25 individuals per million births.
In older patients, significant improvement occurred in those treated with Kanuma as compared to those who received a placebo.
Kanuma is considered an orphan drug -- a drug used in the treatment of extremely rare conditions -- and has been granted FDA breakthrough therapy designation because no other treatments for Wolman disease are currently on the market. Kanuma was also granted priority review and its manufacturer, Alexion Pharmaceuticals, Inc., of Cheshire, Connecticut, received a rare pediatric disease priority review voucher.
