The FDA granted a priority review voucher to Alexion Pharmaceuticals’ Kanuma (sebelipase alfa), an enzyme therapy indicated for treating lysosomal acid lipase deficiency (LAL-D).
LAL-D, a rare genetic and progressive metabolic disease that causes multi-organ damage in infant, pediatric and adult patients, can have sudden and unpredictable clinical complications.
Approximately half of children and adults with the condition go on to experience fibrosis, cirrhosis or liver transplant within three years of the disease’s onset.
The most common adverse reactions in patients with rapidly progressive disease in the first six months of life include diarrhea, vomiting, anemia, coughing, rhinitis, nasopharyngitis and urticaria.
