Paton_2016_Drugs.Today.(Barc)_52_287

Sebelipase alfa was approved for use in 2015 for patients suffering from lysosomal acid lipase deficiency in either of its two forms. The more severe, early-onset form, Wolman disease, occurs in young infants in whom it is normally fatal within the first year of life. Sebelipase alfa has allowed a small number of such infants to achieve a relatively normal growth rate and to survive for 2 or more years. In older children and adults, the enzyme has corrected their dyslipidemia and produced significant improvement in markers of hepatic function. Important unanswered questions remain, such as to what extent treatment with sebelipase alfa alters the long-term cardiovascular and hepatic consequences of this rare recessive genetic disorder. Further research is also required to determine the true frequency of the disorder in different populations and ethnic groups. The high cost of treatment with sebelipase alfa also poses a very significant obstacle for many health plans.