Sebelipase alfa (Kanuma) has been recommended for approval to treat lysosomal acid lipase deficiency in infants, children, and adults, according to a press release from the European Medicine Agency.
Lysosomal acid lipase breaks down fatty material, and LAL deficiency causes a buildup of fatty material in important body organs such as the liver and blood vessels. Patients with LAL deficiency can experience symptoms such as growth failure, enlarged liver, diarrhea, and malabsorption. LAL deficiency is inherited, and is life threatening in its most severe form, especially in infants under 1 year old.
Sebelipase alfa is a long-term enzyme replacement therapy for all patients with LAL deficiency. In four small studies of 106 patients, sebelipase alfa proved effective at replacing the missing enzyme, and improvement was seen in several areas. Infant survival was improved, which is important as no treatment option for infants with LAL deficiency existed before.
Common side effects of the treatment include allergic reaction, transient hyperlipidemia, and development of antidrug antibodies. Any patients, particularly infants, showing signs of hypersensitivity should be monitored further.
Read the full press release on the European Medicine Agency’s website.
