FDA approves Kanuma (sebelipase alfa) to treat lysosomal acid lipase deficiency- Alexion Pharma

Alexion Pharmaceuticals has announced that the FDA has approved Kanuma (sebelipase alfa) for the treatment of patients of all ages with a diagnosis of lysosomal acid lipase deficiency (LAL-D). Kanuma, an enzyme replacement therapy (ERT), is the first therapy approved in the USA for the treatment of patients with LAL-D, a genetic and progressive ultra-rare metabolic disease in which patients suffer multi-organ damage and premature death.

LAL-D (also known as Wolman disease and cholesteryl ester storage disease) is a genetic, chronic, and progressive metabolic disease associated with significant morbidity and premature mortality. It is an ultra-rare disease, which is defined as a disease that affects fewer than 20 patients per one million of the general population. Patients with LAL-D can experience a rapid onset of life-threatening disease manifestations, and without treatment, the youngest patients with LAL-D face rapid disease progression that is typically fatal within a matter of months. In addition, similar to other liver diseases, many patients may be asymptomatic until they experience a severe consequence of the disease.

LAL-D is caused by genetic mutations that result in a marked decrease or loss in LAL enzyme activity in the lysosomes across multiple body tissues, leading to the chronic build-up of cholesteryl esters and triglycerides in the liver, blood vessel walls, and other organs.

The approval of Kanuma was based on data from two clinical studies and a supporting open-label extension study comprising infant, paediatric, and adult patients with LAL-D. Study results showed significant benefit in terms of survival (67%, or 6 out of 9) in patients with the infant form of LAL-D beyond 12 months, compared with 0 out of 21 patients in an untreated historical cohort. In paediatric and adult patients with LAL-D (ages 4 to 58 years), treatment with Kanuma resulted in larger reductions from baseline in ALT values and liver fat content, as measured by MRI, compared to treatment with placebo. Reduced ALT values were generally seen within 2 weeks. In addition, treated patients had significant improvements in lipid parameters, including LDL-C, HDL-C, non-HDL-C, and triglycerides, compared to placebo. The significance of these findings as they relate to cardiovascular morbidity and mortality or progression of liver disease in LAL deficiency has not been established. Continued improvements in ALT, LDL-C and HDL-C were seen in patients treated with Kanuma for up to 36 weeks.

Alexion will offer support to patients with LAL-D through its OneSource programme. OneSource provides each patient and family with personalised support from a dedicated nurse case manager, who can help patients understand their insurance benefits and receive reimbursement assistance. Through OneSource, patients and families can obtain further information regarding third-party foundations and co-pay assistance programs that help patients meet out-of-pocket expenses related to the treatment of LAL-D. For uninsured patients who have no access to insurance, the Alexion Access Foundation, a charitable entity, provides Kanuma free of charge for patients. Kanuma will become available commercially during the first week of January 2016.