NICE issues draft guidance for Kanuma (sebelipase alfa) for lysosomal acid lipase deficiency- Alexion Pharma
NICE has published draft guidance as part of its highly specialised technologies programme that recommends further clinical trials are carried out to demonstrate the benefits of Kanuma (sebelipase alfa), from Alexion Pharma, for treating lysosomal acid lipase (LAL) deficiency, a rare inherited genetic disorder.
The draft guidance recommends that the research should be designed to generate robust evidence about the costs and benefits of long-term treatment with sebelipase alfa compared with shorter term treatment (‘bridging therapy’) followed by blood and marrow transplantation (also known as haematopoietic stem cell transplantation) in people diagnosed with rapidly progressive LAL deficiency before they were 6 months old. Sebelipase alfa is not recommended for treating LAL deficiency in people who did not present with rapidly progressive LAL deficiency before they were 6 months old.
Comment: Kanuma was EU approved to treat patients of all ages with lysosomal acid lipase deficiency in September 2015, and approved by the FDA in December 2015.
Learning Zones
The Learning Zones are an educational resource for healthcare professionals that provide medical information on the epidemiology, pathophysiology and burden of disease, as well as diagnostic techniques and treatment regimens.
1 mins