New enzyme replacement therapies launched

Patients with two rare inherited metabolic conditions can now benefit from treatment with specific enzyme replacement therapies.

Kanuma (sebelipase alfa) is licensed for use in patients with lysosomal acid lipase deficiency, a condition causing chronic build-up of cholesteryl esters and triglycerides in the liver, blood vessel walls and other tissues, leading to multi-organ damage and premature death. 

Further information
View Kanuma drug record
View Strensiq drug record
Manufacturer: Alexion

Strensiq (asfotase alfa) is indicated for the treatment of paediatric-onset hypophosphatasia, a disorder caused by low levels of alkaline phosphatase and characterised by defective bone mineralisation that can lead to bone deformity and destruction, muscle weakness, seizures, respiratory failure and premature death.