Alexion fails to obtain NICE approval for Kanuma

Alexion's infantile genetic disorder therapy Kanuma has been rejected by the National Institute for Health and Care Excellence (NICE), as the healthcare body believes it is not cost-effective enough for widespread use.

Kanuma is intended to treat patients with the inherited genetic condition lysosomal acid lipase deficiency (LAL-D), many of whom do not currently live beyond their first birthday, PharmaTimes reports.

Clinical trials involving Kanuma (sebelipase alfa) showed that it had potential life-lengthening benefits for infants diagnosed with the rare disorder, with five out of nine study subjects living for three years or longer after taking the drug.

Patients with LAL-D do not have the LAL enzyme present in their bodies. LAL is needed to break down fats in the liver, meaning that those with the disease are at greater risk of developing life-threatening liver complications as a result. Kanuma was found to delay these effects, improving quality of life for more than half of patients in an initial trial.

However, at a cost of £500,000 per patient for a course of the drugs, NICE deemed it insufficiently cost-effective at present.

Christine Lavery, group chief executive of the Society for Mucopolysaccharide Diseases, said this ruling was "devastating".

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