NICE has published draft guidance for Alexion’s rare disease drug Kanuma (sebelipase alfa), rejecting it in one of its indications and requesting more evidence in another.
Kanuma treats the rare inherited genetic disorder lysosomal acid lipase (LAL) deficiency. LAL is an enzyme that is responsible for breaking down fats in a part of the cell called the lysosome. Since the LAL enzyme is missing, or deficient, the fats build up in cells primarily in the liver, gastrointestinal and cardiovascular systems.
Based on the average yearly cost over 10 years for a patient starting treatment at 11 years of age, the total cost per person per year of treatment with Kanuma is £491,992, which the NICE committee considered too high.
“The committee considered that, even based on more optimistic assumptions of long-term treatment effect, the cost of sebelipase alfa would be very high, and that it would be higher relative to treatment benefits than it had previously regarded as acceptable,” said Carole Longson, Director of the NICE Health and Technology Evaluation Centre. “As a consequence the committee did not think that it represented good value for money to the NHS to be used to treat everyone with LAL deficiency.”
However, Longson added that the evidence of the benefits of sebelipase alfa for babies with rapidly progressive LAL deficiency was more compelling: “The committee concluded that more research was needed to explore the potential benefits of using sebelipase alfa in babies to stabilise their condition before undertaking a bone marrow stem cell transplant aimed at curing the disease. In these circumstances, it would be likely to represent good value to the NHS.”
As such, the draft guidance calls for more evidence about the costs and benefits of long-term treatment with Kanuma compared with shorter term treatment followed by blood and marrow transplantation in people diagnosed with rapidly progressive LAL deficiency before they were 6 months old.
Rapidly progressing LAL deficiency in babies is usually diagnosed within the first weeks of life. Survival is less than 12 months and the median life expectancy of a baby with rapidly progressing LAL is 3.7 months.
In a statement, Alexion said that it “believes NICE has failed to recognise the transformative clinical innovation of Kanuma for patients with LAL-D”.
“NICE made this initial decision despite the robust medical evidence that shows a major survival benefit in babies and a significant improvement in multiple liver parameters in children and adults with LAL-D.
“The recommendations from NICE ignores the severity of LAL-D and the life-transforming benefits of Kanuma for patients of all ages.
“Alexion is concerned by the NICE recommendation that more research be conducted to compare the benefits of long-term treatment with Kanuma to shorter-term treatment followed by haematopoietic stem cell transplant (HSCT). HSCT is not an effective option for patients with LAL-D, and data has shown that infants still die within the first year of life.”
The company added that Kanuma is the only approved treatment and addresses the underlying cause of LAL-D.