NICE restricts recommendation for rare disease drug Kanuma

NICE says access to the new rare disease drug Kanuma should be restricted to a small group of patients who will benefit most, and has called for further research to generate data on cost- and clinical-effectiveness.

Alexion's Kanuma (sebelipase alfa) is the first ever treatment for lysosomal acid lipase (LAL) deficiency, a rare inherited genetic disorder, which affects around 25 people in every million.

The condition can present in the first few weeks of life as 'rapidly progressing LAL deficiency', or later in life.

Infants with the condition face life-threatening gastrointestinal and liver problems, and have a median life expectancy of just 3.7 months.

Patients in whom the disease presents in later childhood can also suffer very severe symptoms, but some can manage their condition with a variety of supportive therapies. Special diets and drugs for disease complications, such as statins to reduce cholesterol, can slow the progress of the disease.

In these patients, NICE says Alexion has failed to prove the drug's cost- and clinical-effectiveness.

It says the annual treatment costs for a patient starting treatment at 11 years of age would be £491,992, and these patients could be expected to receive ten years of treatment.

Professor Carole Longson MBE, NICE Health Technology Evaluation Centre Director, said: "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.

"As a consequence the committee did not think that sebelipase alfa represented good value for money to the NHS to be used to treat everyone with LAL deficiency."

Professor Longson said the evidence of Kanuma's benefits for babies with rapidly-progressive LAL deficiency was more compelling.

However 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. Nevertheless, it concluded that Kanuma would be "likely to represent good value to the NHS" in these patients.

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.

A public consultation on the draft guidance will now be open until 10 March 2016, with NICE's committee issuing its next draft on 22 March.

Alexion is expected to reply to the draft guidance later today. The draft guidance on sebelipase alfa can be found on the NICE website here.