UPDATE: UK's NICE calls for more research into LAL drug Kanuma; Alexion questions the decision

UK drug cost-effectiveness watchdog the National Institute for Health and Care Excellence (NICE) has today published draft guidance as part of its highly specialized technologies program that recommends further clinical trials are carried out to demonstrate the benefits of Kanuma (sebelipase alfa), from Alexion Pharma (Nasdaq: ALXN), 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The draft guidance recommends that the research should be designed to generate robust evidence about the costs and benefits of long-term treatment with sebelipase alfa compared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 hematopoietic stem cell transplantation) in people diagnosed with rapidly progressive LAL deficiency before they were six 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 six months old. Kanuma was approved by the relevant regulatory authorities in the USA and Europe last fall.

A variety of supportive therapies are used to try and slow the progress of the disease, including special diets and drugs for disease complications (for example, statins to reduce cholesterol). Sebelipase alfa is a recombinant human lysosomal acid lipase enzyme replacement therapy and is the only active treatment available that specifically targets the underlying cause of LAL deficiency.

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 sebelipase alfa therapy is £491,992 ($711,319).

Clear need for LAL treatment, says NICE executive

“LAL deficiency can be a devastating condition, particularly where it progresses rapidly in babies less than 6-months old. There is a clear need for a treatment that can stop it progressing and let people with the condition live as normal a life as possible,” commented NICE Health Technology Evaluation Centre director Carole Longson.

Prof Longson continued: “The committee concluded that sebelipase alfa had a treatment effect compared with best supportive care. There was a lack of data on whether it completely reversed LAL deficiency over the long term and prevented complications of the condition. 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.”

Moreover, she said: “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, the use of sebelipase alpha would be likely to represent good value to the NHS.”

“NICE has failed to recognize transformative innovation”