The National Institute for Health and Care Excellence has published a final evaluation determination rejecting the use of Alexion's Kanuma to treat infants, children and adults with the rare inherited genetic disorder lysosomal acid lipase deficiency (LAL-D).
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, leading to multi-organ damage and potential premature death.
Alexion said it is disappointed with the decision, noting that infants with LAL-D normally do not live to see their first birthday without treatment. In children and adults with LAL-D, around 50 percent could progress to liver complications such as fibrosis or cirrhosis or need a liver transplant within three years of starting symptoms.
In clinical trials with Kanuma (sebelipase alfa), five out of nine infants survived beyond three years of age, achieving normal developmental milestones, the firm noted. But NICE was not convinced that the high cost of the drug - nearly £500,000 per patient - could be justified by its long-term treatment benefits.
"We are particularly devastated by the announcement from NICE to deny babies with LAL D access to life saving therapy," said Christine Lavery, group chief executive of the Society for Mucopolysaccharide Diseases. "These babies need immediate access to treatment following diagnosis. Without treatment children will unquestionably die a cruel and inhumane death within days or weeks".
"Should a right to life be denied when there is a treatment that not only demonstrates clinical benefit but allows children to thrive, grow, meet their developmental milestones and have a normal childhood," she argues.
According to Alexion, NICE has "disregarded" proposed strategies to manage the cost of Kanuma and the expert clinical opinion reflected in the proposed consensus MAA, which was developed alongside expert physicians, patient groups, and NHS England, which it claims "identified the patients with the greatest clinical need for treatment with Kanuma".
"NICE's decision to not recommend Kanuma for the treatment of patients with LAL-D is an injustice to patients in England and based on arbitrary cost-effectiveness measures. This decision, if not reversed, could result in devastating consequences for patients in England with LAL-D who may face significant suffering, multi-organ damage and potential premature death," the firm said in a statement.
"Once again, the UK lags behind other countries in funding medicines for patients suffering from rare and ultra-rare diseases with limited treatment options. The NICE Highly Specialised Technology (HST) process is proving to be both inconsistent and ineffective in assessing the significant medical and human benefits of a treatment for patients with a severe and devastating rare disease for which there are no other treatment options".
But defending the decision, Professor Carole Longson, NICE health technology evaluation centre director, said: "Throughout this extremely complicated evaluation we have given the company many opportunities to improve the terms under which sebelipase alfa could be offered to patients. However, even their best offer to date falls far short given the considerably uncertainties about its longer term benefits and its very high cost."
Alexion said it will now evaluate all potential procedural and other options available so that patients with LAL-D in England can have access to Kanuma, the only approved therapy for the disease.