SEBELIPASE ALFA

The PBAC did not recommend the PBS listing of sebelipase alfa (Kanuma®) for the treatment of children and adults with late-onset lysosomal acid lipase deficiency (LAL-D), a rare genetic disease that causes fats to build up in the liver and other organs.

The PBAC acknowledged LAL-D is an ultra-rare disease and effective treatments are needed. The PBAC noted that there is much more variation in late-onset disease compared to infantile-onset LAL-D. Late-onset LAL-D can affect people in different ways including age of onset, symptoms experienced, and how quickly the disease worsens. The PBAC also noted that LAL-D is currently underdiagnosed. The PBAC welcomed inputs from consumers and healthcare professionals and acknowledged the impact of late-onset LAL-D which can include liver and cardiovascular disease, fatigue and impacts on participation in everyday activities.

The PBAC accepted that sebelipase alfa treatment improves laboratory measures including liver enzymes and cholesterol. However, the proposed eligible population in the submission was not clearly defined and did not account for variation in the disease course between patients. The extent of clinical benefit that might be provided by sebelipase alfa was therefore highly uncertain as it depended on which patients with late-onset LAL-D would be treated. Consequently, the total cost and value for money at the price sought by the supplying pharmaceutical company were highly uncertain.  

The PBAC considered that a resubmission should more clearly define the population who should be treated with sebelipase alfa, and account for the variation in the disease course and expected treatment benefit. This information would better inform estimates of the extent and clinical meaningfulness of treatment benefit, value for money and costs.

Sponsor’s Comment: 
The sponsor had no comment.