NICE Draft Guidance Says BioMarin’s ERT for Batten Disease Not Cost Effective

Rare Daily Staff

The United Kingdom’s National Institute for Health and Care Excellence issued a preliminary recommendation not to recommend that the National Health Service use BioMarin’s enzyme replacement therapy Brineura for a CLN2 disease, a form of Batten disease, saying it does not appear to provide value for money.

CNL2 disease, or neuronal ceroid lipofuscinosis type 2, is a form of Batten disease, a rare, genetic, neurodegenerative disease that is fatal. A deficiency of the enzyme called tripeptidyl peptidase results in a build-up of proteins and lipids in neurons and other cells that prevents them from functioning appropriately. Patients with the condition begin a steep decline in late infancy that includes loss of speech, seizures, loss of mobility, and visual impairment. Children with the condition die between age 8 and early 12. There are an estimated 30 to 50 children in the United Kingdom with the condition with three to six new patients diagnosed each year.

The enzyme replacement therapy carries a list price of $27,900 (£20,107) per 300 mg dose. It’s administered once every two weeks, with a year supply of the therapy costing about $724,000.

“Clinical evidence suggests that, in the short term, [Brineura] improves quality of life, and slows the deterioration of motor and language function,” NICE said in its draft guidance. “However, there is no long-term clinical evidence, so assumptions about long-term disease stabilization and mortality are associated with substantial uncertainty.”

BioMarin said it will provide evidence-based responses to each of the points raised in the committee report over the coming weeks. “Discussions with NICE are still ongoing however, and the committee report allows for the opportunity to put in place a framework with NHS England,” the company said. “BioMarin has previous experience of agreeing frameworks between NICE and NHS England.

The company is also working closely with healthcare professionals and patient groups, such as the Batten Disease Family Association, to provide all requested information to the NICE committee and facilitate the provision of Brineura to eligible patients. 

European regulators approved Brineura for the treatment of CLN2 in June 2017. It is the first and only treatment for CLN2 that has proven to slow down the natural progression of the disease, allowing children to maintain cognitive and motor function for a longer period.

NICE is accepting comments on the preliminary guidance until March 5. A second evaluation committee meeting is scheduled for March 20.

February 12, 2018