NICE’s Highly Specialised Technology committee has approved cerliponase alfa (Brineura) for children with neuronal ceroid lipofuscinosis type 2 (CLN2), as part of a managed access agreement.
The committee noted that although cerliponase alfa is not a cure for CLN2, it is an important development for treating the condition, and it has shown substantial short-term benefits in slowing the rate of progression.
The enzyme replacement therapy consists of recombinant human tripeptidyl peptidase 1. It is expected to restore deficient tripeptidyl peptidase 1 (TPP1) activity in the brain caused by the genetic mutation.
Analyses show that in two-thirds of patients who received cerliponase alfa, their condition stabilised at 48 weeks. Overall, there was significantly less decline in patients who received cerliponase alfa than those who did not.
For some people, the treatment may lead to long-term disease stabilisation and a near normal life expectancy. However, only short-term clinical evidence is available at the moment, so assumptions about long-term outcomes are uncertain.
Over the next few weeks, NICE will be working with NHS England, the pharmaceutical company, clinicians and the Batten Disease Family Association on the details of the managed access agreement.
Cerliponase alfa is administered into the cerebrospinal fluid by infusion via a surgically implanted intracerebroventricular access device. Due to the complex nature of the administration of the drug, NHS England is also working with potential providers to ensure that they have capacity to deliver the treatment to patients.
It is estimated that there are around 30 to 50 children living with CLN2 in the UK.