Children with rare inherited condition to benefit from drug through managed access agreement

12 Sep 2019 01:10 PM

It is estimated that in the UK there are around 30 to 50 children living with the condition.

A drug which slows the decline of a rare inherited condition affecting children is to be made available on the NHS.

NICE’s Highly Specialised Technology committee is supporting a positive recommendation for cerliponase alfa (Brineura, BioMarin) for children with neuronal ceroid lipofuscinosis type 2 (CLN2) – a very rare inherited condition affecting between one and six babies each year in the UK – in the context of a managed access agreement.

Cerliponase alfa is an enzyme replacement therapy administered directly into the brain via a surgically implanted permanent access device.

The independent committee noted that although cerliponase alfa is not a cure for CLN2 disease, it is an important development for treating the condition, and that it has shown substantial short-term benefits in slowing the rate at which it progresses.

Meindert Boysen, director of the Centre for Health Technology Evaluation at NICE, yesterday said:

“This treatment shows great promise in slowing the progression of this devastating condition to allow children to enjoy normal childhood activities for longer which is so important.

“We are pleased to be able to make this early announcement, realising that families have been anxiously waiting for news, while we are carefully working through the details necessary to finalise the managed access agreement.”

Health Secretary Matt Hancock yesterday said:

“I’m absolutely delighted this new treatment will be funded by the NHS, giving families dealing with the devastating impact of Batten disease renewed hope for a better quality of life for their child.

“Through our Long Term Plan, we want all patients to have access to the most pioneering, value for money medicines - this is a great example of how we can work with industry to get treatments to patients as quickly as possible.

“This can only be afforded thanks to the £33.9bn extra we are putting into the NHS, paid for thanks to our strong economy.”

Over the next weeks, NICE will be working with NHS England, BioMarin, clinicians and the Batten Disease Family Association on the details of the managed access agreement. A managed access agreement describes the patient eligibility criteria for access, as well as stopping rules and data collection arrangements. The agreement is likely to include all new patients with CLN2 except those with advanced disease and very low motor and language scores of one or less, because they would be unlikely to benefit.

The full details of the committee discussion and recommendation following its meeting on 29 August 2019 will be provided in the Final Evaluation Document. In line with NICE processes, the recommendation and documentation is subject to internal sign off procedures.

CLN2, also known as Batten disease, is a progressive condition caused by the deficiency of the enzyme tripeptidyl peptidase 1. This results in the abnormal storage of proteins and lipids in neurons and other cells, preventing them from functioning normally.

Symptoms in children with CLN2 begin with developmental delays from around the age of two and can then progress rapidly with the onset of seizures, decline in speech, loss of mobility, involuntary muscle spasms, progressive dementia and visual impairment leading to blindness.

The majority of children with CLN2 live to between eight years and early adolescence; the average life expectancy is 10 years. It is estimated that in the UK there are around 30 to 50 children living with the condition.

There is currently no cure or life-extending treatments for CLN2 and clinical management is limited to symptom relief and supportive and palliative care.

Analyses show that in two thirds of patients who had cerliponase alfa their condition stabilised at 48 weeks. Overall there was significantly less decline than people who had not had cerliponase alfa.

For some people, cerliponase alfa may lead to long-term disease stabilisation and a near normal life expectancy. However, there is only short-term clinical evidence, so assumptions about long-term disease stabilisation and mortality are uncertain.

Due to the complex nature of the administration of the drug, NHS England is working with potential providers to ensure that they have capacity to deliver the treatment to patients.

Each patient will require a detailed assessment and consent process prior to starting the treatment and that needs to be factored into the planning.

NICE will provide a further update on the expected publication date as soon as we can.