Drug for children with rare inherited condition to be made available on NHS

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, 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.”

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.

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